EA201190306A1 - Улучшенная липидная композиция - Google Patents

Улучшенная липидная композиция

Info

Publication number
EA201190306A1
EA201190306A1 EA201190306A EA201190306A EA201190306A1 EA 201190306 A1 EA201190306 A1 EA 201190306A1 EA 201190306 A EA201190306 A EA 201190306A EA 201190306 A EA201190306 A EA 201190306A EA 201190306 A1 EA201190306 A1 EA 201190306A1
Authority
EA
Eurasian Patent Office
Prior art keywords
lipid composition
improved lipid
lipid
formula
improved
Prior art date
Application number
EA201190306A
Other languages
English (en)
Other versions
EA024960B1 (ru
Inventor
Акин Акинг
Джозеф Р. Доркин
Сяоцзюнь Цинь
Вильям Кентли
Мутиах Манохаран
Каллантоттатил Г. Раджив
Джаяпракаш К. Нараянаннаир
Мутусами Джаяраман
Цзяньсинь Чэнь
Стивен Энселл
Original Assignee
Элнилэм Фармасьютикалз, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Элнилэм Фармасьютикалз, Инк. filed Critical Элнилэм Фармасьютикалз, Инк.
Publication of EA201190306A1 publication Critical patent/EA201190306A1/ru
Publication of EA024960B1 publication Critical patent/EA024960B1/ru

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A61K47/551—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1277—Preparation processes; Proliposomes
    • A61K9/1278—Post-loading, e.g. by ion or pH gradient
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C227/00—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C227/14—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton from compounds containing already amino and carboxyl groups or derivatives thereof
    • C07C227/18—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton from compounds containing already amino and carboxyl groups or derivatives thereof by reactions involving amino or carboxyl groups, e.g. hydrolysis of esters or amides, by formation of halides, salts or esters
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C227/00—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C227/38—Separation; Purification; Stabilisation; Use of additives
    • C07C227/40—Separation; Purification
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C29/00—Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom not belonging to a six-membered aromatic ring
    • C07C29/58—Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom not belonging to a six-membered aromatic ring by elimination of halogen, e.g. by hydrogenolysis, splitting-off
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D475/00—Heterocyclic compounds containing pteridine ring systems
    • C07D475/02—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4
    • C07D475/04—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Emergency Medicine (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)

Abstract

Изобретение относится к катионному липиду формулы (1) улучшенной липидной композиции, содержащей катионный липид формулы I, и соответствующим способам применения. Также описаны нацеливающие липиды и специфичные липидные композиции, содержащие указанные нацеливающие липиды.
EA201190306A 2009-06-10 2010-06-10 Улучшенная липидная композиция EA024960B1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US18580009P 2009-06-10 2009-06-10
US24483409P 2009-09-22 2009-09-22
PCT/US2010/038224 WO2010144740A1 (en) 2009-06-10 2010-06-10 Improved lipid formulation

Publications (2)

Publication Number Publication Date
EA201190306A1 true EA201190306A1 (ru) 2013-01-30
EA024960B1 EA024960B1 (ru) 2016-11-30

Family

ID=43309230

Family Applications (3)

Application Number Title Priority Date Filing Date
EA201791744A EA201791744A3 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201690312A EA028860B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201190306A EA024960B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция

Family Applications Before (2)

Application Number Title Priority Date Filing Date
EA201791744A EA201791744A3 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201690312A EA028860B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция

Country Status (24)

Country Link
US (4) US8158601B2 (ru)
EP (2) EP3431076B1 (ru)
JP (4) JP5819291B2 (ru)
KR (7) KR20220038506A (ru)
CN (2) CN104873464B (ru)
AU (4) AU2010259984B2 (ru)
CA (2) CA3014827A1 (ru)
CY (2) CY1120641T1 (ru)
DK (2) DK2440183T3 (ru)
EA (3) EA201791744A3 (ru)
ES (2) ES2901627T3 (ru)
HR (2) HRP20181221T1 (ru)
HU (2) HUE038796T2 (ru)
IL (4) IL216876A (ru)
LT (2) LT3431076T (ru)
MX (4) MX367665B (ru)
NZ (3) NZ622843A (ru)
PL (2) PL2440183T3 (ru)
PT (2) PT2440183T (ru)
SG (3) SG10201403054SA (ru)
SI (2) SI3431076T1 (ru)
SM (2) SMT201800499T1 (ru)
TR (1) TR201811076T4 (ru)
WO (1) WO2010144740A1 (ru)

Families Citing this family (946)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
US7605251B2 (en) 2006-05-11 2009-10-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2713379A1 (en) * 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
CN105267233B (zh) 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
US8546554B2 (en) 2008-09-25 2013-10-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
EP2937418B1 (en) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
EP2373382B1 (en) 2008-12-10 2017-02-15 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
CA2754043A1 (en) * 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
KR20220038506A (ko) * 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US9051567B2 (en) * 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
US8859516B2 (en) 2009-09-15 2014-10-14 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
SI3354657T1 (sl) 2009-09-22 2022-05-31 Medicago Inc. Postopek za pripravo iz rastlin izpeljanih beljakovin
JP5723378B2 (ja) * 2009-11-03 2015-05-27 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． トランスサイレチン（ｔｔｒ）を阻害する脂質製剤化組成物及び方法
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
US20130017223A1 (en) * 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
US8846631B2 (en) 2010-01-14 2014-09-30 Regulus Therapeutics Inc. MicroRNA compositions and methods
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
PT3243526T (pt) 2010-07-06 2020-03-04 Glaxosmithkline Biologicals Sa Distribuição de arn para despoletar múltiplas vias imunitárias
US10487332B2 (en) 2010-07-06 2019-11-26 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of RNA
CN103153284B (zh) 2010-07-06 2015-11-25 诺华股份有限公司 含有其pKa值有利于RNA递送的脂质的脂质体
WO2012016188A2 (en) * 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RU2745848C2 (ru) 2010-08-17 2021-04-01 Сирна Терапьютикс, Инк. ОПОСРЕДУЕМОЕ РНК-ИНТЕРФЕРЕНЦИЕЙ ИНГИБИРОВАНИЕ ЭКСПРЕССИИ ГЕНОВ ВИРУСА ГЕПАТИТА B (HBV) С ПРИМЕНЕНИЕМ МАЛОЙ ИНТЕРФЕРИРУЮЩЕЙ НУКЛЕИНОВОЙ КИСЛОТЫ (миНК)
WO2012027467A1 (en) 2010-08-26 2012-03-01 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF PROLYL HYDROXYLASE DOMAIN 2 (PHD2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP5911870B2 (ja) 2010-08-31 2016-04-27 ノバルティス アーゲー 免疫原をコードするｒｎａの送達のためのｐｅｇ化リポソーム
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
ES2987915T3 (es) 2010-09-20 2024-11-18 Sirna Therapeutics Inc Nuevos lípidos catiónicos de bajo peso molecular para el suministro de oligonucleótidos
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR102266691B1 (ko) * 2010-10-11 2021-06-23 노파르티스 아게 항원 전달 플랫폼
WO2012054365A2 (en) 2010-10-21 2012-04-26 Merck Sharp & Dohme Corp. Novel low molecular weight cationic lipids for oligonucleotide delivery
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
DK2635265T3 (en) 2010-11-05 2018-07-16 Sirna Therapeutics Inc New low molecular weight cyclic amine-containing cationic lipids for oligonucleotide delivery
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
TWI620816B (zh) 2011-03-23 2018-04-11 苜蓿股份有限公司 植物衍生蛋白回收方法
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012151575A2 (en) * 2011-05-05 2012-11-08 Duke University A method of controlling coagulation
WO2012159164A1 (en) * 2011-05-23 2012-11-29 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EP2723351B1 (en) 2011-06-21 2018-02-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP2723756B1 (en) * 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
JP2014522842A (ja) 2011-07-06 2014-09-08 ノバルティス アーゲー 免疫原性組み合わせ組成物およびその使用
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
UA118649C2 (uk) 2011-11-18 2019-02-25 Елнілем Фармасьютікалз, Інк. ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)
DE21212055T1 (de) 2011-12-07 2022-08-04 Alnylam Pharmaceuticals, Inc. Biologisch abbaubare lipide zur freisetzung von wirkstoffen
WO2013086322A1 (en) * 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US9352042B2 (en) * 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501513B2 (en) * 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
WO2013158579A1 (en) 2012-04-19 2013-10-24 Merck Sharp & Dohme Corp. Novel diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9334498B2 (en) 2012-05-10 2016-05-10 Uab Research Foundation Methods and compositions for modulating MIR-204 activity
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
US20140120157A1 (en) * 2012-09-19 2014-05-01 Georgetown University Targeted liposomes
AU2013380825B2 (en) * 2012-10-16 2017-07-20 Biomics Biotechnologies Co., Ltd. Lipidosome preparation, preparation method and application thereof
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
BR112015013105B1 (pt) 2012-12-05 2022-02-08 Alnylam Pharmaceuticals, Inc Agente de rnai de fita dupla capaz de inibir a expressão de pcsk9, seus usos, composição farmacêutica e método de inibição da expressão de pcsk9 em uma célula in vitro
AU2013355258A1 (en) * 2012-12-07 2015-06-11 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
EP3434774A1 (en) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US20160024181A1 (en) * 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
EP3932947A1 (en) 2013-03-14 2022-01-05 Translate Bio MA, Inc. Methods and compositions for delivering mrna coded antibodies
KR102311614B1 (ko) 2013-03-14 2021-10-08 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
CN105143456A (zh) * 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
ES2967701T3 (es) 2013-03-15 2024-05-03 Translate Bio Inc Mejora sinergística de la entrega de ácidos nucleicos mediante formulaciones mezcladas
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
EP2968147A4 (en) * 2013-03-15 2017-02-22 The Penn State Research Foundation Acid stable liposomal compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
AU2014268529C1 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
TWI664187B (zh) 2013-05-22 2019-07-01 阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
WO2015048020A2 (en) * 2013-09-24 2015-04-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for the manufacture of lipid nanoparticles
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
SI3071696T1 (sl) 2013-11-22 2019-11-29 Mina Therapeutics Ltd C/EBP alfa kratko delujoči RNA sestavki in postopki uporabe
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
EP3093283B1 (en) 2014-01-09 2018-12-19 Eisai R&D Management Co., Ltd. Cationic lipid
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
EP4729528A3 (en) 2014-04-25 2026-06-24 Translate Bio, Inc. Methods for purification of messenger rna
US9926556B2 (en) 2014-04-28 2018-03-27 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2015168589A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
CR20160554A (es) 2014-05-01 2017-03-07 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión del factor b del complemento
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
RU2016144908A (ru) 2014-05-02 2018-06-05 Серени Терапеутикс Холдинг Са Маркеры hdl-терапии
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
WO2015184256A2 (en) 2014-05-30 2015-12-03 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2016004318A1 (en) 2014-07-02 2016-01-07 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
KR102410072B1 (ko) * 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
TWI864340B (zh) 2014-10-10 2024-12-01 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3209780A4 (en) 2014-10-24 2018-09-19 University of Maryland, Baltimore Short non-coding protein regulatory rnas (sprrnas) and methods of use
EP3904519A1 (en) * 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
TWI769565B (zh) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
AU2015357562B2 (en) 2014-12-05 2021-10-21 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
CA2969664C (en) 2014-12-26 2022-06-21 Eisai R&D Management Co., Ltd. Cationic lipid
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
EP3270894B1 (en) 2015-03-19 2021-02-24 Translate Bio, Inc. Mrna therapy for pompe disease
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
BR112017021053A2 (en) 2015-04-13 2018-08-14 Alnylam Pharmaceuticals, Inc. angiopoietin type 3 (angpt13) irna compositions and methods of use thereof
WO2016176426A1 (en) 2015-04-28 2016-11-03 Duke University Thrombus imaging aptamers and methods of using same
CA2984636A1 (en) 2015-05-06 2016-11-10 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
CA2991894A1 (en) 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
LT3329002T (lt) 2015-07-31 2021-01-11 Alnylam Pharmaceuticals, Inc. Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai
EP3328439B1 (en) 2015-07-31 2020-04-08 Arcturus Therapeutics, Inc. Multiligand agent for drug delivery
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
US10889813B2 (en) 2015-09-02 2021-01-12 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
WO2017048620A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
HK1251624A1 (zh) 2015-09-24 2019-02-01 Ionis Pharmaceuticals, Inc. Kras表达的调节剂
WO2017055423A1 (en) 2015-10-02 2017-04-06 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugation process
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
LT4119569T (lt) 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje
JP6737885B2 (ja) 2015-11-18 2020-08-12 プロヴィヴィ インコーポレイテッド 昆虫フェロモンの生成のための微生物及び関連する化合物
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
IL308717A (en) 2015-12-07 2024-01-01 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
EP3387129A1 (en) 2015-12-10 2018-10-17 Alnylam Pharmaceuticals, Inc. STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
RS63051B1 (sr) 2015-12-22 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularnu isporuku agenasa
SMT202200310T1 (it) 2015-12-23 2022-09-14 Modernatx Inc Metodi di utilizzo dei polinucleotidi codificanti il ligando di ox40
US12678515B2 (en) 2015-12-30 2026-07-14 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
EP3405579A1 (en) 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
JOP20170057B1 (ar) 2016-03-07 2022-03-14 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة كمركبات علاجية
AU2017248189B2 (en) 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
EP3442590A2 (en) 2016-04-13 2019-02-20 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
WO2017194454A1 (en) 2016-05-09 2017-11-16 Astrazeneca Ab Lipid nanoparticles comprising lipophilic anti-inflammatory agents and methods of use thereof
PL3458083T3 (pl) 2016-05-18 2023-03-13 Modernatx, Inc. Polinukleotydy kodujące interleukinę-12 (il12) i ich zastosowania
CN115837014A (zh) 2016-05-18 2023-03-24 摩登纳特斯有限公司 编码松弛素的多核苷酸
JP7114485B2 (ja) 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
WO2017201332A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency
AU2017268396B2 (en) 2016-05-18 2023-05-18 Modernatx, Inc. Polynucleotides encoding citrin for the treatment of citrullinemia type 2
EP3464311A4 (en) 2016-06-06 2020-03-18 Provivi, Inc. HALF-BIOSYNTHETIC PRODUCTION OF FATTY ALCOHOLS AND FATTY ALDEHYDES
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
AU2017283479B2 (en) 2016-06-13 2023-08-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
ES2948971T3 (es) 2016-06-24 2023-09-22 Eisai R&D Man Co Ltd Lípido catiónico
US11191849B2 (en) 2016-06-30 2021-12-07 Arbutus Biopharma Corporation Compositions and methods for delivering messenger RNA
PL3484524T3 (pl) 2016-07-15 2023-03-20 Ionis Pharmaceuticals, Inc. Związki i sposoby modulacji smn2
US20190321387A1 (en) 2016-07-15 2019-10-24 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
GB201614120D0 (en) * 2016-08-18 2016-10-05 Univ Of Kwazulu-Natal pH-responsive lipids
CN109715205A (zh) 2016-08-19 2019-05-03 库瑞瓦格股份公司 用于癌症治疗的rna
WO2018041921A1 (en) 2016-08-31 2018-03-08 Curevac Ag Mixing device for the production of a liquid nucleic acid composition
JOP20170056B1 (ar) 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
JP7217700B2 (ja) 2016-09-13 2023-02-03 アラーガン、インコーポレイテッド 安定化非タンパク質クロストリジウム毒素組成物
EP3512526A4 (en) 2016-09-16 2020-05-27 Duke University VON WILLEBRAND FACTOR (VWF) TARGETING AGENTS AND METHODS OF USE
WO2018064755A1 (en) 2016-10-03 2018-04-12 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
CA3042015A1 (en) 2016-10-26 2018-05-03 Modernatx, Inc. Messenger ribonucleic acids for enhancing immune responses and methods of use thereof
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
WO2018104538A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rna for treatment or prophylaxis of a liver disease
WO2018104540A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rnas for wound healing
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018115507A2 (en) 2016-12-23 2018-06-28 Curevac Ag Henipavirus vaccine
WO2018115525A1 (en) 2016-12-23 2018-06-28 Curevac Ag Lassa virus vaccine
WO2018115527A2 (en) 2016-12-23 2018-06-28 Curevac Ag Mers coronavirus vaccine
MX2019009070A (es) 2017-02-01 2019-10-30 Modernatx Inc Composiciones de arnm terapeuticas inmunomoduladoras que codifican peptidos de mutacion de oncogenes de activacion.
WO2018146506A1 (en) * 2017-02-10 2018-08-16 Universitat Politècnica De València Therapeutic derivatives
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
JP7332478B2 (ja) 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
RS63953B1 (sr) 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
CA3050614A1 (en) 2017-03-17 2018-09-20 Curevac Ag Rna vaccine and immune checkpoint inhibitors for combined anticancer therapy
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
MA48047A (fr) 2017-04-05 2020-02-12 Modernatx Inc Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
WO2018195165A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
US11820728B2 (en) 2017-04-28 2023-11-21 Acuitas Therapeutics, Inc. Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
EP3625363A1 (en) 2017-05-17 2020-03-25 CureVac Real Estate GmbH Method for determining at least one quality parameter of an rna sample
CN110914442B (zh) 2017-05-17 2025-02-18 普罗维维股份有限公司 用于产生昆虫信息素及相关化合物的微生物
AU2018270111B2 (en) 2017-05-18 2022-07-14 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (IL12) polypeptides and uses thereof
EP4253544A3 (en) 2017-05-18 2023-12-20 ModernaTX, Inc. Modified messenger rna comprising functional rna elements
EP3630200A4 (en) 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
US11015204B2 (en) 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
KR102636537B1 (ko) 2017-05-31 2024-02-15 울트라제닉스 파마수티컬 인코포레이티드 글리코겐 축적 질환 유형 iii에 대한 치료제
JP2020522534A (ja) * 2017-06-06 2020-07-30 ウェイン ステート ユニバーシティＷａｙｎｅ Ｓｔａｔｅ Ｕｎｉｖｅｒｓｉｔｙ カルニチン由来材料に関する方法および組成物
CN109030429B (zh) * 2017-06-09 2022-06-21 南京大学 一种可对活细胞内microRNA21进行原位成像的纳米放大自参比探针
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3638292A1 (en) 2017-06-14 2020-04-22 ModernaTX, Inc. Polynucleotides encoding coagulation factor viii
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
US10780183B2 (en) 2017-06-19 2020-09-22 Translate Bio, Inc. Messenger RNA therapy for the treatment of Friedreich's ataxia
JP7722629B2 (ja) 2017-07-04 2025-08-13 キュアバック エスイー 新規核酸分子
AU2018301829B2 (en) 2017-07-13 2024-08-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression
CA3069868A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
JP7355731B2 (ja) 2017-08-16 2023-10-03 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
JP7461872B2 (ja) 2017-08-17 2024-04-04 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
WO2019036670A2 (en) 2017-08-18 2019-02-21 Modernatx, Inc. EFFECTIVE MRNA VACCINES
US11602557B2 (en) 2017-08-22 2023-03-14 Cure Vac SE Bunyavirales vaccine
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
EP3684931A1 (en) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
BR112020004351A2 (pt) 2017-10-19 2020-09-08 Curevac Ag moléculas de ácido nucleico artificial
CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
SG11202003247RA (en) 2017-11-08 2020-05-28 Curevac Ag Rna sequence adaptation
WO2019094702A1 (en) 2017-11-10 2019-05-16 Cocoon Biotech Inc. Ocular applications of silk-based products
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
JP7424976B2 (ja) 2017-11-22 2024-01-30 モダーナティエックス・インコーポレイテッド プロピオン酸血症の治療用のプロピオニルＣｏＡカルボキシラーゼアルファ及びベータサブユニットをコードするポリヌクレオチド
EP3714252B1 (en) 2017-11-22 2023-05-31 Beckman Coulter, Inc. Diluent preparation module und method for supplying such diluent
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11931406B2 (en) 2017-12-13 2024-03-19 CureVac SE Flavivirus vaccine
WO2019126097A1 (en) 2017-12-18 2019-06-27 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
MA51306A (fr) 2017-12-20 2020-10-28 Translate Bio Inc Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase
WO2019131580A1 (ja) 2017-12-27 2019-07-04 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
US11802146B2 (en) 2018-01-05 2023-10-31 Modernatx, Inc. Polynucleotides encoding anti-chikungunya virus antibodies
CA3088522A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
US20190314291A1 (en) 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
EP3773702A2 (en) 2018-04-05 2021-02-17 CureVac AG Novel yellow fever nucleic acid molecules for vaccination
KR20200141080A (ko) * 2018-04-09 2020-12-17 오르제네시스 인코포레이티드 바이옥솜 입자, 레독솜, 방법 및 조성물
US20210163928A1 (en) 2018-04-11 2021-06-03 Modernatx, Inc. Messenger rna comprising functional rna elements
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
CA3091558A1 (en) 2018-04-17 2019-10-24 Curevac Ag Novel rsv rna molecules and compositions for vaccination
IL277970B2 (en) 2018-05-09 2025-03-01 Ionis Pharmaceuticals Inc Oligomeric compounds comprising modified oligonucleotides and conjugated groups for use in the treatment of end-stage renal disease, chronic kidney disease and coronary artery disease
CA3100050A1 (en) 2018-05-11 2019-11-14 Lupagen, Inc. Systems and methods for closed loop, real-time modifications of patient cells
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
MA52709A (fr) 2018-05-23 2021-03-31 Modernatx Inc Administration d'adn
US12076439B2 (en) 2018-05-24 2024-09-03 Translate Bio, Inc. Thioester cationic lipids
CN118436618A (zh) 2018-05-30 2024-08-06 川斯勒佰尔公司 信使rna疫苗及其用途
EP3802507A1 (en) 2018-05-30 2021-04-14 Translate Bio, Inc. Vitamin cationic lipids
MA52762A (fr) 2018-05-30 2021-04-14 Translate Bio Inc Lipides cationiques comprenant une fraction stéroïdienne
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
ES2964547T3 (es) 2018-06-08 2024-04-08 Fujifilm Corp Compuesto, sal del mismo y partículas lipídicas
US20210260178A1 (en) 2018-06-27 2021-08-26 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
CN112543629A (zh) 2018-06-28 2021-03-23 阿斯利康(瑞典)有限公司 外泌体胞外囊泡及其使用方法
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
WO2020023533A1 (en) 2018-07-23 2020-01-30 Translate Bio, Inc. Dry power formulations for messenger rna
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP3833397A4 (en) 2018-08-08 2023-06-14 Arcturus Therapeutics, Inc. COMPOSITIONS AND AGENTS AGAINST NON-ALCOHOLIC STEATOHEPATITIS
WO2020033791A1 (en) 2018-08-09 2020-02-13 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
WO2020037102A1 (en) 2018-08-15 2020-02-20 University Of Florida Research Foundation, Inc. Methods of sensitizing tumors to treatment with immune checkpoint inhibitors
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
EP3843709A1 (en) 2018-08-29 2021-07-07 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
MA53545A (fr) 2018-09-02 2021-07-14 Modernatx Inc Polynucléotides codant pour l'acyl-coa déshydrogénase à très longue chaîne pour le traitement de l'insuffisance en acyl-coa déshydrogénase à très longue chaîne
US20220054610A1 (en) 2018-09-12 2022-02-24 University Of Florida Research Foundation, Inc. Slow-cycling cell-rna based nanoparticle vaccine to treat cancer
JP2022500436A (ja) 2018-09-13 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
US12644102B2 (en) 2018-09-13 2026-06-02 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex E1-α, E1-β, and E2 subunits for the treatment of maple syrup urine disease
MA53615A (fr) 2018-09-14 2021-07-21 Modernatx Inc Polynucléotides codant pour le polypeptide a1, de la famille de l'uridine diphosphate glycosyltransférase 1, pour le traitement du syndrome de crigler-najjar
JP7556848B2 (ja) 2018-09-14 2024-09-26 モデルナティエックス インコーポレイテッド ｍＲＮＡ治療薬を使用したがんを治療するための方法及び組成物
EP3849617A1 (en) 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
JP2022500003A (ja) 2018-09-18 2022-01-04 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物およびその使用方法
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
US12151029B2 (en) 2018-09-19 2024-11-26 Modernatx, Inc. PEG lipids and uses thereof
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
WO2020069169A1 (en) 2018-09-27 2020-04-02 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
ES2951836T3 (es) * 2018-10-01 2023-10-25 Alnylam Pharmaceuticals Inc Lípidos biodegradables para el suministro de agentes activos
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
CN112888451A (zh) 2018-10-19 2021-06-01 川斯勒佰尔公司 信使rna的无泵包封
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
US20220001026A1 (en) 2018-11-08 2022-01-06 Modernatx, Inc. Use of mrna encoding ox40l to treat cancer in human patients
CA3117866A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
US20220016265A1 (en) 2018-11-09 2022-01-20 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
WO2020097379A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Peg lipidoid compounds
KR102860605B1 (ko) 2018-11-09 2025-09-15 트랜슬레이트 바이오 인코포레이티드 에스테르, 티오에스테르, 이황화물, 및 무수물 모이어티가 삽입된 2,5-디옥소피페라진
EP3880174A2 (en) 2018-11-12 2021-09-22 Translate Bio, Inc. Methods for inducing immune tolerance
US12257305B2 (en) 2018-11-21 2025-03-25 Translate Bio, Inc. Cationic lipid compounds
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
CA3121191A1 (en) 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
WO2020128816A2 (en) 2018-12-20 2020-06-25 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
SG11202106378VA (en) 2018-12-21 2021-07-29 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
AU2019410737A1 (en) 2018-12-21 2021-06-10 CureVac SE RNA for malaria vaccines
WO2020146344A1 (en) 2019-01-07 2020-07-16 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
PL3908568T3 (pl) 2019-01-11 2024-12-02 Acuitas Therapeutics, Inc. Lipidy do dostarczania środków czynnych w nanocząstkach lipidowych
CN113939282A (zh) 2019-01-31 2022-01-14 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
KR20210133218A (ko) 2019-01-31 2021-11-05 모더나티엑스, 인크. 볼텍스 믹서 및 연계된 방법, 시스템 및 이의 장치
EP3920950A1 (en) 2019-02-08 2021-12-15 CureVac AG Coding rna administered into the suprachoroidal space in the treatment of ophtalmic diseases
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
WO2020205473A1 (en) 2019-03-29 2020-10-08 Decerna Pharmaceuticals, Inc. Compositions and methods for the treatment of kras associated diseases or disorders
EP3947646A1 (en) 2019-04-05 2022-02-09 Precision BioSciences, Inc. Methods of preparing populations of genetically-modified immune cells
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
WO2020214946A1 (en) 2019-04-18 2020-10-22 Translate Bio, Inc. Cystine cationic lipids
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
EP3959195B1 (en) 2019-04-22 2023-11-08 Translate Bio, Inc. Thioester cationic lipids
CN111849968A (zh) 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
EP3962521A1 (en) 2019-05-02 2022-03-09 University of Florida Research Foundation, Inc. Compositions for treatment of diffuse intrinsic pontine glioma
WO2020227085A1 (en) 2019-05-03 2020-11-12 Translate Bio, Inc. Di-thioester cationic lipids
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
MA55896A (fr) 2019-05-07 2022-03-16 Modernatx Inc Polynucléotides servant à perturber l'activité de cellule immunitaire et procédés pour les utiliser
EP3965830A1 (en) 2019-05-07 2022-03-16 ModernaTX, Inc. Differentially expressed immune cell micrornas for regulation of protein expression
WO2020227642A1 (en) 2019-05-08 2020-11-12 Modernatx, Inc. Compositions for skin and wounds and methods of use thereof
WO2020227615A1 (en) 2019-05-08 2020-11-12 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
JP7686574B2 (ja) 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状ｒｎａ組成物及び方法
EP3976593A1 (en) 2019-05-31 2022-04-06 Translate Bio, Inc. Macrocyclic lipids
CA3142608A1 (en) 2019-06-04 2020-12-10 Cocoon Biotech Inc. Silk-based products, formulations, and methods of use
CA3143865A1 (en) * 2019-06-07 2020-12-10 Fujifilm Corporation Lipid composition
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
JP7416096B2 (ja) 2019-06-20 2024-01-17 プレシジョン ナノシステムズ ユーエルシー 核酸送達のためのイオン化可能な脂質
EP3986858A1 (en) 2019-06-21 2022-04-27 Translate Bio, Inc. Tricine and citric acid lipids
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
EP3987027A1 (en) 2019-06-24 2022-04-27 ModernaTX, Inc. Endonuclease-resistant messenger rna and uses thereof
MA56517A (fr) 2019-06-24 2022-04-27 Modernatx Inc Arn messager comprenant des éléments d'arn fonctionnels et leurs utilisations
KR102928289B1 (ko) 2019-06-29 2026-02-20 글로벌 라이프 사이언시즈 솔루션즈 캐나다 유엘씨 핵산 전달을 위한 이온화 가능한 지질
CA3146369A1 (en) 2019-07-08 2021-01-14 Translate Bio, Inc. Improved mrna-loaded lipid nanoparticles and processes of making the same
ES2982157T3 (es) 2019-07-18 2024-10-14 Institut Nat De La Sante Et De Larecherche Medicale Inserm Mutante de RAC2 para su uso en la ablación de la hematopoyesis y en el tratamiento de neoplasias malignas hematopoyéticas
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
AU2020315962B2 (en) 2019-07-23 2026-02-12 Translate Bio, Inc. Stable compositions of mRNA-loaded lipid nanoparticles and processes of making
CA3149386A1 (en) 2019-07-31 2021-02-04 Modernatx, Inc. Compositions and methods for delivery of rna interference agents to immune cells
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
WO2021026358A1 (en) 2019-08-07 2021-02-11 Moderna TX, Inc. Compositions and methods for enhanced delivery of agents
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2021028439A1 (en) 2019-08-14 2021-02-18 Curevac Ag Rna combinations and compositions with decreased immunostimulatory properties
GB2600859B (en) 2019-08-14 2024-04-03 Acuitas Therapeutics Inc Improved lipid nanoparticles for delivery of nucleic acids
WO2021034639A1 (en) 2019-08-16 2021-02-25 Regeneron Pharmaceuticals, Inc. High concentration anti-c5 formulations
CN114929205B (zh) 2019-09-06 2024-09-03 世代生物公司 包括末端封闭式dna和可切割脂质的脂质纳米颗粒组合物及其使用方法
EP4031524A1 (en) 2019-09-19 2022-07-27 ModernaTX, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
AU2020351220A1 (en) 2019-09-19 2022-04-07 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
CA3148158A1 (en) * 2019-09-20 2021-03-25 Longcheng Li Nucleic acid molecule for treating thrombocytopenia and use thereof
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
AU2020355000A1 (en) 2019-09-23 2022-03-17 Sepia Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
CN114728017B (zh) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
CA3158013A1 (en) 2019-10-15 2021-04-22 Modernatx, Inc. Mrnas encoding granulocyte-macrophage colony stimulating factor for treating parkinson's disease
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
EP4048316A1 (en) 2019-10-21 2022-08-31 Translate Bio, Inc. Compositions, methods and uses of messenger rna
CA3158320A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
CN111205994B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株具核梭杆菌动物亚种菌株及其应用
CN111004738B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株具核梭杆菌多形亚种分离株及其应用
CN110903998B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株肠道分离的具核梭杆菌文氏亚种菌株及其应用
MX2022004712A (es) 2019-10-25 2022-08-04 Regeneron Pharma Regimenes de dosificacion para tratar o prevenir enfermedades asociadas a c5.
WO2021087036A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
IL292865A (en) 2019-11-13 2022-07-01 Alnylam Pharmaceuticals Inc Methods and preparations for the treatment of angiotensinogen-related disorder - (agt)
EP4059505B1 (en) 2019-11-15 2024-02-21 FUJIFILM Corporation Lipid composition
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
WO2021117770A1 (ja) * 2019-12-10 2021-06-17 富士フイルム株式会社 医薬組成物及び処置剤
WO2021117769A1 (ja) * 2019-12-10 2021-06-17 富士フイルム株式会社 医薬組成物及び処置剤
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them
CN115176007A (zh) 2019-12-16 2022-10-11 阿尔尼拉姆医药品有限公司 含类PATATIN磷脂酶结构域3(PNPLA3)iRNA组合物及其使用方法
MX2022007756A (es) 2019-12-20 2022-09-27 Translate Bio Inc Suministro rectal de arn mensajero.
JP2023508881A (ja) 2019-12-20 2023-03-06 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善されたプロセス
CA3162019A1 (en) 2019-12-20 2021-06-24 Gemma NAVARRO Lipid nanoparticles for delivery of nucleic acids
CN110974954B (zh) * 2019-12-24 2021-03-16 珠海丽凡达生物技术有限公司 一种用于增强核酸疫苗免疫效果的脂质纳米颗粒及其制备方法
EP4087544A1 (en) 2020-01-10 2022-11-16 ModernaTX, Inc. Methods of making tolerogenic dendritic cells
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
TW202140043A (zh) 2020-01-15 2021-11-01 美商戴瑟納製藥股份有限公司 4’-o-亞甲基膦酸酯核酸及其類似物
KR20230002300A (ko) * 2020-01-21 2023-01-05 이더알엔에이 이뮤노테라피스 엔브이 지질 나노입자
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US20230285297A1 (en) 2020-01-31 2023-09-14 Modernatx, Inc. Methods of preparing lipid nanoparticles
DE202021004130U1 (de) 2020-02-04 2022-10-26 Curevac Ag Coronavirus-Vakzine
PH12022552080A1 (en) 2020-02-10 2024-02-12 Alnylam Pharmaceuticals Inc Compositions and methods for silencing vegf-a expression
CN115397989A (zh) 2020-02-18 2022-11-25 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
CN115427021A (zh) 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
CA3174708A1 (en) * 2020-03-04 2021-09-10 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
MX2022011009A (es) 2020-03-06 2022-10-07 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de ketohexocinasa (khk) y metodos de uso de las mismas.
JP2023517644A (ja) 2020-03-09 2023-04-26 アークトゥラス・セラピューティクス・インコーポレイテッド コロナウイルスワクチン組成物及び方法
WO2021183720A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral DNA vectors and their use for therapeutic expression of factor ix
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
AU2021247163A1 (en) 2020-04-01 2022-10-20 University Of Florida Research Foundation, Incorporated Multilamellar RNA nanoparticle vaccine against SARS-CoV-2
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. COMPOSITIONS OF ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) ARNI AGENTS AND METHODS OF USE THEREOF
IL297121A (en) 2020-04-06 2022-12-01 Alnylam Pharmaceuticals Inc Compositions and methods for silencing myoc expression
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
AU2021251754A1 (en) 2020-04-07 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing SCN9A expression
EP4132951A4 (en) 2020-04-09 2025-06-11 Verve Therapeutics, Inc. CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B
JP2023522957A (ja) 2020-04-21 2023-06-01 フラッグシップ パイオニアリング， インコーポレイテッド 二機能性分子およびその使用方法
JP7844347B2 (ja) 2020-04-27 2026-04-13 アルナイラム ファーマシューティカルズ， インコーポレイテッド アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法
MX2022013606A (es) 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
AU2021269042A1 (en) 2020-05-07 2023-02-02 Translate Bio, Inc. Optimized nucleotide sequences encoding SARS-CoV-2 antigens
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
AU2021270587A1 (en) 2020-05-14 2022-12-08 Modernatx, Inc. LNP compositions comprising an mRNA therapeutic and an effector molecule
ES3066134T3 (en) 2020-05-14 2026-05-11 Translate Bio Inc Peg lipidoid compounds
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
JP7873638B2 (ja) 2020-05-15 2026-06-12 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子製剤
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
JP7883957B2 (ja) 2020-05-18 2026-07-02 マックス バイオロジー カンパニー リミテッド 脂質ポリマー組成物及び使用方法
IL298363A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
EP4157319A1 (en) 2020-05-28 2023-04-05 Modernatx, Inc. Use of mrnas encoding ox40l, il-23 and il-36gamma for treating cancer
WO2021239880A1 (en) 2020-05-29 2021-12-02 Curevac Ag Nucleic acid based combination vaccines
EP4158032A2 (en) 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
US20230203510A1 (en) 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
AU2021285812A1 (en) 2020-06-01 2023-01-05 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
WO2021247535A1 (en) 2020-06-01 2021-12-09 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
WO2025056938A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
IL299196A (en) 2020-06-23 2023-02-01 Modernatx Inc LNP preparations that include mRNA treatments with extended lifespan
US20240307556A1 (en) 2020-07-02 2024-09-19 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
IL299571A (en) 2020-07-08 2023-02-01 Janssen Sciences Ireland Unlimited Co Rna replicon vaccines against hbv
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
KR20230051172A (ko) 2020-07-16 2023-04-17 아퀴타스 테라퓨틱스 인크. 지질 나노 입자에 사용하기 위한 양이온성 지질
KR20230040353A (ko) 2020-07-17 2023-03-22 그린라이트 바이오사이언시스, 아이엔씨. 유전 장애를 위한 핵산 치료제
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
EP4172194A1 (en) 2020-07-31 2023-05-03 CureVac SE Nucleic acid encoded antibody mixtures
JP2023537499A (ja) 2020-08-04 2023-09-01 ディセルナ ファーマシューティカルズ インコーポレイテッド オリゴヌクレオチドの全身送達
TW202220637A (zh) 2020-08-06 2022-06-01 美商現代公司 製備脂質奈米顆粒之方法
US12178921B2 (en) 2020-08-14 2024-12-31 Arcturus Therapeutics, Inc. Method of lyophilizing lipid nanoparticles
WO2022043551A2 (en) 2020-08-31 2022-03-03 Curevac Ag Multivalent nucleic acid based coronavirus vaccines
IL300947A (en) 2020-09-03 2023-04-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc As a mass of large RNA lipid nanoparticles
JP7518909B2 (ja) 2020-09-14 2024-07-18 富士フイルム株式会社 脂質組成物
WO2022066916A1 (en) 2020-09-23 2022-03-31 Translate Bio, Inc. Piperazine-based cationic lipids
JP7844452B2 (ja) 2020-09-23 2026-04-13 トランスレイト バイオ， インコーポレイテッド Ｔｅｓベースのカチオン性脂質
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
AU2021350020A1 (en) * 2020-09-25 2023-04-27 DNARx Systems and methods for expressing biomolecules in a subject
JP2023543925A (ja) 2020-10-05 2023-10-18 マックス バイオロジー カンパニー リミテッド カンナビノイド含有組成物及び疾患の処置及び予防のための使用
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
CA3194863A1 (en) 2020-10-06 2022-04-14 Shrirang KARVE Improved process and formulation of lipid nanoparticles
US20230365995A1 (en) 2020-10-07 2023-11-16 Precision Biosciences, Inc. Lipid nanoparticle compositions
US12678406B2 (en) 2020-10-12 2026-07-14 Translate Bio Inc. Process of preparing ice-based lipid nanoparticles
CA3198411A1 (en) 2020-10-12 2022-04-21 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
US12576030B2 (en) 2020-11-09 2026-03-17 Translate Bio, Inc. Compositions for delivery of codon-optimized mRNA
CA3200595A1 (en) 2020-11-13 2022-05-19 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
CA3199784A1 (en) 2020-11-13 2022-05-19 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
US20240041785A1 (en) * 2020-11-16 2024-02-08 BioNTech SE Compositions and methods for stabilization of lipid nanoparticle mrna vaccines
KR20230108728A (ko) 2020-11-18 2023-07-18 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현을 조절하기 위한 화합물 및 방법
JP7872268B2 (ja) 2020-11-25 2026-06-09 トランスレイト バイオ， インコーポレイテッド 安定な液状脂質ナノ粒子製剤
WO2022112855A1 (en) * 2020-11-27 2022-06-02 Guangzhou Ribobio Co., Ltd Lipid compound and the composition thereof
WO2022110099A1 (en) 2020-11-30 2022-06-02 Guangzhou Argorna Biopharmaceuticals Co., Ltd. Coronavirus vaccines and uses thereof
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
US20240299309A1 (en) 2020-12-22 2024-09-12 CureVac SE Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
JP2024500879A (ja) * 2020-12-22 2024-01-10 コーネル ユニヴァーシティー 双性イオン性脂質ナノ粒子組成物およびその使用方法
MX2023007574A (es) 2020-12-22 2023-09-29 CureVac SE "vacuna de arn contra variantes de sars-cov-2.
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
AU2021412833A1 (en) 2020-12-28 2023-07-06 Arcturus Therapeutics, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
EP4277929A1 (en) 2021-01-14 2023-11-22 Translate Bio, Inc. Methods and compositions for delivering mrna coded antibodies
CA3170747A1 (en) 2021-01-27 2022-08-04 Moritz THRAN Method of reducing the immunostimulatory properties of in vitro transcribed rna
WO2022168884A1 (ja) 2021-02-04 2022-08-11 塩野義製薬株式会社 カチオン性脂質
US20240123076A1 (en) 2021-02-08 2024-04-18 The Board Of Regents Of The University Of Texas System Unsaturated dendrimers compositions, related formulations, and methods of use thereof
WO2022174079A1 (en) 2021-02-12 2022-08-18 Modernatx, Inc. Lnp compositions comprising payloads for in vivo therapy
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
CA3211059A1 (en) 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
MX2023009757A (es) 2021-02-26 2023-09-04 Ethris Gmbh Formulaciones para la formacion de aerosoles y aerosoles para la administracion de acido nucleico.
BR112023017737A2 (pt) 2021-03-04 2023-10-03 Alnylam Pharmaceuticals Inc Composições de irna de angiopoietina-semelhantes 3 (angptl3) e métodos de uso das mesmas
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
MX2023011227A (es) 2021-03-22 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion dirigida a celulas.
MX2023011243A (es) 2021-03-23 2023-12-14 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de los mismos.
US20240189449A1 (en) 2021-03-24 2024-06-13 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
US20240207374A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
US20250268994A1 (en) 2021-03-25 2025-08-28 Translate Bio, Inc. Optimized Nucleotide Sequences Encoding the Extracellular Domain of Human ACE2 Protein or a Portion Thereof
EP4313152A1 (en) 2021-03-26 2024-02-07 GlaxoSmithKline Biologicals S.A. Immunogenic compositions
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
EP4312988A2 (en) 2021-03-31 2024-02-07 CureVac SE Syringes containing pharmaceutical compositions comprising rna
EP4313109A1 (en) 2021-03-31 2024-02-07 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
JP2024512780A (ja) 2021-04-01 2024-03-19 モデルナティエックス インコーポレイテッド 多価ｒｎａ組成物中のｒｎａ種の識別及び比率決定のための方法
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
TW202309002A (zh) 2021-04-15 2023-03-01 美商轉譯生技公司 基於「古德」緩衝液的陽離子脂質
CA3215606A1 (en) 2021-04-19 2022-10-27 Shrirang KARVE Improved compositions for delivery of mrna
JP2024517427A (ja) 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法
EP4326227A1 (en) 2021-04-23 2024-02-28 ModernaTX, Inc. Stabilized formulations
WO2022226318A1 (en) 2021-04-23 2022-10-27 Modernatx, Inc. Isoquinoline-stabilized lipid nanoparticle formulations
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
JP2024515788A (ja) 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスｄｎａベクター及びその使用
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
US20240226028A1 (en) 2021-04-29 2024-07-11 Modernatx, Inc. Lyophilization methods for preparing lipid formulated therapeutics
CA3171589A1 (en) 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
US20240238418A1 (en) 2021-05-07 2024-07-18 University Of Florida Research Foundation, Inc. Car t cell therapy method
WO2022240806A1 (en) 2021-05-11 2022-11-17 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ， インコーポレイテッド ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
WO2022246020A1 (en) 2021-05-19 2022-11-24 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
CN113546180A (zh) * 2021-05-25 2021-10-26 重庆医科大学 一种具有心肌靶向性的基因递送载体及其制备方法
MX2023014008A (es) 2021-05-27 2023-12-11 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion de anhidrasa carbonica 2.
CN117580820A (zh) * 2021-05-28 2024-02-20 内诺维什疗法股份有限公司 Mc3型脂质及其在脂质纳米颗粒的制备中的用途
AU2022282812A1 (en) * 2021-05-28 2023-11-09 Nanovation Therapeutics Inc. Mc3-type lipids and use thereof in the preparation of lipid nanoparticles
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
WO2022256395A1 (en) 2021-06-02 2022-12-08 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
MX2023014168A (es) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este.
JP2024523000A (ja) 2021-06-08 2024-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド シュタルガルト病及び／又は網膜結合タンパク質４（ｒｂｐ４）関連障害を治療又は予防するための組成物及び方法
WO2022260772A1 (en) * 2021-06-09 2022-12-15 Carnegie Mellon University Lipid nanoparticle formulations for gastrointestinal delivery
US20240384277A1 (en) 2021-06-15 2024-11-21 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
US20240376445A1 (en) 2021-06-22 2024-11-14 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
CN117615753A (zh) * 2021-06-24 2024-02-27 泰纳治疗公司 脂质纳米颗粒及其制备方法
KR102516680B1 (ko) * 2021-06-24 2023-04-03 주식회사 테르나테라퓨틱스 지질 나노 입자 및 그 제조방법
EP4360624A4 (en) * 2021-06-24 2025-06-25 Therna Therapeutics LIPID NANOPARTICLES AND PROCESS FOR THEIR PREPARATION
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for the treatment of angiotensinogen-related disorder (AGT-)
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
EP4362920A1 (en) 2021-07-01 2024-05-08 Translate Bio, Inc. Compositions for delivery of mrna
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
US20240316165A1 (en) 2021-07-12 2024-09-26 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
TW202325312A (zh) 2021-07-23 2023-07-01 美商艾拉倫製藥股份有限公司 β－鏈蛋白（ＣＴＮＮＢ１）ｉＲＮＡ組成物及其使用方法
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
WO2023008415A1 (ja) 2021-07-27 2023-02-02 ＳＴＡＮＤ Ｔｈｅｒａｐｅｕｔｉｃｓ株式会社 ペプチドタグおよび当該ペプチドタグをコードする核酸
WO2023009499A1 (en) 2021-07-27 2023-02-02 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease type 1a (gsd1a)
JP2024529437A (ja) 2021-07-29 2024-08-06 アルナイラム ファーマシューティカルズ， インコーポレイテッド ３－ヒドロキシ－３－メチルグリタル－ＣＯＡレダクターゼ（ＨＭＧＣＲ）ｉＲＮＡ組成物およびその使用方法
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377331A2 (en) 2021-07-30 2024-06-05 CureVac SE Mrnas for treatment or prophylaxis of liver diseases
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
EP4381059A1 (en) 2021-08-02 2024-06-12 ModernaTX, Inc. Extraction-less reverse phase (rp) chromatography of mrna encapsulated in lipid nanoparticles for mrna purity assessment
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
EP4380576A4 (en) 2021-08-03 2026-04-29 Verve Therapeutics Inc RNA TARGETED COMPOSITIONS AND ADMINISTRATION METHODS
CA3228255A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
WO2023015261A1 (en) 2021-08-04 2023-02-09 Modernatx, Inc. Mrnas encoding chimeric metabolic reprogramming polypeptides and uses thereof
WO2023014974A1 (en) 2021-08-06 2023-02-09 University Of Iowa Research Foundation Double stranded mrna vaccines
US20250281418A1 (en) 2021-08-11 2025-09-11 Modernatx, Inc. Lipid nanoparticle formulations and methods of synthesis thereof
WO2023019181A1 (en) 2021-08-11 2023-02-16 Modernatx, Inc. Sars-cov-2 lipid nanoparticle vaccine formulations
CN118076737A (zh) 2021-08-13 2024-05-24 阿尔尼拉姆医药品有限公司 因子XII（F12）iRNA组合物及其使用方法
TW202325311A (zh) 2021-08-31 2023-07-01 美商艾拉倫製藥公司 誘導細胞死亡之DFFA樣效應子B (CIDEB) iRNA組合物及其使用方法
WO2023031855A1 (en) 2021-09-03 2023-03-09 Glaxosmithkline Biologicals Sa Substitution of nucleotide bases in self-amplifying messenger ribonucleic acids
EP4763287A2 (en) 2021-09-03 2026-06-24 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
IL309502A (en) 2021-09-03 2024-02-01 CureVac SE Lipid nanoparticles for nucleic acid delivery involving phosphatidylserine
CN115772089B (zh) * 2021-09-07 2025-06-10 广州谷森制药有限公司 阳离子脂质化合物
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法
AR127073A1 (es) 2021-09-17 2023-12-13 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
JP2024535888A (ja) 2021-09-20 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
US20250017867A1 (en) 2021-10-01 2025-01-16 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
AU2022357561A1 (en) 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
WO2023056401A1 (en) 2021-10-01 2023-04-06 Modernatx, Inc. Rna formulations for high volume distribution, and methods of using the same for treating a disease or condition caused by or associated with human cytomegalovirus
US20240401045A1 (en) 2021-10-01 2024-12-05 Adarx Pharmaceuticals, Inc. Angiotensinogen-modulating compositions and methods of use thereof
US20250281583A1 (en) 2021-10-13 2025-09-11 Modernatx, Inc. Composition of mrna-encoded il15 fusion proteins and methods of use thereof
EP4416284A1 (en) 2021-10-15 2024-08-21 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
EP4419706A1 (en) 2021-10-18 2024-08-28 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2023069895A1 (en) 2021-10-18 2023-04-27 Modernatx, Inc. Markerless dna production
WO2023070132A1 (en) * 2021-10-22 2023-04-27 Ohio State Innovation Foundation Immunotherapies for the treatment of cancer
AU2022371688A1 (en) * 2021-10-22 2024-06-06 Ohio State Innovation Foundation Compositions and methods of the delivery of active agents including nucleic acids
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
KR20240090876A (ko) 2021-10-26 2024-06-21 아스트라제네카 아베 핵산 세그먼트의 전달을 위한 신규한 지질
AR127477A1 (es) 2021-10-29 2024-01-31 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA EL FACTOR B DEL COMPLEMENTO (CFB) Y MÉTODOS DE USO DE LAS MISMAS
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
EP4426340A1 (en) 2021-11-01 2024-09-11 ModernaTX, Inc. Polynucleotides encoding integrin beta-6 and methods of use thereof
WO2023081526A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
JP2024545572A (ja) 2021-11-10 2024-12-10 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全の治療のための組成物及び方法
KR102772764B1 (ko) 2021-11-10 2025-02-27 주식회사 에스엠엘바이오팜 트레할로즈 유도체 및 신규 구조 유지 화합물을 포함하는 핵산 의약품 전달용 지질나노입자의 약학 조성물
JP2024546431A (ja) 2021-11-11 2024-12-24 アークトゥラス・セラピューティクス・インコーポレイテッド Ｒｎａ送達のためのイオン化可能なカチオン性脂質
CA3237573A1 (en) 2021-11-18 2023-05-25 Astrazeneca Ab Novel lipids for delivery of nucleic acid segments
AU2022391744A1 (en) 2021-11-18 2024-06-27 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
WO2023092151A1 (en) * 2021-11-22 2023-05-25 Ohio State Innovation Foundation Compositions and methods for the treatment of neurodegenerative disorders
MX2024006407A (es) 2021-11-24 2024-08-19 Flagship Pioneering Innovations Vi Llc Composiciones de inmunogenos del virus varicela-zoster y sus usos.
WO2023096990A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovation Vi, Llc Coronavirus immunogen compositions and their uses
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
US20250360194A1 (en) 2021-12-08 2025-11-27 Modernatx, Inc. Herpes simplex virus mrna vaccines
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
JP2025500257A (ja) 2021-12-15 2025-01-09 モデルナティエックス インコーポレイテッド 脂質ナノ粒子の封入効率の決定
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
TW202333654A (zh) 2021-12-16 2023-09-01 美商現代公司 用於製備脂質奈米顆粒之方法
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
AU2022420620A1 (en) 2021-12-23 2024-07-04 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
US20250082777A1 (en) 2022-01-10 2025-03-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
CN114044741B (zh) 2022-01-13 2022-04-15 北京悦康科创医药科技股份有限公司 一种阳离子脂质化合物、包含其的组合物及用途
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023135298A1 (en) 2022-01-17 2023-07-20 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of inducing cell death of a population of solid tumor cells
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
JP2025508332A (ja) 2022-01-27 2025-03-26 ビオンテック エスエー 単純ヘルペスウイルス抗原の送達のための薬学的組成物及び関連方法
EP4469091A1 (en) 2022-01-28 2024-12-04 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023152365A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of the 15-lipoxygenase for the treatment of lymphedema
EP4479085A1 (en) 2022-02-18 2024-12-25 ModernaTX, Inc. Mrnas encoding checkpoint cancer vaccines and uses thereof
US20250327041A1 (en) 2022-02-24 2025-10-23 Io Biotech Aps Nucleotide delivery of cancer therapy
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
EP4493212A1 (en) 2022-03-14 2025-01-22 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
EP4494644A4 (en) 2022-03-15 2025-06-11 FUJIFILM Corporation LIPID COMPOSITION
EP4493548A1 (en) 2022-03-16 2025-01-22 Translate Bio, Inc. Asymmetric piperazine-based cationic lipids
WO2023177904A1 (en) 2022-03-18 2023-09-21 Modernatx, Inc. Sterile filtration of lipid nanoparticles and filtration analysis thereof for biological applications
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
US20250214046A1 (en) 2022-04-01 2025-07-03 Modernatx, Inc. Cross mixers for lipid nanoparticle production, and methods of operating the same
WO2023196399A1 (en) 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023196988A1 (en) 2022-04-07 2023-10-12 Modernatx, Inc. Methods of use of mrnas encoding il-12
JP2025511756A (ja) 2022-04-08 2025-04-16 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー ワクチン及び関連する方法
WO2023200893A1 (en) * 2022-04-13 2023-10-19 Oregon State University Small molecule enhancers of antisense oligo activity
CN121318878A (zh) 2022-04-13 2026-01-13 赛诺菲 基于“古德”缓冲液的阳离子脂质
WO2023201296A1 (en) 2022-04-15 2023-10-19 Modernatx, Inc. Ribosomal engagement potency assay
WO2023212696A1 (en) 2022-04-29 2023-11-02 Modernatx, Inc. Lyophilized human cytomegalovirus vaccines
WO2023215498A2 (en) 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
WO2023220083A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
WO2023220734A2 (en) * 2022-05-13 2023-11-16 The Trustees Of The University Of Pennsylvania Bisphosphonate lipids, lipid nanoparticle compositions comprising the same, and methods of use thereof for targeted delivery
JP2025516638A (ja) 2022-05-13 2025-05-30 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー 二本鎖ｄｎａ組成物及び関連する方法
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
CA3248209A1 (en) 2022-05-25 2023-11-30 CureVac SE Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH)
WO2023230601A1 (en) 2022-05-27 2023-11-30 Beam Therapeutics Inc. Identification of nanoparticles for preferential tissue or cell targeting
WO2023239756A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
KR20250022763A (ko) 2022-06-10 2025-02-17 캠프4 테라퓨틱스 코포레이션 조절 rna를 표적으로 하는 안티센스 올리고뉴클레오타이드를 사용하여 프로그래뉼린 발현을 조절하는 방법
CN117263818B (zh) * 2022-06-14 2026-05-08 杭州高田生物医药有限公司 阳离子脂质化合物及其制备方法和应用
WO2023242817A2 (en) 2022-06-18 2023-12-21 Glaxosmithkline Biologicals Sa Recombinant rna molecules comprising untranslated regions or segments encoding spike protein from the omicron strain of severe acute respiratory coronavirus-2
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
US20250375524A1 (en) 2022-06-30 2025-12-11 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
WO2024015890A1 (en) 2022-07-13 2024-01-18 Modernatx, Inc. Norovirus mrna vaccines
AU2023316015A1 (en) 2022-07-25 2025-02-13 Centre Hospitalier Universitaire De Toulouse Use of apelin for the treatment of lymphedema
EP4562163A1 (en) 2022-07-26 2025-06-04 ModernaTX, Inc. Engineered polynucleotides for temporal control of expression
WO2024026487A1 (en) 2022-07-29 2024-02-01 Modernatx, Inc. Lipid nanoparticle compositions comprising phospholipid derivatives and related uses
US20250375391A1 (en) 2022-07-29 2025-12-11 Modernatx, Inc. Lipid nanoparticle compositions comprising surface lipid derivatives and relates uses
WO2024026475A1 (en) 2022-07-29 2024-02-01 Modernatx, Inc. Compositions for delivery to hematopoietic stem and progenitor cells (hspcs) and related uses
WO2024030369A1 (en) 2022-08-01 2024-02-08 Modernatx, Inc. Extraction-less reverse phase (rp) chromatography for mrna purity assessment
US20240042021A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024040254A2 (en) 2022-08-19 2024-02-22 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
WO2024044147A1 (en) 2022-08-23 2024-02-29 Modernatx, Inc. Methods for purification of ionizable lipids
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
EP4577243A1 (en) 2022-08-26 2025-07-02 Ethris GmbH Stable lipid or lipidoid nanoparticle suspensions
WO2024050483A1 (en) 2022-08-31 2024-03-07 Modernatx, Inc. Variant strain-based coronavirus vaccines and uses thereof
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
CN120152962A (zh) 2022-09-06 2025-06-13 国立大学法人九州大学 脂质纳米粒子
CN118265692B (zh) 2022-09-09 2025-06-13 厦门赛诺邦格生物科技股份有限公司 一种碳核阳离子脂质
CN119907856A (zh) 2022-09-15 2025-04-29 阿尔尼拉姆医药品有限公司 第13型17β-羟基类固醇脱氢酶(HSD17B13)iRNA组合物及其使用方法
JP2025531268A (ja) 2022-09-19 2025-09-19 チューン セラピューティクス インコーポレイテッド Ｔ細胞機能を調節するための組成物、システム、および方法
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
US20260151470A1 (en) 2022-09-23 2026-06-04 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
IL319414A (en) 2022-09-23 2025-05-01 BioNTech SE Compositions for delivering antigens at the liver level and related methods
CA3267272A1 (en) 2022-09-26 2024-04-04 Glaxosmithkline Biologicals Sa FLU VIRUS VACCINES
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
WO2024091918A2 (en) 2022-10-25 2024-05-02 Modernatx, Inc. Methods of lipid nanoparticle production in cross-mixers
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
WO2024097639A1 (en) 2022-10-31 2024-05-10 Modernatx, Inc. Hsa-binding antibodies and binding proteins and uses thereof
EP4612296A1 (en) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
US20260176618A1 (en) 2022-11-03 2026-06-25 Modernatx, Inc. Chemical stability of mrna
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102799A1 (en) 2022-11-08 2024-05-16 Flagship Pioneering Innovations Vi, Llc Compositions and methods for producing circular polyribonucleotides
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
EP4620948A4 (en) 2022-11-18 2026-06-03 Korea Research Inst Of Bioscience And Biotechnology LIPID COMPOUND AND COMPOSITION FOR THE ADMINISTRATION OF AN ACTIVE SUBSTANCE
EP4622630A1 (en) 2022-11-21 2025-10-01 Translate Bio, Inc. Compositions of dry powder formulations of messenger rna and methods of use thereof
US20260191905A1 (en) 2022-12-01 2026-07-09 Modernatx, Inc. Gpc3-specific antibodies, binding domains, and related proteins and uses thereof
US20260207525A1 (en) 2022-12-01 2026-07-23 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
JP2025541726A (ja) 2022-12-01 2025-12-23 ジェネレーション バイオ カンパニー 細胞標的化のためのステルス脂質ナノ粒子組成物
EP4627084A1 (en) 2022-12-01 2025-10-08 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
IL321026A (en) 2022-12-08 2025-07-01 Recode Therapeutics Inc Nanoparticle formulations and their uses
EP4630033A1 (en) 2022-12-09 2025-10-15 Institut National de la Santé et de la Recherche Médicale Novel human antiviral genes related to the eleos and lamassu prokaryotic systems
TW202440929A (zh) 2022-12-14 2024-10-16 加拿大商普羅維登斯治療控股公司 用於感染性疾病的組合物和方法
EP4385523A1 (en) 2022-12-14 2024-06-19 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid-based topical injection formulations
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於將治療劑遞送至骨之組成物和方法
EP4634390A2 (en) 2022-12-15 2025-10-22 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2023394992A1 (en) 2022-12-15 2025-07-31 Sanofi Pasteur Inc. Mrna encoding influenza virus-like particle
WO2024130158A1 (en) 2022-12-16 2024-06-20 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding extended serum half-life interleukin-22 for the treatment of metabolic disease
WO2024133160A1 (en) 2022-12-19 2024-06-27 Glaxosmithkline Biologicals Sa Hepatitis b compositions
TW202438514A (zh) 2022-12-20 2024-10-01 法商賽諾菲公司 鼻病毒mrna疫苗
CN119095585A (zh) * 2022-12-21 2024-12-06 苏州艾博生物科技有限公司 包含固醇修饰的磷脂的脂质纳米颗粒
US20260218178A1 (en) 2022-12-22 2026-07-30 Mina Therapeutics Limited Chemically Modified saRNA Compositions and Methods of Use
CN115975173B (zh) * 2022-12-27 2025-07-11 中国药科大学 一种酰胺键连接的聚乙二醇-磷脂成脂材料及其制备方法与应用
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
EP4642481A2 (en) 2022-12-29 2025-11-05 Popvax Private Limited Multitarget vaccines and therapeutics
JP2026503277A (ja) 2023-01-09 2026-01-28 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 心筋梗塞を経験した患者における有害な虚血後心臓リモデリングを処置するためのアンジオポエチン様４の組み換えフィブリノーゲン様ドメインの使用
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
EP4648754A1 (en) * 2023-01-09 2025-11-19 Northmirs, Inc. Microrna-based particle for the treatment of dysregulated immune response
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
EP4648793A1 (en) 2023-01-11 2025-11-19 ModernaTX, Inc. Personalized cancer vaccines
WO2024153636A1 (en) 2023-01-17 2024-07-25 Institut National de la Santé et de la Recherche Médicale Vasorin as a biomarker and biotarget in nephrology
JP2026504911A (ja) 2023-01-19 2026-02-10 アルトス ラブス，インコーポレーテッド 臓器移植における再生因子の使用
WO2024156835A1 (en) 2023-01-27 2024-08-02 Institut National de la Santé et de la Recherche Médicale Use of amphiregulin (areg) in methods of treating vascular hyperpermeability
US20260232791A1 (en) 2023-01-27 2026-08-13 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus glycoprotein c, glycoprotein d, and glycoprotein e antigens and related methods
JP2026503718A (ja) 2023-01-30 2026-01-29 モデルナティエックス インコーポレイテッド エプスタイン・バーウイルスｍＲＮＡワクチン
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
EP4658302A1 (en) 2023-02-02 2025-12-10 GlaxoSmithKline Biologicals S.A. Immunogenic composition
WO2024160936A1 (en) 2023-02-03 2024-08-08 Glaxosmithkline Biologicals Sa Rna formulation
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reverser molecules and methods of using them
US20240293318A1 (en) 2023-02-13 2024-09-05 Flagship Pioneering Innovations Vii, Llc Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
KR20250151437A (ko) 2023-02-17 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 변형된 우라실을 포함하는 dna 조성물
TW202446956A (zh) 2023-02-17 2024-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的胞嘧啶的ｄｎａ組成物
WO2024178305A1 (en) 2023-02-24 2024-08-29 Modernatx, Inc. Compositions of mrna-encoded il-15 fusion proteins and methods of use thereof for treating cancer
WO2024182301A2 (en) 2023-02-27 2024-09-06 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding galactose-1-phosphate uridylyltransferase (galt) for the treatment of galactosemia
GB202303019D0 (en) 2023-03-01 2023-04-12 Glaxosmithkline Biologicals Sa Method of lyophilisation
KR20250153298A (ko) 2023-03-08 2025-10-24 큐어백 에스이 핵산 전달을 위한 신규의 지질 나노입자 제형
EP4676526A2 (en) 2023-03-10 2026-01-14 ModernaTX, Inc. Nucleic acid influenza vaccines and respiratory virus combination vaccines
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
EP4680276A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
WO2024197033A1 (en) 2023-03-21 2024-09-26 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of heart failure
WO2024194484A1 (en) 2023-03-23 2024-09-26 Institut National de la Santé et de la Recherche Médicale Modulating the expression and/or activity of gas7 for modulating viral replication
WO2024206329A1 (en) 2023-03-27 2024-10-03 Modernatx, Inc. Nucleic acid molecules encoding bi-specific secreted engagers and uses thereof
WO2024206126A1 (en) 2023-03-27 2024-10-03 Modernatx, Inc. Cd16-binding antibodies and uses thereof
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024206835A1 (en) 2023-03-30 2024-10-03 Modernatx, Inc. Circular mrna and production thereof
WO2024211518A1 (en) 2023-04-07 2024-10-10 Astrazeneca Ab Fluidic mixer unit device for nanoparticle production
WO2024215721A1 (en) 2023-04-10 2024-10-17 Modernatx, Inc. Lyme disease vaccines
WO2024216128A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
MX2025012337A (es) 2023-04-17 2026-02-03 Sanofi Pasteur Inc Formulaciones de polvo seco reconstituibles y métodos de uso de las mismas
KR20260003750A (ko) * 2023-04-19 2026-01-07 프라임 메디슨, 아이엔씨. 지질 나노입자 (lnp) 전달 시스템 및 제제
EP4698652A2 (en) 2023-04-20 2026-02-25 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
JP2026518411A (ja) 2023-04-27 2026-06-08 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム インフルエンザウイルスワクチン
EP4701657A1 (en) 2023-04-27 2026-03-04 GlaxoSmithKline Biologicals S.A. Influenza virus vaccines
EP4704816A1 (en) 2023-05-03 2026-03-11 ModernaTX, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
CN121038807A (zh) 2023-05-03 2025-11-28 生物技术欧洲股份公司 优化的csp变体及相关方法
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
DE102023001946A1 (de) * 2023-05-12 2024-11-14 Friedrich-Schiller-Universität Jena, Körperschaft des öffentlichen Rechts Nanopartikel für den Transport von Wirkstoffen mit anionischen Gruppen, Verfahren zu deren Herstellung und deren Verwendung
EP4716741A2 (en) 2023-05-23 2026-04-01 Flagship Labs 114, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
WO2024249328A2 (en) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
WO2024254552A1 (en) 2023-06-08 2024-12-12 Modernatx, Inc. Stabilized flavivirus vaccines
WO2024259373A1 (en) 2023-06-14 2024-12-19 Modernatx, Inc. Compounds and compositions for delivery of therapeutic agents
CN121712891A (zh) 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 Lrrk2调节组合物及其使用方法
WO2024263826A1 (en) 2023-06-22 2024-12-26 Modernatx, Inc. Sars-cov-2 t cell vaccines
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
JP2026524354A (ja) 2023-06-28 2026-07-22 サノフィ ワクチンズ ユーエス インコーポレイテッド 核酸送達のためのジアンヒドロヘキシトール系イオン化可能な脂質
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CN121532514A (zh) 2023-07-07 2026-02-13 上海环码生物医药有限公司 用于季节性流感的环状rna疫苗及使用方法
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP4743577A1 (en) 2023-07-13 2026-05-20 Korro Bio, Inc. RNA EDITING OLIGONUCLEOTIDES AND THEIR USES
EP4745237A1 (en) 2023-07-14 2026-05-20 Yun Ho Bio Co., Ltd ARNI AGENT TO INHIBIT THE EXPRESSION AND USE OF KETOHEXOKINASE
WO2025019352A2 (en) 2023-07-14 2025-01-23 Modernatx, Inc. Mers-cov mrna vaccines
EP4746902A2 (en) 2023-07-21 2026-05-27 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
EP4497433A1 (en) 2023-07-24 2025-01-29 Ludwig-Maximilians-Universität München Nano-in-micro encapsulated sirna dry powder and pharmaceutical product containing said dry powder
EP4751736A1 (en) 2023-07-25 2026-06-03 The University of Osaka Composition for use in vaccine
GB202311382D0 (en) 2023-07-25 2023-09-06 Glaxosmithkline Biologicals Sa Lyophilised compostion
WO2025029700A1 (en) 2023-07-28 2025-02-06 Modernatx, Inc. Vlp enteroviral vaccines
CN121909284A (zh) 2023-07-31 2026-04-21 图恩疗法股份有限公司 用于调节il-2基因表达的组合物和方法
EP4754248A1 (en) 2023-07-31 2026-06-10 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025030154A1 (en) 2023-08-03 2025-02-06 The Trustees Of The University Of Pennsylvania Pharmaceutical compositions for delivery of herpes simplex virus glycoprotein b antigens and related methods
EP4754255A1 (en) 2023-08-04 2026-06-10 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025034612A1 (en) 2023-08-04 2025-02-13 Modernatx, Inc. Varicella-zoster virus mrna vaccine
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
CN121712488A (zh) 2023-08-14 2026-03-20 埃泽瑞斯公司 非全身性mRNA施用
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025042806A2 (en) 2023-08-21 2025-02-27 Modernatx, Inc. C-met binding antibodies, nucleic acids encoding same, and methods of use
CN120035670A (zh) 2023-08-23 2025-05-23 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
CN121729225A (zh) 2023-08-25 2026-03-24 埃泽瑞斯公司 具有增强药物应用的特定表面活性剂性质的稳定脂质和类脂质纳米颗粒制剂
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
EP4771042A1 (en) 2023-08-29 2026-07-08 Shanghai Circode Biomed Co., Ltd Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025054383A1 (en) 2023-09-06 2025-03-13 Modernatx, Inc. Chemical stability of mrna
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
CN121844046A (zh) 2023-09-11 2026-04-10 C·米库尔卡 用于递送肠促胰岛素剂的rna组合物
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025059290A1 (en) 2023-09-14 2025-03-20 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
IL327035A (en) 2023-09-18 2026-05-01 Flagship Pioneering Innovations Vii Llc Ionizable lipidoid compositions and their medical uses
EP4780812A1 (en) 2023-09-20 2026-07-29 Sanofi Good" buffer-based cationic lipids for nucleic acid delivery
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025072482A1 (en) 2023-09-27 2025-04-03 Modernatx, Inc. Immunoglobulin a protease polypeptides, polynucleotides, and uses thereof
EP4784753A2 (en) 2023-09-27 2026-08-05 Judo Bio Inc. Slc6a19-targeting modulatory nucleic acid agents
EP4784172A1 (en) 2023-09-27 2026-08-05 Judo Bio Inc. Polymyxins for delivery of agents to the kidney
AU2024348221A1 (en) 2023-09-27 2026-04-30 Judo Bio Inc. Aminoglycosides for delivery of agents to the kidney
WO2025067433A1 (zh) 2023-09-28 2025-04-03 云合智药（苏州）生物科技有限公司 靶向整合素的化合物及其在RNAi剂中的应用
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025083211A1 (en) 2023-10-20 2025-04-24 Institut National de la Santé et de la Recherche Médicale Use of factor h for the treatment of dementia
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
CN118995714B (zh) 2024-09-26 2025-12-12 杭州天龙药业有限公司 靶向抑制AGT基因表达的siRNA及其在治疗高血压中的应用
CN117210468B (zh) 2023-11-06 2024-02-20 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN122497525A (zh) 2023-11-07 2026-07-31 欧纳医疗公司 环状rna组合物
KR20260113064A (ko) 2023-11-14 2026-07-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 이온화 가능한 리피도이드 조성물 및 이의 치료적 용도
FR3155424A1 (fr) 2023-11-15 2025-05-23 BioNTech SE Compositions immunogènes contre le sars-cov-2
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
AU2024384477A1 (en) 2023-11-21 2026-05-21 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
US12553042B2 (en) 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
WO2025117969A1 (en) 2023-12-01 2025-06-05 Orna Therapeutics, Inc. Process for manufacturing lipid nanoparticles
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025137646A1 (en) 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
WO2025140551A1 (en) 2023-12-29 2025-07-03 Neuro3 Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING NaV1.7 (SCN9A) -ASSOCIATED DISORDERS
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025160127A1 (en) 2024-01-23 2025-07-31 Modernatx, Inc. Ionizable lipids and compositions thereof for delivery of therapeutic agents
WO2025160381A1 (en) 2024-01-26 2025-07-31 Modernatx, Inc. Methods of preparing lipid nanoparticles
WO2025166202A1 (en) 2024-01-31 2025-08-07 Modernatx, Inc. Lipid nanoparticle compositions comprising sialic acid derivatives and the uses thereof
AU2025217182A1 (en) 2024-02-02 2026-08-06 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2025184429A1 (en) 2024-02-28 2025-09-04 Modernatx, Inc. Bromodomain and extra-terminal domain (bet) epigenetic reader decoy
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025196065A1 (en) 2024-03-20 2025-09-25 Sanofi Novel homocysteine based lipids and their use for delivery of nucleic acids
WO2025202937A1 (en) 2024-03-26 2025-10-02 BioNTech SE Cancer vaccines
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025213135A1 (en) * 2024-04-04 2025-10-09 The Brigham And Women's Hospital, Inc. Lipid nanoparticles for the treatment of vascular diseases
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
WO2025215072A1 (en) 2024-04-10 2025-10-16 Institut National de la Santé et de la Recherche Médicale Class i lanthipeptides with anti-viral function
CN118388360B (zh) 2024-04-17 2025-04-04 北京悦康科创医药科技股份有限公司 含有苯环结构的长效脾靶向阳离子脂质化合物、包含其的组合物及用途
WO2025226656A1 (en) 2024-04-23 2025-10-30 Modernatx, Inc. Hepatitis b virus mrna vaccines
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease
WO2025229572A1 (en) 2024-05-01 2025-11-06 Glaxosmithkline Biologicals Sa Epstein-barr virus antigen-encoding messenger ribonucleic acid and antigen protein vaccines
CN118240844B (zh) 2024-05-27 2024-12-13 北京悦康科创医药科技股份有限公司 带状疱疹mRNA疫苗及其制备方法和应用
US12685787B2 (en) 2024-05-30 2026-07-21 Lipovectra Inc. Lipid nanoparticles with RNA for cosmetic treatment
WO2025248096A1 (en) 2024-05-31 2025-12-04 Institut National de la Santé et de la Recherche Médicale Antimicrobial peptides having liquid-liquid phase separation activities
WO2025250938A2 (en) 2024-05-31 2025-12-04 Modernatx, Inc. Respiratory syncytial virus and metapneumovirus vaccines
WO2025255199A1 (en) 2024-06-05 2025-12-11 Modernatx, Inc. Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
TW202615058A (zh) 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 失養強直性肌肉蛋白質激酶（ｄｍｐｋ）ｉｒｎａ組成物及其使用方法
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
AU2025205193A1 (en) 2024-07-08 2026-01-29 Hangzhou Tianlong Pharmaceutical Co., Ltd. Double-Stranded RNAi Agents For Targeting And Regulating HBV Gene Expression, And A Use Thereof
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026015882A1 (en) 2024-07-12 2026-01-15 BioNTech SE Hsv antigen fragments and related methods
WO2026022584A1 (en) 2024-07-22 2026-01-29 Glaxosmithkline Biologicals Sa Methods of delaying dementia
WO2026024954A2 (en) 2024-07-24 2026-01-29 Altos Labs, Inc. Use of regeneration factors in organ transplantation
WO2026025031A1 (en) 2024-07-26 2026-01-29 Modernatx, Inc. Mrna encoding mageb2 tcer molecule
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026033123A1 (en) 2024-08-08 2026-02-12 Sanofi Pasteur Inc. Lipid nanoparticle formulations for mrna delivery
WO2026044017A2 (en) 2024-08-21 2026-02-26 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2026044055A1 (en) 2024-08-23 2026-02-26 Modernatx, Inc. Muc16 binding molecules, nucleic acids encoding same, and methods of use
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions
WO2026055342A1 (en) 2024-09-04 2026-03-12 Arsenal Biosciences, Inc. Synthetic pathway activators
WO2026055461A1 (en) 2024-09-05 2026-03-12 Aperture Therapeutics, Inc. Antibody oligonucleotide conjugates comprising an antisense polynucleotide agent conjugated to a cd33 antibody and methods of use thereof
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026075820A1 (en) 2024-10-03 2026-04-09 Modernatx, Inc. 4-1bb binding molecules, nucleic acids encoding same, and methods of use
WO2026080379A1 (en) 2024-10-07 2026-04-16 Modernatx, Inc. Lyme disease mrna vaccines
WO2026082899A2 (en) 2024-10-16 2026-04-23 BioNTech SE Rna for preventing or treating tuberculosis
WO2026085300A1 (en) 2024-10-16 2026-04-23 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026085234A1 (en) 2024-10-18 2026-04-23 Modernatx, Inc. 3' untranslated region sequences for extending messenger ribonucleic acids half-life
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
GB202415505D0 (en) 2024-10-21 2024-12-04 King S College London Lipid nanoparticles for cardiac protection
WO2026096575A1 (en) 2024-10-29 2026-05-07 Modernatx, Inc. Mrna encoding concatemeric prame epitope polypeptide
WO2026093547A1 (en) 2024-10-31 2026-05-07 Institut National de la Santé et de la Recherche Médicale Methods and vaccine compositions for the treatment of cancer
WO2026102260A1 (en) 2024-11-08 2026-05-15 Editas Medicine, Inc. Crispr-related methods and compositions targeting angiopoietin like 3 (angptl3)
WO2026112050A2 (en) 2024-11-21 2026-05-28 Modernatx, Inc. Claudin 6 binding molecules, nucleic acids encoding same, and methods of use
WO2026117673A1 (en) 2024-11-30 2026-06-04 Mirus Bio Llc Transfection compositions comprising cationic polymers, cationic lipids, and stabilizing agents
WO2026122478A1 (en) 2024-12-02 2026-06-11 Korro Bio, Inc. Extrahepatic delivery formulations and methods
WO2026119817A1 (en) 2024-12-02 2026-06-11 BioNTech SE Agents and methods for targeted delivery of immune effector cells
WO2026122685A1 (en) 2024-12-03 2026-06-11 Orna Therapeutics, Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2026122882A2 (en) 2024-12-06 2026-06-11 Modernatx, Inc. Solid tumor t cell engagers and uses thereof
WO2026128724A1 (en) 2024-12-11 2026-06-18 Modernatx, Inc. Stabilized flavivirus vaccines
WO2026125590A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026125598A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026136379A1 (en) 2024-12-17 2026-06-25 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2026143002A2 (en) 2024-12-23 2026-07-02 Sanofi Pasteur Inc. Dianhydrohexitol based ionizable lipids for nucleic acid delivery
WO2026143086A1 (en) 2024-12-24 2026-07-02 Modernatx, Inc. Shuffled polypeptide vaccines
WO2026148356A1 (en) 2025-01-06 2026-07-09 BioNTech SE Coronavirus and influenza immunogenic combinations
WO2026151698A1 (en) 2025-01-07 2026-07-16 Modernatx, Inc. Lag3 binding molecules, nucleic acids encoding same, and methods of use
WO2026152023A1 (en) 2025-01-10 2026-07-16 Sail Biomedicines, Inc. Lipid compounds, lipid nanoparticle compositions and use thereof with polyribonucleotides
WO2026150132A1 (en) 2025-01-12 2026-07-16 BioNTech SE Combination therapy comprising an rna cancer vaccine and a multispecific binding agent targeting pd-1/pd-l1 and vegf/vegfr for cancer treatment
WO2026152164A1 (en) 2025-01-13 2026-07-16 Sail Biomedicines, Inc. Cd5-targeted lipid nanoparticles encapsulating polyribonucleotides encoding chimeric antigen receptors, and methods to generate car-expressing t cells in vivo
WO2026155976A1 (en) 2025-01-14 2026-07-23 Modernatx, Inc. Pd-1 binding proteins, nucleic acids encoding same, and methods of use
WO2026161677A1 (en) 2025-01-24 2026-07-30 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026161895A1 (en) 2025-01-27 2026-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for epigenetic regulation of lipoprotein a (lpa) gene expression
WO2026162682A1 (en) 2025-01-29 2026-08-06 BioNTech SE Compositions for delivery of plasmodium rh5 antigens and related methods
WO2026162552A1 (en) 2025-01-31 2026-08-06 BioNTech SE Agents and methods for targeted delivery to cells

Family Cites Families (239)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US105A (en) 1836-12-15 knight
US5218A (en) 1847-08-07 Improvement in plows
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (ru) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5008050A (en) 1984-06-20 1991-04-16 The Liposome Company, Inc. Extrusion technique for producing unilamellar vesicles
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
AU587989B2 (en) 1984-10-16 1989-09-07 Mitsubishi Chemical Corporation DMA fragments, expression vectors, proteins, hosts, and process for production of the proteins
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
ATE89314T1 (de) 1985-02-13 1993-05-15 Scios Nova Inc Menschlicher metallothionein ii-promotor in saeugetierexpressionssystemen.
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
WO1987002062A1 (en) 1985-10-04 1987-04-09 Biotechnology Research Partners, Ltd. Recombinant apolipoproteins and methods
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
GB8712540D0 (en) 1987-05-28 1987-07-01 Ucb Sa Expression of human proapolipoprotein a-i
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5149782A (en) 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US4927637A (en) 1989-01-17 1990-05-22 Liposome Technology, Inc. Liposome extrusion method
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5168045A (en) 1989-08-18 1992-12-01 The Scripps Research Institute Diagnostic systems and methods using polypeptide analogs of apolipoprotein e
US5182364A (en) 1990-02-26 1993-01-26 The Scripps Research Institute Polypeptide analogs of apolipoprotein E
US5473039A (en) 1989-08-18 1995-12-05 The Scripps Research Institute Polypeptide analogs of apolipoprotein E, diagnostic systems and methods using the analogs
US5177189A (en) 1989-08-18 1993-01-05 The Scripps Research Institute Polypeptide analogs of Apolipoprotein E
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
WO1991013080A1 (en) 1990-02-20 1991-09-05 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
JPH06502300A (ja) 1990-08-03 1994-03-17 サノフィ 遺伝子発現の抑制のための化合物及び方法
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
WO1992005186A1 (en) 1990-09-20 1992-04-02 Gilead Sciences Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE69132510T2 (de) 1990-11-08 2001-05-03 Hybridon, Inc. Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
WO1993000443A1 (en) 1991-06-26 1993-01-07 Bio-Technology General Corp. Purification of recombinant apolipoprotein e from bacteria
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
SE9103701D0 (sv) 1991-12-13 1991-12-13 Kabi Pharmacia Ab Apolipoprotein
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
CA2135646A1 (en) 1992-05-11 1993-11-25 Kenneth G. Draper Method and reagent for inhibiting viral replication
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US6372886B1 (en) 1992-06-23 2002-04-16 Arch Development Corp. Expression and purification of kringle domains of human apolipoprotein (a) in E. coli
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
SE9203753D0 (sv) 1992-12-11 1992-12-11 Kabi Pharmacia Ab Expression system for producing apolipoprotein ai-m
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5532130A (en) 1993-07-20 1996-07-02 Dyad Pharmaceutical Corporation Methods and compositions for sequence-specific hybridization of RNA by 2'-5' oligonucleotides
CA2170869C (en) 1993-09-03 1999-09-14 Phillip Dan Cook Amine-derivatized nucleosides and oligonucleosides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5539083A (en) 1994-02-23 1996-07-23 Isis Pharmaceuticals, Inc. Peptide nucleic acid combinatorial libraries and improved methods of synthesis
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
MX9504664A (es) 1994-03-07 1997-05-31 Dow Chemical Co Conjugados de dendrimeros bioactivos y/o dirigidos.
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
EP0751961A1 (en) 1994-03-22 1997-01-08 Research Corporation Technologies, Inc. Eating suppressant peptides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
AU3559695A (en) 1994-09-30 1996-04-26 Inex Pharmaceuticals Corp. Glycosylated protein-liposome conjugates and methods for their preparation
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
SE9500778D0 (sv) 1995-03-03 1995-03-03 Pharmacia Ab Process for producing a protein
US5801155A (en) 1995-04-03 1998-09-01 Epoch Pharmaceuticals, Inc. Covalently linked oligonucleotide minor grove binder conjugates
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
US5672662A (en) 1995-07-07 1997-09-30 Shearwater Polymers, Inc. Poly(ethylene glycol) and related polymers monosubstituted with propionic or butanoic acids and functional derivatives thereof for biotechnical applications
US5672685A (en) 1995-10-04 1997-09-30 Duke University Source of apolipoprotein E and method of isolating apolipoprotein E
US6444806B1 (en) 1996-04-30 2002-09-03 Hisamitsu Pharmaceutical Co., Inc. Conjugates and methods of forming conjugates of oligonucleotides and carbohydrates
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6046166A (en) 1997-09-29 2000-04-04 Jean-Louis Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6037323A (en) 1997-09-29 2000-03-14 Jean-Louis Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6004925A (en) 1997-09-29 1999-12-21 J. L. Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6335437B1 (en) 1998-09-07 2002-01-01 Isis Pharmaceuticals, Inc. Methods for the preparation of conjugated oligomers
ATE397488T1 (de) 1998-11-13 2008-06-15 Heriot William A Vorrichtung zur herstellung von liposomen
WO2000030444A1 (en) * 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7390780B2 (en) * 1999-04-23 2008-06-24 Alza Corporation Gene delivery mediated by liposome-DNA complex with cleavable peg surface modification
AU780535B2 (en) 1999-08-27 2005-03-24 Inex Pharmaceuticals Corp. Compositions for stimulating cytokine secretion and inducing an immune response
US6395437B1 (en) 1999-10-29 2002-05-28 Advanced Micro Devices, Inc. Junction profiling using a scanning voltage micrograph
EP1261620A2 (en) * 2000-02-07 2002-12-04 Roche Diagnostics Corporation Cationic amphiphiles for use in nucleic acid transfection
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
WO2002069369A2 (en) 2000-12-08 2002-09-06 Coley Pharmaceutical Gmbh Cpg-like nucleic acids and methods of use thereof
US20040009216A1 (en) 2002-04-05 2004-01-15 Rodrigueza Wendi V. Compositions and methods for dosing liposomes of certain sizes to treat or prevent disease
US8025587B2 (en) 2008-05-16 2011-09-27 Taylor Made Golf Company, Inc. Golf club
US7745651B2 (en) * 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
JP5639338B2 (ja) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド リポソームの製造システムおよび製造方法
EP1986697B1 (en) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
EP2081949B1 (en) 2006-09-22 2014-12-10 GE Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
CA3144493A1 (en) 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
EP3100718B1 (en) * 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
WO2009132131A1 (en) * 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
EP2937418B1 (en) * 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
US20120149875A1 (en) * 2009-01-12 2012-06-14 Ge Healthcare Bio-Sciences Ab Affinity chromatography matrix
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
DE102009039097B3 (de) 2009-08-27 2010-11-25 Siemens Aktiengesellschaft Verfahren zum Übertragen von Daten in einem Sensornetzwerk, Sensorknoten und Zentral-Rechner
US10077232B2 (en) * 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US8315599B2 (en) 2010-07-09 2012-11-20 Telecommunication Systems, Inc. Location privacy selector
US10867398B2 (en) 2017-11-21 2020-12-15 Reliance Core Consulting LLC Methods, systems, apparatuses and devices for facilitating motion analysis in an environment
KR102318555B1 (ko) 2020-03-19 2021-10-29 한국과학기술연구원 광소자용 역나노콘과 그 제조방법

Also Published As

Publication number Publication date
SG10201912450XA (en) 2020-03-30
IL216876A0 (en) 2012-02-29
US8802644B2 (en) 2014-08-12
SI3431076T1 (sl) 2022-04-29
US20170143631A1 (en) 2017-05-25
AU2021201228B2 (en) 2022-07-07
JP6132321B2 (ja) 2017-05-24
EP2440183B1 (en) 2018-07-18
KR20220038506A (ko) 2022-03-28
SG176786A1 (en) 2012-01-30
CN102625696B (zh) 2015-06-03
LT3431076T (lt) 2021-10-25
US20120183602A1 (en) 2012-07-19
SG10201403054SA (en) 2014-10-30
AU2021201228A1 (en) 2021-03-11
CY1124769T1 (el) 2022-11-25
JP2015232048A (ja) 2015-12-24
KR20120081065A (ko) 2012-07-18
CN104873464A (zh) 2015-09-02
CN104873464B (zh) 2018-06-22
KR101766408B1 (ko) 2017-08-10
AU2017202702A1 (en) 2017-05-18
NZ596958A (en) 2014-04-30
CA2764609C (en) 2018-10-02
HK1212620A1 (zh) 2016-06-17
US20100324120A1 (en) 2010-12-23
DK2440183T3 (en) 2018-10-01
PL2440183T3 (pl) 2019-01-31
EP2440183A4 (en) 2013-02-27
US8158601B2 (en) 2012-04-17
WO2010144740A1 (en) 2010-12-16
EA201690312A1 (ru) 2016-08-31
KR20170091798A (ko) 2017-08-09
PT3431076T (pt) 2021-10-26
MX342785B (es) 2016-10-12
KR102374518B1 (ko) 2022-03-16
LT2440183T (lt) 2018-08-10
SMT202100696T1 (it) 2022-01-10
AU2019204984A1 (en) 2019-08-01
EP3431076B1 (en) 2021-10-06
EA201791744A2 (ru) 2018-03-30
PT2440183T (pt) 2018-10-30
US20150166465A1 (en) 2015-06-18
SMT201800499T1 (it) 2018-11-09
US9394234B2 (en) 2016-07-19
CA3014827A1 (en) 2010-12-16
AU2019204984B2 (en) 2021-01-28
MX367665B (es) 2019-08-30
EA024960B1 (ru) 2016-11-30
KR20200006176A (ko) 2020-01-17
JP5819291B2 (ja) 2015-11-24
KR102205886B1 (ko) 2021-01-21
MX2019010340A (es) 2019-10-14
AU2017202702B2 (en) 2019-05-02
AU2010259984B2 (en) 2017-03-09
DK3431076T3 (da) 2021-12-20
IL244945B (en) 2020-06-30
JP2012530059A (ja) 2012-11-29
NZ622843A (en) 2015-10-30
EA201791744A3 (ru) 2018-07-31
KR102066189B1 (ko) 2020-01-14
MX2011013320A (es) 2012-02-28
KR20210008938A (ko) 2021-01-25
IL290077A (en) 2022-03-01
PL3431076T3 (pl) 2022-01-31
EP3431076A1 (en) 2019-01-23
CN102625696A (zh) 2012-08-01
NZ712719A (en) 2017-03-31
TR201811076T4 (tr) 2018-08-27
IL274826A (en) 2020-07-30
CA2764609A1 (en) 2010-12-16
KR20230098713A (ko) 2023-07-04
JP6592144B2 (ja) 2019-10-16
EP2440183A1 (en) 2012-04-18
HUE056773T2 (hu) 2022-03-28
JP6359719B2 (ja) 2018-07-18
SI2440183T1 (sl) 2018-10-30
HRP20181221T1 (hr) 2018-10-05
KR20190065474A (ko) 2019-06-11
ES2689168T3 (es) 2018-11-08
EA028860B1 (ru) 2018-01-31
IL244945A0 (en) 2016-05-31
KR101987962B1 (ko) 2019-06-11
HUE038796T2 (hu) 2018-11-28
HRP20211619T1 (hr) 2022-02-04
AU2010259984A1 (en) 2012-01-12
JP2017122126A (ja) 2017-07-13
IL216876A (en) 2016-04-21
JP2018141019A (ja) 2018-09-13
CY1120641T1 (el) 2019-12-11
ES2901627T3 (es) 2022-03-23

Similar Documents

Publication Publication Date Title
EA201791744A3 (ru) Улучшенная липидная композиция
JOP20180102B1 (ar) مركب صيدلاني
EA200971140A1 (ru) Триарильные соединения и композиции, их содержащие
CY1119684T1 (el) Αντι-ιικες ενωσεις
EA201001680A1 (ru) Соединения бензолсульфонамидтиазола и оксазола
EA201500653A1 (ru) Композиции, содержащие триазольное соединение
EA201190242A1 (ru) Липидные композиции
EA201190235A1 (ru) Замещенные 1-цианоэтилгетероциклилкарбоксамидные соединения 750
EA201490596A1 (ru) Новые производные дигидрохинолин-2-она
EA201071339A1 (ru) Фосфорсодержащие производные в качестве ингибиторов киназы
EA201490567A1 (ru) Новые бициклические производные дигидрохинолин-2-она
EA201290183A1 (ru) Бензодиазепиновый ингибитор бромодомена
GEP20156266B (en) Spiroheterocyclic n-oxypiperidines as pesticides
CR11861A (es) Compuestos organicos
EA200870514A1 (ru) Ацетиленовые гетероарильные соединения
EA201490846A1 (ru) Новые производные арилхинолина
BRPI1015043A2 (pt) derivados de diazahomoadamantano e métodos de uso dos mesmos
EA201590887A1 (ru) Композиция
EA201171246A1 (ru) Содержащая трегалулозу композиция, ее получение и применение
EA201101478A1 (ru) Композиции и способы модулирования связывания ретинола с ретинолсвязывающим белком 4 (rbp4)
EA201300443A1 (ru) Способ получения дигидроптеридинонов и их промежуточных продуктов
EA201300337A1 (ru) Композиция, содержащая пирипиропеновый инсектицид и основание
EA201270253A1 (ru) Производные n1-ацил-5-фторпиримидинона
BR112014004523A2 (pt) composto e composição aromatizante
EA201071367A1 (ru) Соли соединений ингибиторов вич

Legal Events

Date Code Title Description
TC4A Change in name of a patent proprietor in a eurasian patent