MA53609A - Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose - Google Patents

Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose

Info

Publication number
MA53609A
MA53609A MA053609A MA53609A MA53609A MA 53609 A MA53609 A MA 53609A MA 053609 A MA053609 A MA 053609A MA 53609 A MA53609 A MA 53609A MA 53609 A MA53609 A MA 53609A
Authority
MA
Morocco
Prior art keywords
glycogenosis
phosphatase
treatment
polynucleotides encoded
glucose
Prior art date
Application number
MA053609A
Other languages
English (en)
Inventor
Jingsong Cao
Athanasios Dousis
Paolo G V Martini
Vladimir Presnyak
Original Assignee
Modernatx Inc
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 Modernatx Inc filed Critical Modernatx Inc
Publication of MA53609A publication Critical patent/MA53609A/fr

Links

Classifications

    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Optics & Photonics (AREA)
  • Dermatology (AREA)
  • Nanotechnology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
MA053609A 2018-09-13 2019-09-12 Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose MA53609A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862730940P 2018-09-13 2018-09-13
US201962812800P 2019-03-01 2019-03-01

Publications (1)

Publication Number Publication Date
MA53609A true MA53609A (fr) 2021-07-21

Family

ID=68084944

Family Applications (1)

Application Number Title Priority Date Filing Date
MA053609A MA53609A (fr) 2018-09-13 2019-09-12 Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose

Country Status (9)

Country Link
US (2) US20230009009A1 (fr)
EP (1) EP3849595B1 (fr)
JP (1) JP2022500436A (fr)
CN (1) CN113164561A (fr)
AU (1) AU2019337637A1 (fr)
CA (1) CA3112208A1 (fr)
MA (1) MA53609A (fr)
MX (1) MX2021003015A (fr)
WO (1) WO2020056147A2 (fr)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021247535A1 (fr) * 2020-06-01 2021-12-09 Modernatx, Inc. Nanoparticules lipidiques contenant des polynucléotides codant pour la glucose-6-phosphatase et leurs utilisations
IL299196A (en) * 2020-06-23 2023-02-01 Modernatx Inc LNP preparations that include mRNA treatments with extended lifespan
US20240207374A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2023009499A1 (fr) * 2021-07-27 2023-02-02 Modernatx, Inc. Polynucléotides codant pour la glucose-6-phosphatase pour le traitement de la glycogénose de type 1a (gsd1a)
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
WO2023246218A1 (fr) * 2022-06-20 2023-12-28 成都威斯津生物医药科技有限公司 Lipide ionisable pour l'administration d'acides nucléiques et composition associée
WO2025021183A1 (fr) * 2023-07-27 2025-01-30 上海复宏汉霖生物医药有限公司 Polynucléotide codant pour l'hormone adrénocorticotropique, composition associée et méthode associée
CN117771354B (zh) * 2024-02-23 2024-07-05 云南省畜牧兽医科学院 一种预防O型口蹄疫的mRNA-LNP疫苗及其制备方法
CN119587504B (zh) * 2024-12-10 2025-11-14 华中科技大学同济医学院附属协和医院 一种靶向骨组织巨噬细胞的纳米颗粒及其制备方法与应用

Family Cites Families (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
US5563250A (en) 1987-12-02 1996-10-08 Neorx Corporation Cleavable conjugates for the delivery and release of agents in native form
US5505931A (en) 1993-03-04 1996-04-09 The Dow Chemical Company Acid cleavable compounds, their preparation and use as bifunctional acid-labile crosslinking agents
US5723332A (en) 1993-11-26 1998-03-03 British Technology Group Limited Translational enhancer DNA
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6004573A (en) 1997-10-03 1999-12-21 Macromed, Inc. Biodegradable low molecular weight triblock poly(lactide-co-glycolide) polyethylene glycol copolymers having reverse thermal gelation properties
WO1999029758A1 (fr) 1997-12-12 1999-06-17 Samyang Corporation Poly[acide alpha-(omega-aminoalkyl) glycolique] pour le transport d'un agent bioactif par voie tissulaire et penetration cellulaire
US6517869B1 (en) 1997-12-12 2003-02-11 Expression Genetics, Inc. Positively charged poly(alpha-(omega-aminoalkyl)lycolic acid) for the delivery of a bioactive agent via tissue and cellular uptake
ES2356342T3 (es) 1998-01-05 2011-04-07 The University Of Washington Mejora de transporte utilizando agentes de alteración de membrana.
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
CN1333678A (zh) 1998-05-20 2002-01-30 表达遗传学公司 定向肝细胞的聚乙二醇接枝的聚－l－赖氨酸的聚合基因载体
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
CN1313873A (zh) 1998-07-13 2001-09-19 表达遗传学公司 作为可溶性,生物可降解基因送递载体的聚－l－赖氨酸的聚酯类似物
EP1102785B1 (fr) 1999-06-07 2013-02-13 Arrowhead Research Corporation Compositions de diffusion de medicaments au moyen de molecules sensibles au ph
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
AU2764801A (en) 2000-01-07 2001-07-24 University Of Washington Enhanced transport of agents using membrane disruptive agents
US6727066B2 (en) * 2000-07-28 2004-04-27 Incyte Corporation Genes expressed in treated human C3A liver cell cultures
US6696038B1 (en) 2000-09-14 2004-02-24 Expression Genetics, Inc. Cationic lipopolymer as biocompatible gene delivery agent
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US6673549B1 (en) * 2000-10-12 2004-01-06 Incyte Corporation Genes expressed in C3A liver cell cultures treated with steroids
US6897196B1 (en) 2001-02-07 2005-05-24 The Regents Of The University Of California pH sensitive lipids based on ortho ester linkers, composition and method
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US6586524B2 (en) 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
EP1412065A2 (fr) 2001-07-27 2004-04-28 President And Fellows Of Harvard College Appareil et procedes de melange de flux laminaires
EP2428570B1 (fr) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Molécules de micro arn
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
AU2003217531A1 (en) 2002-05-02 2003-11-17 Massachusetts Eye And Ear Infirmary Ocular drug delivery systems and use thereof
US7883720B2 (en) 2003-07-09 2011-02-08 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
WO2005013901A2 (fr) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants
WO2005084180A2 (fr) 2003-12-19 2005-09-15 University Of Cincinnati Polyamides et complexes a base de polyamide pour la distribution de leurres oligonucleotidiques
CA2563533C (fr) 2004-04-15 2013-10-01 Shmuel A. Ben-Sasson Compositions capables de faciliter la penetration a travers une barriere biologique
US20050261233A1 (en) * 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
US8057821B2 (en) 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
WO2006063249A2 (fr) 2004-12-10 2006-06-15 Justin Hanes Copolymeres sequences de poly(ether-anhydride) fonctionnalises
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US8273339B2 (en) 2005-04-12 2012-09-25 Nektar Therapeutics Polymer-based compositions and conjugates of antimicrobial agents
AU2006265896B2 (en) 2005-06-30 2012-05-31 Archemix Corp. Materials and methods for the generation of fully 2'-modified nucleic acid transcripts
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
PL2578685T3 (pl) * 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
CN102973929B (zh) 2005-09-01 2017-04-26 诺华疫苗和诊断有限两合公司 含血清群c脑膜炎球菌的多价疫苗
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP1968643A2 (fr) 2005-12-16 2008-09-17 Diatos Conjugués peptidiques de pénétration cellulaire pour la délivrance d'acides nucléiques dans une cellule
JP5295785B2 (ja) 2006-02-20 2013-09-18 エファ・ユニバーシティ・インダストリー・コラボレイション・ファウンデイション 細胞膜透過性ペプチド
EP2319542B1 (fr) 2006-02-21 2018-03-21 Nektar Therapeutics Polymères dégradables segmentés et conjugués obtenus à partir de ceux-ci
WO2012024613A1 (fr) 2010-08-19 2012-02-23 Wintrance Corporation Conjugués synergiques biomolécule-polymère
US8313777B2 (en) 2006-10-05 2012-11-20 The Johns Hopkins University Water-dispersible oral, parenteral, and topical formulations for poorly water soluble drugs using smart polymeric nanoparticles
US20100210792A1 (en) 2006-12-05 2010-08-19 David Taft Drug delivery
EP2120859B1 (fr) 2006-12-21 2013-11-20 Stryker Corporation Formulations à libération entretenue comprenant des cristaux de bmp-7
AU2007337810B2 (en) 2006-12-22 2014-02-13 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
WO2008082613A2 (fr) 2006-12-27 2008-07-10 Nektar Therapeutics Al, Corporation Conjugués polymère-fraction à activité facteur ix comprenant une liaison dégradable
WO2008103276A2 (fr) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions et méthodes de potentialisation de l'activité de molécules biologiquement actives
LT2134353T (lt) 2007-03-30 2017-03-27 Xisle Pharma Ventures Trust Dvifazė lipidų vezikulių kompozicija ir gimdos kaklelio displazijos gydymo būdas, skiriant intravaginaliai
EP3434259A1 (fr) 2007-05-04 2019-01-30 Marina Biotech, Inc. Lipides d'acides aminés et utilisations associées
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
ATE530169T1 (de) 2007-09-28 2011-11-15 Bind Biosciences Inc Krebszellen-targeting mit nanopartikeln
EP2271699A1 (fr) 2008-03-14 2011-01-12 Egen, Inc. Poly(alkylène imines) ramifiées et réticulées, biodégradables
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
EA020753B1 (ru) 2008-06-16 2015-01-30 Бинд Терапьютикс, Инк. Терапевтические полимерные наночастицы, содержащие алкалоиды vinca, и их применение
JP2011525180A (ja) 2008-06-16 2011-09-15 バインド バイオサイエンシズ インコーポレイテッド 治療的標的化ナノ粒子の製作に用いるためのジブロックコポリマーで官能化された標的薬の製造方法
AU2009268923B2 (en) 2008-06-16 2015-09-17 Pfizer Inc. Drug loaded polymeric nanoparticles and methods of making and using same
WO2010005726A2 (fr) 2008-06-16 2010-01-14 Bind Biosciences Inc. Nanoparticules polymères thérapeutiques avec inhibiteurs de mtor et procédés de fabrication et d’utilisation associés
WO2010030763A2 (fr) 2008-09-10 2010-03-18 Bind Biosciences, Inc. Fabrication de nanoparticles à rendement élevé
WO2010053572A2 (fr) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Lipidoïdes aminoalcool et leurs utilisations
EA201100765A1 (ru) 2008-12-15 2012-04-30 Бинд Биосаиэнсис Наночастицы длительной циркуляции для замедленного высвобождения терапевтических средств
WO2010087791A1 (fr) 2009-01-27 2010-08-05 Utc Power Corporation Réacteur de conversion à la vapeur d'eau intégré, refroidi de manière distributive et atomiseur
PT2414322T (pt) 2009-03-20 2022-12-07 Egen Inc Derivados de poliamina
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
RU2573409C2 (ru) 2009-11-04 2016-01-20 Дзе Юниверсити Оф Бритиш Коламбиа Содержащие нуклеиновые кислоты липидные частицы и относящиеся к ним способы
WO2011062965A2 (fr) 2009-11-18 2011-05-26 University Of Washington Through Its Center For Commercialization Monomères de ciblage et polymère ayant des blocs de ciblage
WO2011069529A1 (fr) 2009-12-09 2011-06-16 Curevac Gmbh Solution contenant du mannose pour la lyophilisation, la transfection et/ou l'injection d'acides nucléiques
JP5965844B2 (ja) 2009-12-15 2016-08-10 バインド セラピューティックス インコーポレイテッド 高いガラス転移温度または高分子量のコポリマーを有する治療用ポリマーナノ粒子組成物
EP2512487A4 (fr) 2009-12-15 2013-08-07 Nanoparticules polymères thérapeutiques comprenant de corticostéroïdes, et procédés pour les fabriquer et les utiliser
EP2512459A4 (fr) 2009-12-15 2013-08-07 Nanoparticules polymères thérapeutiques comportant de l'épothilone et leurs procédés de fabrication et d'utilisation
WO2011106086A1 (fr) 2010-02-25 2011-09-01 Albert Einstein College Of Medicine Of Yeshiva University Polymères d'albumine pégylés et leurs utilisations
EP3391877A1 (fr) 2010-04-08 2018-10-24 The Trustees of Princeton University Préparation de nanoparticules lipidiques
CN104922071A (zh) 2010-04-09 2015-09-23 帕西拉制药有限公司 用于配制大直径合成膜囊泡的方法
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
NZ704191A (en) 2010-06-14 2016-05-27 Hoffmann La Roche Cell-penetrating peptides and uses therof
US20130196948A1 (en) 2010-06-25 2013-08-01 Massachusetts Insitute Of Technology Polymers for biomaterials and therapeutics
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
FI4005592T3 (fi) 2010-07-06 2023-01-13 Virionin kaltaisia kuljetuspartikkeleita itsereplikoituville rna-molekyyleille
CA2804591C (fr) 2010-07-06 2019-01-22 Novartis Ag Emulsions cationiques huile-dans-eau
US20130211249A1 (en) 2010-07-22 2013-08-15 The Johns Hopkins University Drug eluting hydrogels for catheter delivery
EP2449113B8 (fr) 2010-07-30 2015-11-25 CureVac AG Complexation d'acides nucléiques avec des composants cationiques réticulés par des liaisons disulfure pour la transfection et l'immunostimulation
US9121065B2 (en) 2010-08-09 2015-09-01 The Trustees Of The University Of Pennsylvania Nanoparticle-oligonucleotide hybrid structures and methods of use thereof
US20120225129A1 (en) 2010-08-20 2012-09-06 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
RS63890B1 (sr) 2010-08-31 2023-02-28 Glaxosmithkline Biologicals Sa Mali lipozomi za isporuku rnk koja kodira imunogen
EP3542789A3 (fr) 2010-08-31 2020-01-01 GlaxoSmithKline Biologicals SA Lipides adaptés pour une administration liposomale d'arn codant pour une protéine
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
WO2012039979A2 (fr) 2010-09-10 2012-03-29 The Johns Hopkins University Diffusion rapide de grosses nanoparticules polymères dans le cerveau de mammifères
JP5927194B2 (ja) 2010-09-24 2016-06-01 マリンクロッド エルエルシー 治療ナノキャリアおよび／または診断ナノキャリアのターゲティングのためのアプタマーコンジュゲート
EA201390600A1 (ru) 2010-10-22 2013-09-30 Байнд Терапьютикс, Инк. Терапевтические наночастицы с сополимерами с большим молекулярным весом
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
US20140066363A1 (en) 2011-02-07 2014-03-06 Arun K. Bhunia Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2489371A1 (fr) 2011-02-18 2012-08-22 Instituto Nacional de Investigacion y Tecnologia Agraria y Alimentaria Support peptidique pour l'administration de médicaments
US8846850B2 (en) 2011-02-22 2014-09-30 Rutgers, The State University Of New Jersey Amphiphilic macromolecules for nucleic acid delivery
CA2831613A1 (fr) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Administration et formulation d'acides nucleiques genetiquement modifies
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EP4074693A1 (fr) 2011-06-08 2022-10-19 Translate Bio, Inc. Lipides clivables
KR20140067070A (ko) 2011-08-31 2014-06-03 말린크로트 엘엘씨 Ｈ-포스포네이트에 의한 나노입자 ｐｅｇ 개질
WO2013039857A1 (fr) 2011-09-12 2013-03-21 modeRNA Therapeutics Acides nucléiques modifiés et leurs procédés d'utilisation
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
EP2785326A2 (fr) 2011-11-29 2014-10-08 The University of North Carolina at Chapel Hill Particules manipulées de façon géométrique et procédés de modulation de réponses des macrophages ou immunitaires
WO2013086322A1 (fr) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipides biodégradables ramifiés à terminaisons alkyle et cycloalkyle destinés à l'administration d'agents actifs
DE21212055T1 (de) 2011-12-07 2022-08-04 Alnylam Pharmaceuticals, Inc. Biologisch abbaubare lipide zur freisetzung von wirkstoffen
EP2787977A4 (fr) 2011-12-09 2015-05-06 Univ California Encapsulation liposomale de médicaments
WO2013106086A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013106072A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013106073A1 (fr) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprenant des polymères réticulés de liaison à des cations et utilisations associées
WO2013105101A1 (fr) 2012-01-13 2013-07-18 Department Of Biotechnology Nanoparticules lipidiques solides encapsulant un médicament hydrophile/amphiphile et leur procédé de préparation
US9415020B2 (en) 2012-01-19 2016-08-16 The Johns Hopkins University Nanoparticle formulations with enhanced mucosal penetration
WO2013116126A1 (fr) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Nouveaux lipides cationiques biodégradables de faible masse moléculaire pour la délivrance d'oligonucléotides
US10416167B2 (en) 2012-02-17 2019-09-17 University Of Georgia Research Foundation, Inc. Nanoparticles for mitochondrial trafficking of agents
CN104394853B (zh) 2012-02-19 2017-10-10 纳维基因股份有限公司 多孔性纳米结构在递送中的用途
WO2013124867A1 (fr) 2012-02-21 2013-08-29 Amrita Vishwa Vidyapeetham University Polymer - polymer or polymer - protein core - shell nano medicine loaded with multiple drug molecules
WO2013151664A1 (fr) 2012-04-02 2013-10-10 modeRNA Therapeutics Polynucléotides modifiés pour la production de protéines
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
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
WO2014093924A1 (fr) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Molécules d'acide nucléique modifiées et leurs utilisations
WO2014164253A1 (fr) 2013-03-09 2014-10-09 Moderna Therapeutics, Inc. Régions non traduites hétérologues pour arnm
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
JP6649265B2 (ja) * 2013-11-26 2020-02-19 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 糖原病の処置のためのアデノ随伴ウイルスベクター
WO2015130584A2 (fr) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Adjuvants de vaccins sous forme de nanoparticules lipidiques et systèmes d'administration d'antigènes
EP4729528A3 (fr) * 2014-04-25 2026-06-24 Translate Bio, Inc. Procédés de purification d'arn messager
EP3236984B1 (fr) * 2014-12-23 2020-07-22 The U.S.A. as represented by the Secretary, Department of Health and Human Services Vecteurs à base de virus adéno-associé codant pour une g6pc modifiée, et utilisations de ces derniers
JP2017019215A (ja) * 2015-07-13 2017-01-26 富士フイルム株式会社 着色積層体およびその製造方法
WO2017100551A1 (fr) * 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. Séquences utr hétérologues pour l'amplification de l'expression d'arnm
EP3405579A1 (fr) * 2016-01-22 2018-11-28 Modernatx, Inc. Acides ribonucléiques messagers pour la production de polypeptides de liaison intracellulaires et leurs procédés d'utilisation
WO2017191274A2 (fr) * 2016-05-04 2017-11-09 Curevac Ag Arn codant pour une protéine thérapeutique
EP3658672A1 (fr) * 2017-07-24 2020-06-03 Modernatx, Inc. Arnm modifié codant une glucose-6-phosphatase et utilisations associées
US20240207374A1 (en) * 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof

Also Published As

Publication number Publication date
CA3112208A1 (fr) 2020-03-19
US20230009009A1 (en) 2023-01-12
EP3849595A2 (fr) 2021-07-21
WO2020056147A2 (fr) 2020-03-19
US20250170070A1 (en) 2025-05-29
EP3849595B1 (fr) 2025-11-19
MX2021003015A (es) 2021-07-15
JP2022500436A (ja) 2022-01-04
AU2019337637A1 (en) 2021-04-22
WO2020056147A3 (fr) 2020-04-16
CN113164561A (zh) 2021-07-23

Similar Documents

Publication Publication Date Title
MA45041A (fr) Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1
MA47719A (fr) Esketamine pour le traitement de la dépression
MA45036A (fr) Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
EP3893940A4 (fr) Polythérapie pour le traitement de la dystrophie musculaire
EP3458106A4 (fr) Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
EP3448875A4 (fr) Compositions pour le traitement de maladies
EP3448987A4 (fr) Compositions pour le traitement de maladies
EP3600281A4 (fr) Traitement d'association pour le traitement ou la prévention de tumeurs
EP3370703A4 (fr) Combinaison de gemcabène pour le traitement de maladies cardiovasculaires
EP3359074A4 (fr) Électroporation pour le traitement de l'obésité ou le diabète
MA45481A (fr) Utilisation d'exosomes pour le traitement de maladies
EP3503890A4 (fr) Utilisation de la pridopidine pour le traitement des dystonies
EP3504187A4 (fr) Utilisation de la pridopidine pour le traitement du déclin fonctionnel
MA41449A (fr) Polythérapies pour le traitement de cancers
MA47820A (fr) Traitement de la glycogénose de type iii
EP3630196A4 (fr) Polythérapies pour le traitement de cancers
EP3335041A4 (fr) Biomarqueurs pour le traitement de la pelade
MA50801A (fr) Polynucléotides codant pour la phénylalanine hydroxylase pour le traitement de la phénylcétonurie
EP3362065A4 (fr) Polythérapie pour le traitement de malignités
EP3328374A4 (fr) Antagonistes de la glutamine pour le traitement de déficits cognitifs
MA55218A (fr) Eskétamine pour le traitement de la dépression
EP3436014A4 (fr) Polythérapie pour le traitement de la leucémie myéloïde aiguë
EP3766497A4 (fr) Médicament pour le traitement de la toux
EP3541379A4 (fr) Compositions pour le traitement de l'hypertension
EP3386967A4 (fr) Combinaisons pour le traitement de calculs rénaux