ATE536418T1 - Lipidverkapselte interferenz-rna - Google Patents

Lipidverkapselte interferenz-rna

Info

Publication number
ATE536418T1
ATE536418T1 AT05757651T AT05757651T ATE536418T1 AT E536418 T1 ATE536418 T1 AT E536418T1 AT 05757651 T AT05757651 T AT 05757651T AT 05757651 T AT05757651 T AT 05757651T AT E536418 T1 ATE536418 T1 AT E536418T1
Authority
AT
Austria
Prior art keywords
interference rna
lipid
lipid encapsulated
based formulations
encapsulated
Prior art date
Application number
AT05757651T
Other languages
English (en)
Inventor
Ian Maclachlan
Lorne R Palmer
James Heyes
Original Assignee
Protiva Biotherapeutics 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 Protiva Biotherapeutics Inc filed Critical Protiva Biotherapeutics Inc
Application granted granted Critical
Publication of ATE536418T1 publication Critical patent/ATE536418T1/de

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    • 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
    • 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
    • A61K31/70—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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/35—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AT05757651T 2004-06-07 2005-06-07 Lipidverkapselte interferenz-rna ATE536418T1 (de)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US57796104P 2004-06-07 2004-06-07
US57807504P 2004-06-07 2004-06-07
US61074604P 2004-09-17 2004-09-17
US67942705P 2005-05-09 2005-05-09
PCT/CA2005/000886 WO2005121348A1 (en) 2004-06-07 2005-06-07 Lipid encapsulated interfering rna

Publications (1)

Publication Number Publication Date
ATE536418T1 true ATE536418T1 (de) 2011-12-15

Family

ID=35503071

Family Applications (1)

Application Number Title Priority Date Filing Date
AT05757651T ATE536418T1 (de) 2004-06-07 2005-06-07 Lipidverkapselte interferenz-rna

Country Status (7)

Country Link
US (4) US7799565B2 (de)
EP (1) EP1766035B1 (de)
JP (1) JP4796062B2 (de)
AT (1) ATE536418T1 (de)
AU (1) AU2005252273B2 (de)
CA (1) CA2569664C (de)
WO (1) WO2005121348A1 (de)

Families Citing this family (559)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE60334618D1 (de) 2002-06-28 2010-12-02 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
KR101168440B1 (ko) * 2003-07-16 2012-07-27 프로티바 바이오쎄라퓨틱스, 인코포레이티드 지질 캡슐화된 간섭 ｒｎａ
NZ581166A (en) * 2003-09-15 2011-06-30 Protiva Biotherapeutics Inc Polyethyleneglycol-modified lipid compounds and uses thereof
EP2899278A1 (de) 2004-03-12 2015-07-29 Alnylam Pharmaceuticals Inc. iRNS-Wirkstoffe für VEGF-Targeting
ATE536418T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
AU2005259799A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
JP2008509205A (ja) * 2004-08-13 2008-03-27 アイシー・ベック・リミテッド ポリマー修飾ｓｉＲＮＡリポソームを含むベクター
GB0418172D0 (en) * 2004-08-13 2004-09-15 Ic Vec Ltd Vector
ATE514776T1 (de) 2004-10-05 2011-07-15 California Inst Of Techn Aptamer-regulierte nukleinsäuren und verwendungen davon
AU2005306533B2 (en) * 2004-11-17 2012-05-31 Arbutus Biopharma Corporation siRNA silencing of apolipoprotein B
US9393315B2 (en) 2011-06-08 2016-07-19 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing siRNA activity
TR201909609T4 (tr) 2005-08-23 2019-07-22 Univ Pennsylvania Modifiye edilmiş nükleosidleri içeren rna ve kullanım yöntemleri.
US20070054873A1 (en) * 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
US20070087045A1 (en) * 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
TR201809912T4 (tr) 2006-02-14 2018-07-23 Bp Corp North America Inc Ksilanazlar, bunları kodlayan nükleik asitler ve bunları yapmak ve kullanmak için yöntemler.
CA2645225A1 (en) 2006-03-07 2007-09-13 Verenium Corporation Aldolases, nucleic acids encoding them and methods for making and using them
MX2008011477A (es) 2006-03-07 2008-09-23 Cargill Inc Aldolasas, acidos nucleicos que las codifican y metodos para hacerlas y usarlas.
SG171676A1 (en) 2006-05-11 2011-06-29 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
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US8138160B2 (en) * 2006-08-03 2012-03-20 Warsaw Orthopedic, Inc. Reagents, methods and systems to suppress pro-inflammatory cytokines
US20080171716A1 (en) * 2006-08-16 2008-07-17 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
US20100099737A1 (en) * 2006-08-24 2010-04-22 Gerald Krystal Compositions and methods for treating myelosuppression
MX363224B (es) * 2006-10-03 2019-03-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
WO2008058291A2 (en) * 2006-11-09 2008-05-15 California Institute Of Technology Modular aptamer-regulated ribozymes
KR100817024B1 (ko) * 2006-11-09 2008-03-26 재단법인 목암생명공학연구소 핵산 또는 약물을 간에 특이적으로 전달하는 복합체 및이를 포함하는 약학적 조성물
KR100825519B1 (ko) * 2007-01-05 2008-04-25 주식회사 바이오폴리메드 키토산 기재 고분자 접합체 및 그 제조방법
EA019514B1 (ru) 2007-04-27 2014-04-30 Те Риджентс Оф Те Юниверсити Оф Калифорния Сенсоры coрастений, нуклеиновые кислоты, кодирующие их, способы их получения и применение
WO2009002719A1 (en) * 2007-06-22 2008-12-31 The Board Of Regents Of The University Of Texas System Liposomal inhibitory nucleic acid against stat proteins
US20090082217A1 (en) * 2007-07-16 2009-03-26 California Institute Of Technology Selection of nucleic acid-based sensor domains within nucleic acid switch platform
US8716255B2 (en) 2007-08-10 2014-05-06 British Columbia Cancer Agency Branch Microrna compositions and methods for the treatment of myelogenous leukemia
US8367815B2 (en) * 2007-08-28 2013-02-05 California Institute Of Technology Modular polynucleotides for ligand-controlled regulatory systems
US20120165387A1 (en) 2007-08-28 2012-06-28 Smolke Christina D General composition framework for ligand-controlled RNA regulatory systems
US8865667B2 (en) 2007-09-12 2014-10-21 California Institute Of Technology Higher-order cellular information processing devices
CN103757036B (zh) 2007-10-03 2019-10-01 维莱尼姆公司 木聚糖酶、编码它们的核酸以及其制备和应用方法
CA2702494A1 (en) 2007-10-19 2009-04-23 The Regents Of The University Of California Compositions and methods for ameliorating cns inflammation, psychosis, delirium, ptsd or ptss
JP2011503188A (ja) * 2007-11-14 2011-01-27 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ステロール修飾両親媒性脂質
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
CA2711236A1 (en) * 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
EP2224912B1 (de) * 2008-01-02 2016-05-11 TEKMIRA Pharmaceuticals Corporation Verbesserte zusammensetzungen und verfahren zur freisetzung von nukleinsäuren
WO2009088949A1 (en) 2008-01-03 2009-07-16 Verenium Corporation Transferases and oxidoreductases, nucleic acids encoding them and methods for making and using them
EP2706122A3 (de) 2008-01-03 2014-06-18 Verenium Corporation Isomerasen, Nukleinsäuren zur Codierung davon und Verfahren zur Herstellung und Verwendung davon
MX2010008394A (es) * 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
SG188121A1 (en) * 2008-03-05 2013-03-28 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of eg5 and vegf genes
RU2501859C2 (ru) 2008-03-31 2013-12-20 Нэшнл Инститьют Оф Эдванст Индастриал Сайенс Энд Текнолоджи Модифицированная липидами двухцепочечная рнк, обладающая мощным эффектом рнк-интерференции
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
EP2281041B1 (de) 2008-04-15 2014-07-02 Protiva Biotherapeutics Inc. Abdämpfen der csn5 genexpression unter verwendung von interferierender rns
WO2009129395A1 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
WO2009129385A1 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
US20110224447A1 (en) * 2008-08-18 2011-09-15 Bowman Keith A Novel Lipid Nanoparticles and Novel Components for Delivery of Nucleic Acids
US20100099738A1 (en) * 2008-09-10 2010-04-22 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
TW201019963A (en) * 2008-09-10 2010-06-01 Abbott Lab Polyethylene glycol lipid conjugates and uses thereof
WO2010033248A2 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
EP3584320A1 (de) 2008-09-25 2019-12-25 Alnylam Pharmaceuticals, Inc. Zusammensetzungen aus lipidformulierungen und verfahren zur hemmung der serum-amyloid-a-genexpression
EP2743265B1 (de) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Verbesserte Aminolipide und Verfahren zur Freisetzung von Nukleinsäuren
DK2344639T3 (en) 2008-10-20 2015-07-27 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETINE
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
CA3029724A1 (en) 2008-11-10 2010-05-14 Muthiah Manoharan Lipids and compositions for the delivery of therapeutics
KR101727333B1 (ko) * 2008-11-26 2017-04-14 추가이 세이야쿠 가부시키가이샤 베시클 제제
WO2010061881A1 (ja) 2008-11-26 2010-06-03 中外製薬株式会社 Ｃ型肝炎ウイルスの働きを阻害するオリゴリボヌクレオチドまたはペプチド核酸
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US8329882B2 (en) 2009-02-18 2012-12-11 California Institute Of Technology Genetic control of mammalian cells with synthetic RNA regulatory systems
EP2406376A1 (de) * 2009-03-12 2012-01-18 Alnylam Pharmaceuticals, Inc. Lipidformulierte zusammensetzungen und verfahren zur hemmung der expression von eg5- und vegf-genen
US9145555B2 (en) 2009-04-02 2015-09-29 California Institute Of Technology Integrated—ligand-responsive microRNAs
EP3504967A1 (de) * 2009-05-05 2019-07-03 Arbutus Biopharma Corporation Verfahren zur abgabe von oligonukleotiden an immunzellen
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
KR20120050429A (ko) * 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Ｐｃｓｋ9 유전자를 표적으로 하는 지질 제형된 ｄｓｒｎａ
EP2449106B1 (de) * 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Zusammensetzungen und verfahren zur ausschaltung von apolipoprotein b
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
AP3574A (en) 2009-08-14 2016-02-08 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
WO2011038031A1 (en) 2009-09-22 2011-03-31 Alnylam Pharmaceuticals, Inc. Dual targeting sirna agents
CA2775092A1 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
EP2496238A4 (de) 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc Zusammensetzungen aus lipidformulierungen und verfahren zur hemmung der transthyretinexpression
AU2016250459B2 (en) * 2009-12-01 2018-06-28 Translate Bio, Inc. Delivery Of mRNA For The Augmentation Of Proteins And Enzymes In Human Genetic Diseases
AU2014250713B2 (en) * 2009-12-01 2016-07-28 Translate Bio, Inc. Delivery Of mRNA For The Augmentation Of Proteins And Enzymes In Human Genetic Diseases
RS58405B1 (sr) * 2009-12-01 2019-04-30 Translate Bio Inc Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima
MX348474B (es) 2009-12-23 2017-06-14 Novartis Ag * Lipidos, composiciones de lipido, y metodos de uso de los mismos.
US9670487B2 (en) 2010-01-22 2017-06-06 Sirna Therapeutics, Inc. Cationic lipids for oligonucleotide delivery
KR101852210B1 (ko) 2010-03-24 2018-04-25 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
WO2011120053A1 (en) 2010-03-26 2011-09-29 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
WO2011136368A1 (ja) 2010-04-28 2011-11-03 協和発酵キリン株式会社 カチオン性脂質
JP5902617B2 (ja) 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
JP2013527856A (ja) 2010-05-12 2013-07-04 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 陽イオン性脂質およびその使用方法
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
MX342608B (es) 2010-07-06 2016-10-06 Novartis Ag * Particulas de suministro similares a viriones para moleculas de arn de autorreplicacion.
US10487332B2 (en) 2010-07-06 2019-11-26 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of RNA
US9770463B2 (en) 2010-07-06 2017-09-26 Glaxosmithkline Biologicals Sa Delivery of RNA to different cell types
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
BR112013002738A2 (pt) 2010-08-04 2017-06-27 Cizzle Biotechnology Ltd métodos e compostos para o diagnóstico e o tratamento do câncer
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2609135A4 (de) 2010-08-26 2015-05-20 Massachusetts Inst Technology Poly(beta-amino-alkohole), ihre herstellung und verwendungen davon
RS63367B1 (sr) 2010-08-31 2022-07-29 Glaxosmithkline Biologicals Sa Pegilovani lipozomi za isporuku rnk koja kodira imunogen
RS63984B1 (sr) 2010-08-31 2023-03-31 Glaxosmithkline Biologicals Sa Mali lipozomi za isporuku rnk koja kodira imunogen
ES2727583T3 (es) 2010-08-31 2019-10-17 Glaxosmithkline Biologicals Sa Lípidos adecuados para la administración liposómica de ARN que codifica proteínas
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
LT4108671T (lt) 2010-10-01 2025-01-10 Modernatx, Inc. Modifikuoti nukleozidai, nukleotidai, nukleorūgštys bei jų panaudojimas
ES2716243T3 (es) 2010-10-11 2019-06-11 Glaxosmithkline Biologicals Sa Plataformas de suministro de antígenos
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
EP2691443B1 (de) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Konjugierte lipomers und ihre verwendung
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
CA3198966A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Cleavable lipids
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
SMT201900445T1 (it) 2011-06-08 2019-09-09 Translate Bio Inc Composizione di nanoparticelle lipidiche e metodi per il rilascio di mrna
EP3854413A1 (de) 2011-07-06 2021-07-28 GlaxoSmithKline Biologicals SA Immunogene kombinationszusammensetzungen und ihre verwendung
ES2861428T3 (es) 2011-07-06 2021-10-06 Glaxosmithkline Biologicals Sa Liposomas que presentan una relación N:P útil para suministro de moléculas de ARN
PT2750707T (pt) 2011-08-31 2019-01-23 Glaxosmithkline Biologicals Sa Lipossomas peguilados para distribuição de arn que codifica imunogénio
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
SMT202200229T1 (it) 2011-10-03 2022-07-21 Modernatx Inc Nucleosidi, nucleotidi e acidi nucleici modificati e loro usi
EA032088B1 (ru) 2011-10-27 2019-04-30 Массачусетс Инститьют Оф Текнолоджи Аминокислотные производные, функционализованные на n-конце, способные образовывать микросферы, инкапсулирующие лекарственное средство
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
IN2014CN03463A (de) 2011-11-18 2015-10-09 Alnylam Pharmaceuticals Inc
CA3165769A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
AU2012353463B2 (en) 2011-12-12 2017-08-24 Kyowa Hakko Kirin Co., Ltd. Lipid nanoparticles containing combinations of cationic lipids
WO2013089151A1 (ja) 2011-12-12 2013-06-20 協和発酵キリン株式会社 カチオン性脂質を含有するドラッグデリバリーシステムのための脂質ナノ粒子
RU2649364C2 (ru) 2011-12-16 2018-04-02 Модерна Терапьютикс, Инк. Составы на основе модифицированного нуклеозида, нуклеотида и нуклеиновой кислоты
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
PT2817287T (pt) 2012-02-24 2018-12-28 Arbutus Biopharma Corp Lípidos catiónicos trialquílicos e seus métodos de utilização
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
EP2830596B1 (de) 2012-03-29 2020-12-30 Translate Bio, Inc. Aus lipiden gewonnene neutrale nanopartikel
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
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
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
EP3597741A1 (de) 2012-04-27 2020-01-22 Duke University Genetische korrektur mutierter gene
EP3536787A1 (de) 2012-06-08 2019-09-11 Translate Bio, Inc. Nukleaseresistente polynukleotide und verwendungen davon
CA2876155C (en) 2012-06-08 2022-12-13 Ethris Gmbh Pulmonary delivery of mrna to non-lung target cells
US10689660B2 (en) 2012-06-22 2020-06-23 The Regents Of The University Of California Compositions and methods for mediating plant stomatal development in response to carbon dioxide and applications for engineering drought tolerance in plants
JP6108197B2 (ja) 2012-07-02 2017-04-05 日油株式会社 三級アミノ基含有脂質の製造方法
TW201726600A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
WO2014013995A1 (ja) 2012-07-16 2014-01-23 協和発酵キリン株式会社 KRAS遺伝子発現抑制RNAi医薬組成物
EP2882706A1 (de) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Aminhaltige lipoide und ihre verwendungen
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
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
EP2929035A1 (de) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidnanopartikel zur freisetzung von mrna
DK3327127T3 (da) 2012-12-12 2021-06-28 Broad Inst Inc Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser
JP2016506416A (ja) 2013-01-10 2016-03-03 ノバルティス アーゲー インフルエンザウイルス免疫原性組成物およびその使用
US20160032316A1 (en) 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
CN105209490A (zh) 2013-03-14 2015-12-30 夏尔人类遗传性治疗公司 用于递送mrna编码的抗体的方法和组合物
EP4446413A3 (de) 2013-03-14 2024-12-18 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
WO2014140856A2 (en) 2013-03-15 2014-09-18 Graham Lord Mir-142 and antagonists thereof for treating disease
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP4614148A3 (de) 2013-03-15 2025-12-17 Translate Bio, Inc. Synergistische verbesserung der abgabe von nukleinsäuren über gemischte formulierungen
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
AU2014281030B2 (en) 2013-06-17 2020-07-09 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CA2919226C (en) 2013-07-23 2024-05-14 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
JP6525435B2 (ja) 2013-10-22 2019-06-12 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド メッセンジャーｒｎａの送達のための脂質製剤
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
EP3060303B1 (de) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna-therapie für argininosuccinat-synthase-mangel
WO2015075557A2 (en) 2013-11-22 2015-05-28 Mina Alpha Limited C/ebp alpha compositions and methods of use
CN105960265A (zh) 2013-12-04 2016-09-21 阿克赛医药公司 利用经化学修饰的寡核苷酸处理伤口愈合的方法
AU2014361826A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using particle delivery components
AU2014361784A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
SG10201804974RA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
RS58337B1 (sr) 2014-03-24 2019-03-29 Translate Bio Inc Irnk terapija za lečenje očnih oboljenja
EP3636742B1 (de) 2014-04-25 2025-11-05 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
US20170051290A1 (en) 2014-05-01 2017-02-23 Rxi Pharmaceuticals Corporation Methods for treatment of disorders in the front of the eye utilizing nucleic acid molecules
EP3148552B1 (de) 2014-05-30 2019-07-31 Translate Bio, Inc. Biologisch abbaubare lipide zur freisetzung von nukleinsäuren
EP3153172A4 (de) 2014-06-04 2018-01-17 Kyowa Hakko Kirin Co., Ltd. Ckap5-gen-ausschaltende pharmazeutische zusammensetzung
CA2952121A1 (en) 2014-06-13 2015-12-17 Childrens' Medical Center Corporation Products and methods to isolate mitochondria
SG11201610670WA (en) 2014-06-24 2017-01-27 Shire Human Genetic Therapies Stereochemically enriched compositions for delivery of nucleic acids
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
JP6778175B2 (ja) 2014-07-16 2020-10-28 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
GB201414464D0 (en) 2014-08-14 2014-10-01 Technion Res & Dev Foundation Compositions and methods for therapeutics prescreening
EP3180426B1 (de) 2014-08-17 2019-12-25 The Broad Institute, Inc. Gemonbearbeitung mit cas9-nickasen
PL3185957T3 (pl) 2014-08-29 2022-11-14 Alnylam Pharmaceuticals, Inc. Patisiran do zastosowania w leczeniu amyloidozy związanej z transtyretyną
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
AU2015328012A1 (en) 2014-10-02 2017-05-11 Arbutus Biopharma Corporation Compositions and methods for silencing Hepatitis B virus gene expression
EP3542825A1 (de) 2014-11-10 2019-09-25 Ethris GmbH Induktion von osteogenese durch bereitstellung von bmp-codierender rna
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2016100974A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
AU2015369725A1 (en) 2014-12-24 2017-06-29 Massachusetts Institute Of Technology CRISPR having or associated with destabilization domains
EP3256487A4 (de) 2015-02-09 2018-07-18 Duke University Zusammensetzungen und verfahren zur epigenombearbeitung
JP6895892B2 (ja) 2015-03-19 2021-06-30 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療
WO2016197132A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
FI3430134T3 (fi) 2015-06-18 2023-01-13 Uusia CRISPR-entsyymejä ja järjestelmiä
IL293323B2 (en) 2015-06-18 2024-01-01 Massachusetts Inst Technology Crispr enzyme mutations reducing off-target effects
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA2990172A1 (en) 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
HRP20240865T1 (hr) 2015-06-29 2024-10-11 Acuitas Therapeutics Inc. Lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
KR20180026739A (ko) 2015-07-06 2018-03-13 알엑스아이 파마슈티칼스 코포레이션 슈퍼옥시드 디스뮤타제 1 (sod1)을 표적화하는 핵산 분자
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CN108350455A (zh) 2015-07-29 2018-07-31 阿布特斯生物制药公司 用于使b型肝炎病毒基因表达沉默的组合物和方法
DK3329002T3 (da) 2015-07-31 2021-01-11 Alnylam Pharmaceuticals Inc Transthyretin (ttr)-irna-sammensætninger og fremgangsmåder til anvendelse deraf til behandling eller forebyggelse af ttr-forbundne sygdomme
US9856481B2 (en) 2015-08-13 2018-01-02 Ann & Robert H. Lurie Children's Hospital MicroRNA treatment of fibrosis
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
EP3337908A4 (de) 2015-08-18 2019-01-23 The Broad Institute, Inc. Verfahren und zusammensetzungen zur veränderung der funktion und struktur von chromatinschleifen und/oder -domänen
EP3340967B1 (de) 2015-08-24 2024-05-22 Halo-Bio Rnai Therapeutics, Inc. Polynukleotid-nanopartikel zur modulation der genexpression und verwendungen davon
ES2910425T3 (es) 2015-09-17 2022-05-12 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP4089175A1 (de) 2015-10-13 2022-11-16 Duke University Genom-engineering mit typ-i-crispr-systemen in eukaryotischen zellen
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
JP2018531037A (ja) 2015-10-19 2018-10-25 アールエックスアイ ファーマシューティカルズ コーポレーション 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物
CN109153980B (zh) 2015-10-22 2023-04-14 布罗德研究所有限公司 Vi-b型crispr酶和系统
EP3368687B1 (de) 2015-10-27 2021-09-29 The Broad Institute, Inc. Zusammensetzungen und verfahren gegen krebsspezifische sequenzvariationen
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
WO2017075531A1 (en) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
US11903974B2 (en) 2015-11-30 2024-02-20 Flagship Pioneering Innovations V, Inc. Methods and compositions relating to chondrisomes from cultured cells
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
US20200123499A1 (en) 2015-12-02 2020-04-23 Massachusetts Institute Of Technology Method for efficient generation of neurons from non-neuronal cells
AU2016366978B2 (en) 2015-12-10 2022-07-28 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
CA3007297A1 (en) 2015-12-22 2017-06-29 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
WO2017111172A1 (ja) 2015-12-25 2017-06-29 協和発酵キリン株式会社 カチオン性脂質としての化合物
DK4272834T3 (da) 2016-01-15 2025-12-15 Childrens Medical Ct Corp Terapeutisk anvendelse af mitokondrier og kombinerede mitokondrielle midler
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3445853A1 (de) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1-komplexe mit reduzierter indelaktivität
AU2017253089B2 (en) 2016-04-19 2023-07-20 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
EP3445856A1 (de) 2016-04-19 2019-02-27 The Broad Institute Inc. Neuartige crispr-enzyme und -systeme
EP3842530A1 (de) 2016-06-13 2021-06-30 Translate Bio, Inc. Messenger-rna-therapie zur behandlung von ornithintranscarbamylasemangel
KR20190019168A (ko) 2016-06-17 2019-02-26 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
MX2018016389A (es) 2016-06-30 2019-08-16 Arbutus Biopharma Corp Composiciones y metodos para suministro de arn mensajero.
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
EP3500294A4 (de) 2016-08-22 2020-07-29 Arbutus Biopharma Corporation Anti-pd-1-antikörper oder fragmente davon zur behandlung von hepatitis b
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
DK3532103T3 (da) 2016-10-26 2026-03-02 Acuitas Therapeutics Inc Formuleringer af lipid-nanopartikler
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (de) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilisierte formulierungen von lipidnanopartikeln
US20180153822A1 (en) 2016-11-10 2018-06-07 Translate Bio, Inc. Process of Preparing mRNA-Loaded Lipid Nanoparticles
CA3041350A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Subcutaneous delivery of messenger rna
EP3565891B1 (de) 2017-01-09 2023-04-12 Whitehead Institute for Biomedical Research Verfahren zur veränderung der genexpression durch stören von transkriptionsfaktor-multimeren, die regulatorische schleifen strukturieren
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
US10961184B2 (en) 2017-03-07 2021-03-30 Translate Bio, Inc. Polyanionic delivery of nucleic acids
EP3596042B1 (de) 2017-03-15 2022-01-12 Modernatx, Inc. Kristallformen von aminolipiden
RS63953B1 (sr) 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
US11739308B2 (en) 2017-03-15 2023-08-29 The Broad Institute, Inc. Cas13b orthologues CRISPR enzymes and systems
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
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
WO2018187590A1 (en) 2017-04-05 2018-10-11 Modernatx, Inc. Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
EP3610009A1 (de) 2017-04-12 2020-02-19 The Broad Institute, Inc. Neuartige typ-vi-crispr-orthologe und systeme
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
WO2018200943A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
ES3052812T3 (en) 2017-05-08 2026-01-14 Flagship Pioneering Innovations V Inc Compositions for facilitating membrane fusion and uses thereof
US11173190B2 (en) 2017-05-16 2021-11-16 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3638797A1 (de) 2017-06-13 2020-04-22 Flagship Pioneering Innovations V, Inc. Zusammensetzungen mit curons und verwendungen davon
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (de) 2017-06-15 2021-03-24 Modernatx, Inc. Rna-formulierungen
EP3641834B1 (de) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger-rna-therapie zur behandlung von friedreich-ataxie
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
ES2997124T3 (en) 2017-08-17 2025-02-14 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
US11447773B2 (en) 2017-09-08 2022-09-20 Mina Therapeutics Limited Stabilized HNF4A saRNA compositions and methods of use
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
WO2019060746A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. SYSTEMS, METHODS, AND COMPOSITIONS FOR THE TARGETED EDITING OF NUCLEIC ACIDS
EP3692152B1 (de) 2017-10-04 2025-09-03 The Broad Institute, Inc. Verfahren und zusammensetzungen zur veränderung der funktion und struktur von chromatinschleifen und/oder -domänen
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
EP3710039A4 (de) 2017-11-13 2021-08-04 The Broad Institute, Inc. Verfahren und zusammensetzungen zur behandlung von krebs durch targeting des clec2d-klrb1-wegs
WO2019118806A1 (en) 2017-12-14 2019-06-20 Solid Biosciences Inc. Non-viral production and delivery of genes
MA51306A (fr) 2017-12-20 2020-10-28 Translate Bio Inc Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
EP3740580A4 (de) 2018-01-19 2021-10-20 Duke University Genom-engineering mit crispr-cas-systemen bei eukaryonten
WO2019152802A1 (en) 2018-02-02 2019-08-08 Translate Bio, Inc. Cationic polymers
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
CA3097912A1 (en) 2018-05-15 2019-11-21 Translate Bio, Inc. Subcutaneous delivery of messenger rna
EP3794008A1 (de) 2018-05-16 2021-03-24 Translate Bio, Inc. Kationische ribose
CA3100254A1 (en) 2018-05-24 2019-11-28 Translate Bio, Inc. Thioester cationic lipids
JP2021525725A (ja) 2018-05-30 2021-09-27 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａワクチンおよびその使用
EP3802507A1 (de) 2018-05-30 2021-04-14 Translate Bio, Inc. Kationische vitamin-lipide
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
ES2989886T3 (es) 2018-05-30 2024-11-28 Translate Bio Inc Lípidos catiónicos que comprenden una fracción esteroidal
DK3807413T3 (da) 2018-06-18 2025-08-25 Res Inst Nationwide Childrens Hospital Adenoassocieret virusvektorfremføring af muskelspecifik mikrodystrofin til behandling af muskeldystrofi
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
SG11202100602SA (en) 2018-07-23 2021-02-25 Translate Bio Inc Dry power formulations for messenger rna
US20210163944A1 (en) 2018-08-07 2021-06-03 The Broad Institute, Inc. Novel cas12b enzymes and systems
TWI837161B (zh) 2018-08-10 2024-04-01 日商武田藥品工業股份有限公司 陽離子性脂質
JP7436990B2 (ja) 2018-08-10 2024-02-22 国立大学法人京都大学 カチオン性脂質を用いた心筋細胞へのトランスフェクション方法
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
CN112839639A (zh) 2018-08-29 2021-05-25 川斯勒佰尔公司 制备负载mRNA的脂质纳米颗粒的改进方法
GB2592504B (en) 2018-09-04 2023-02-15 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
JP7425066B2 (ja) 2018-09-04 2024-01-30 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
WO2020056294A1 (en) 2018-09-14 2020-03-19 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020061284A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
JP7410135B2 (ja) 2018-09-19 2024-01-09 モデルナティエックス インコーポレイテッド 治療薬の細胞内送達のための化合物及び組成物
EP3853305B1 (de) 2018-09-19 2024-10-02 ModernaTX, Inc. Hochreine peg-lipide und verwendungen davon
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
CA3113449A1 (en) 2018-09-21 2020-03-26 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
US11980673B2 (en) 2018-10-09 2024-05-14 The University Of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
AU2019361129A1 (en) 2018-10-18 2021-05-20 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US20210378962A1 (en) 2018-10-19 2021-12-09 Translate Bio, Inc. Pumpless encapsulation of messenger rna
WO2020097511A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
WO2020097376A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
EP3876999A4 (de) * 2018-11-09 2022-08-31 Arbutus Biopharma Corporation Negativ geladene peg-lipid-konjugate
WO2020097384A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
EP3876994A2 (de) 2018-11-09 2021-09-15 Translate Bio, Inc. Peg-lipidoid-verbindungen
AU2019378763B2 (en) 2018-11-12 2025-06-26 Translate Bio, Inc. Methods for inducing immune tolerance
WO2020106903A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
AU2020206109B2 (en) 2019-01-07 2024-12-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
JP7523449B2 (ja) 2019-01-11 2024-07-26 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
HUE065017T2 (hu) 2019-02-14 2024-04-28 Ethris Gmbh Poliribonukleotid ciliogenezisre gyakorolt hatásának elemzése
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020206231A1 (en) 2019-04-05 2020-10-08 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
EP3953473A1 (de) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna-zusammensetzungen und verfahren zur verwendung
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
US20220233444A1 (en) 2019-04-22 2022-07-28 Translate Bio, Inc. Thioester cationic lipids
WO2020227085A1 (en) 2019-05-03 2020-11-12 Translate Bio, Inc. Di-thioester cationic lipids
WO2020232276A1 (en) 2019-05-14 2020-11-19 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
IL288284B2 (en) 2019-05-22 2024-06-01 Massachusetts Inst Technology Circular rna compositions and methods
EP3976593A1 (de) 2019-05-31 2022-04-06 Translate Bio, Inc. Makrocyclische lipide
WO2020255014A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Recombinant interleukin 12 construct and uses thereof
EP3986454A1 (de) 2019-06-18 2022-04-27 Janssen Sciences Ireland Unlimited Company Kombination aus hepatitis-b-virus (hbv) -impfstoffen und anti-pd-1- oder anti-pd-l1-antikörper
AU2020295796A1 (en) 2019-06-18 2022-02-10 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai
CN114206379A (zh) 2019-06-18 2022-03-18 爱尔兰詹森科学公司 乙型肝炎病毒(hbv)疫苗和抗pd-1抗体的组合
WO2020255010A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of recombinant interleukin 12 construct and hepatitis b virus (hbv) vaccines
MA56536A (fr) 2019-06-20 2022-04-27 Janssen Sciences Ireland Unlimited Co Administration de nanoparticules lipidiques ou de liposomes de vaccins contre le virus de l'hépatite b (vhb)
CN114401942B (zh) 2019-06-21 2024-01-26 川斯勒佰尔公司 三(羟甲基)甲基甘氨酸和柠檬酸脂质
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
KR20220078557A (ko) 2019-07-08 2022-06-10 트랜슬레이트 바이오 인코포레이티드 개선된 mrna 로딩된 지질 나노입자 및 이의 제조 방법
MX2022000934A (es) 2019-07-23 2022-02-14 Translate Bio Inc Composiciones estables de nanoparticulas lipidicas cargadas de arnm y procesos de fabricacion.
CA3148819A1 (en) 2019-07-30 2021-02-04 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
UA129330C2 (uk) 2019-08-14 2025-03-19 Акітас Терап'Ютікс, Інк. Поліпшені ліпідні наночастинки для доставляння нуклеїнових кислот
CA3154618A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
US20240197825A1 (en) 2019-09-20 2024-06-20 Translate Bio, Inc. mRNA Encoding Engineered CFTR
US12076438B2 (en) 2019-10-09 2024-09-03 Translate Bio, Inc. Compositions, methods and uses of messenger RNA
WO2021081058A1 (en) 2019-10-21 2021-04-29 Translate Bio, Inc. Compositions, methods and uses of messenger rna
US20230002766A1 (en) 2019-11-08 2023-01-05 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
WO2021113777A2 (en) 2019-12-04 2021-06-10 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4076393A2 (de) 2019-12-20 2022-10-26 Translate Bio, Inc. Rektale verabreichung von messenger-rna
WO2021127641A1 (en) 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
EP4085136A1 (de) 2019-12-31 2022-11-09 Phio Pharmaceuticals Corp. Chemisch modifizierte oligonukleotide mit verbesserter systemischer abgabe
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
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
GB2629329B (en) 2020-03-04 2025-02-26 Verve Therapeutics Inc Formulation comprising GalNAc-lipid nanoparticle
EP4121018A4 (de) 2020-03-17 2024-07-03 Genevant Sciences Gmbh Kationische lipide zur lipidnanopartikelabgabe von therapeutika an hepatische stellatzellen
US20230134550A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CA3172572A1 (en) 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
AU2021250814A1 (en) 2020-03-30 2022-10-13 BioNTech SE RNA compositions targeting Claudin-18.2
AU2021269042A1 (en) 2020-05-07 2023-02-02 Translate Bio, Inc. Optimized nucleotide sequences encoding SARS-CoV-2 antigens
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
US20230190954A1 (en) 2020-05-07 2023-06-22 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2021231697A1 (en) 2020-05-14 2021-11-18 Translate Bio, Inc. Peg lipidoid compounds
CN115916158A (zh) 2020-05-15 2023-04-04 翻译生物公司 用于mRNA递送的脂质纳米颗粒配制品
US20240042015A1 (en) 2020-05-19 2024-02-08 Orna Therapeutics, Inc. Circular rna compositions and methods
MX2022016332A (es) 2020-06-15 2023-04-03 Res Inst Nationwide Childrens Hospital Administracion de vectores de virus adenoasociados para distrofias musculares.
WO2025056938A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
US20240307556A1 (en) 2020-07-02 2024-09-19 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
CN116367854A (zh) 2020-07-08 2023-06-30 杨森科学爱尔兰无限公司 针对hbv的rna复制子疫苗
BR112023000327A2 (pt) 2020-07-16 2023-01-31 Acuitas Therapeutics Inc Lipídeos catiônicos para o uso em nanopartículas lipídicas
EP4189098A1 (de) 2020-07-27 2023-06-07 Anjarium Biosciences AG Zusammensetzungen von dna-molekülen, verfahren zu ihrer herstellung und verfahren zu ihrer verwendung
AU2021358777A1 (en) 2020-10-06 2023-06-15 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022076547A1 (en) 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions
WO2022081548A1 (en) 2020-10-12 2022-04-21 Translate Bio, Inc. Improved process of preparing ice-based lipid nanoparticles
KR20230087536A (ko) 2020-10-12 2023-06-16 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 프로세스
JP2023546175A (ja) 2020-10-14 2023-11-01 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド 脂質ナノ粒子製造の方法及びそれに由来する組成物
EP4240326A1 (de) 2020-11-09 2023-09-13 Translate Bio, Inc. Verbesserte zusammensetzungen zur abgabe von codonoptimierter mrna
CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
CN116723829A (zh) 2020-11-25 2023-09-08 翻译生物公司 稳定的液体脂质纳米颗粒配制品
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
WO2022133230A1 (en) 2020-12-18 2022-06-23 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
EP4267740A1 (de) 2020-12-28 2023-11-01 Arcturus Therapeutics, Inc. Auf hbv abzielende transkriptionsaktivator-ähnliche effektornukleasen (talens)
JP2024504614A (ja) 2021-01-14 2024-02-01 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡがコードする抗体を送達する方法および組成物
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022204549A1 (en) 2021-03-25 2022-09-29 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
DK4319803T3 (da) 2021-04-08 2025-12-15 Vaxthera Sas Coronavirusvaccine omfattende et mosaikprotein
AU2022261865A1 (en) 2021-04-19 2023-11-30 Translate Bio, Inc. Improved compositions for delivery of mrna
EP4326860A1 (de) 2021-04-20 2024-02-28 Anjarium Biosciences AG Zusammensetzungen von dna-molekülen zur codierung von amylo-alpha-1,6-glucosidase, 4-alpha-glucanotransferase, verfahren zur herstellung davon und verfahren zur verwendung davon
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
CN117881786A (zh) 2021-04-27 2024-04-12 世代生物公司 表达治疗性抗体的非病毒dna载体及其用途
CA3211623A1 (en) 2021-05-24 2022-12-01 Bo YING Lipid compounds and lipid nanoparticle compositions
US20240271162A1 (en) 2021-06-11 2024-08-15 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
JP2024527559A (ja) 2021-07-01 2024-07-25 インダプタ セラピューティクス インコーポレイテッド 操作されたナチュラルキラー（ｎｋ）細胞及び関連する方法
JP2024527541A (ja) 2021-07-01 2024-07-25 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａの送達のための組成物
EP4377459A2 (de) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der expression von frataxin (fxn)
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
IL310244A (en) 2021-08-03 2024-03-01 Alnylam Pharmaceuticals Inc Transthyretin (TTR) IRNA compositions and methods of using them
JP2024530647A (ja) 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
WO2023015264A1 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
EP4381070A2 (de) 2021-08-04 2024-06-12 Phio Pharmaceuticals Corp. Chemisch modifizierte oligonukleotide
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
US20250134824A1 (en) 2021-08-16 2025-05-01 Glaxosmithkline Biologicals Sa Low-dose lyophilized rna vaccines and methods for preparing and using the same
US20240350410A1 (en) 2021-08-16 2024-10-24 Glaxosmithkline Biologicals Sa Freeze-drying of lipid nanoparticles (lnps) encapsulating rna and formulations thereof
TW202328067A (zh) 2021-09-14 2023-07-16 美商雷納嘉德醫療管理公司 環狀脂質及其使用方法
CN118317944A (zh) 2021-09-14 2024-07-09 雷纳嘉德医疗管理公司 非环状脂质及其使用方法
US20250049729A1 (en) * 2021-10-25 2025-02-13 Duke University Poegma-based lipid nanoparticles
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
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
CN118829625A (zh) 2021-11-16 2024-10-22 赛欧生物医药股份有限公司 新型可电离脂质和脂质纳米颗粒以及它们的使用方法
MX2024006134A (es) 2021-11-22 2024-07-29 Sail Biomedicines Inc Lipidos ionizables novedosos y nanoparticulas de lipido y metodos para usar los mismos.
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
IL313486A (en) 2021-12-16 2024-08-01 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
WO2023122752A1 (en) 2021-12-23 2023-06-29 Renagade Therapeutics Management Inc. Constrained lipids and methods of use thereof
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
IL314324A (en) 2022-01-27 2024-09-01 BioNTech SE Pharmaceutical preparations for administration of herpes simplex virus antigens and related methods
US20250332112A1 (en) 2022-01-31 2025-10-30 Genevant Sciences Gmbh Ionizable cationic lipids for lipid nanoparticles
EP4473097A1 (de) 2022-02-02 2024-12-11 Sana Biotechnology, Inc. Verfahren zur wiederholungsdosierung und verabreichung von lipidpartikeln oder viralen vektoren sowie entsprechende systeme und verwendungen
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
CA3245624A1 (en) 2022-03-25 2023-09-28 Sail Biomedicines, Inc. NEW IONIZABLE LIPIDS AND LIPID NANOPARTICLES AND THEIR METHODS OF USE
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
AU2023251104A1 (en) 2022-04-07 2024-10-17 Renagade Therapeutics Management Inc. Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
WO2023214405A1 (en) 2022-05-01 2023-11-09 Yeda Research And Development Co. Ltd. Reexpression of hnf4a to alleviate cancer-associated cachexia
WO2023215481A1 (en) 2022-05-05 2023-11-09 The Board Of Trustees Of The Leland Stanford Junior University INTERFERING RNA THERAPY FOR PLN-R14del CARDIOMYOPATHY
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
EP4522650A1 (de) 2022-05-13 2025-03-19 BioNTech SE Gegen hiv gerichtete rna-zusammensetzungen
AU2023275780B2 (en) 2022-05-25 2026-03-05 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2023230295A1 (en) 2022-05-25 2023-11-30 BioNTech SE Rna compositions for delivery of monkeypox antigens and related methods
EP4518845A1 (de) 2022-05-30 2025-03-12 BioNTech SE Komplexe zur verabreichung von nukleinsäuren
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
CA3258303A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Compositions of lipid nanoparticles and their uses
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
AU2023299307A1 (en) 2022-06-30 2025-01-09 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
EP4554967A2 (de) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Zusammensetzungen, systeme und verfahren zur gezielten transkriptionsaktivierung
CN117582417A (zh) * 2022-08-09 2024-02-23 湖南健瑞医药科技有限公司 药物-脂质颗粒及其制备方法和应用
CN117582512B (zh) * 2022-08-09 2025-08-26 湖南长星生物医药有限公司 金属-多酚复合物颗粒及其制备方法和应用
CN117582416B (zh) * 2022-08-09 2025-05-27 湖南长星生物医药有限公司 药物-脂质颗粒及其制备方法和应用
JP2025528195A (ja) 2022-08-12 2025-08-26 ライフ エディット セラピューティクス，インコーポレイティド Ｒｎａガイドヌクレアーゼ並びにその活性断片及びバリアント並びに使用方法
KR20250077608A (ko) 2022-08-19 2025-05-30 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통한 b형 간염 바이러스 조절용 조성물, 시스템 및 방법
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
KR20250056256A (ko) 2022-08-31 2025-04-25 세일 바이오메디슨스, 인크. 신규 이온화성 지질 및 지질 나노입자, 그리고 이의 사용 방법
CA3268005A1 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems and methods of T lymphocyte function modulation
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
CA3267942A1 (en) 2022-09-23 2024-03-28 BioNTech SE COMPOSITIONS FOR THE ADMINISTRATION OF HEPATIC STAGE ANTIGENS AND ASSOCIATED METHODS
EP4590331A1 (de) 2022-09-23 2025-07-30 BioNTech SE Zusammensetzungen zur abgabe von plasmodium-csp-antigenen und zugehörige verfahren
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
IL319682A (en) 2022-10-06 2025-05-01 BioNTech SE RNA complexes targeting claudin-18.2
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
EP4615517A1 (de) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Zirkuläre rna-zusammensetzungen
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
EP4622630A1 (de) 2022-11-21 2025-10-01 Translate Bio, Inc. Zusammensetzungen aus trockenpulverformulierungen von messenger-rna und verfahren zur verwendung davon
AU2023406483A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
EP4626402A1 (de) 2022-12-01 2025-10-08 Generation Bio Co. Lipidnanopartikel mit nukleinsäuren und lipidverankerten polymeren
EP4626444A2 (de) 2022-12-01 2025-10-08 Generation Bio Co. Lipidnanopartikel mit nukleinsäuren, ionisierbaren lipiden, sterolen, lipidverankerte polymere und helferlipiden, deren verwendungen
WO2024125597A1 (en) 2022-12-14 2024-06-20 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
EP4633673A1 (de) 2022-12-15 2025-10-22 Sanofi Pasteur Inc. Mrna, die für influenzavirus-ähnliche partikel kodiert
AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
AR131438A1 (es) 2022-12-20 2025-03-19 Sanofi Sa VACUNA DE ARNm DE RINOVIRUS
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024141955A1 (en) 2022-12-28 2024-07-04 BioNTech SE Rna compositions targeting hiv
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
CN120435314A (zh) 2022-12-29 2025-08-05 波普瓦克斯私人有限公司 多靶疫苗和治疗剂
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
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)
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
KR20250169625A (ko) 2023-04-17 2025-12-03 사노피 파스퇴르 인크 재구성 가능한 건조 분말 제형 및 이의 사용 방법
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
WO2024228150A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
EP4705481A2 (de) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Zirkuläre rna-zusammensetzungen und verfahren
WO2025024324A1 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025024335A2 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025024337A1 (en) 2023-07-24 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025026545A1 (en) 2023-08-01 2025-02-06 BioNTech SE Ionizable thioplipids and uses thereof
TW202519509A (zh) 2023-08-01 2025-05-16 德商拜恩技術股份公司 可離子化硫脂質及其用途
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
WO2025027576A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025030097A2 (en) 2023-08-03 2025-02-06 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
WO2025027579A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
CN121488040A (zh) 2023-08-10 2026-02-06 浙江海昶生物医药股份有限公司 Toll样受体激动剂及其应用
CN121752254A (zh) 2023-08-11 2026-03-27 浙江海昶生物医药股份有限公司 一种具有靶向性的脂质组合物及其制备方法
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025057088A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025124711A1 (en) 2023-12-13 2025-06-19 BioNTech SE Glycolipid compositions
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025134062A2 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025134066A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
TW202546224A (zh) 2024-02-12 2025-12-01 美商生命編輯治療學公司 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質
TW202543583A (zh) 2024-03-01 2025-11-16 加拿大商阿奎特斯醫療公司 用於將治療劑包覆於脂質奈米顆粒中之材料及方法
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
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
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
WO2025240356A1 (en) * 2024-05-14 2025-11-20 Recode Therapeutics, Inc. Lipid nanoparticles for hepatic delivery
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2025252759A1 (en) 2024-06-03 2025-12-11 Bio-Sourcing Transient expression by lipid nanoparticle formulations
WO2025262460A1 (en) 2024-06-21 2025-12-26 BioNTech SE Lipid compositions for nucleic acid delivery
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof
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
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
WO2026018213A1 (en) 2024-07-18 2026-01-22 Vaxthera Sas A broadly protective mosaic vaccine against highly pathogenic avian influenza viruses
WO2026020171A2 (en) 2024-07-19 2026-01-22 Aera Therapeutics, Inc. Chimeric antigen receptor constructs
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

Family Cites Families (98)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
EP0032578B1 (de) * 1980-01-16 1984-07-25 Hans Georg Prof. Dr. Weder Verfahren und Dialysiereinrichtung zur Herstellung von Bilayer-Vesikeln und Verwendung der Bilayer-Vesikel
US4598051A (en) * 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4515736A (en) * 1983-05-12 1985-05-07 The Regents Of The University Of California Method for encapsulating materials into liposomes
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5208036A (en) * 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5545412A (en) * 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US5320906A (en) * 1986-12-15 1994-06-14 Vestar, Inc. Delivery vehicles with amphiphile-associated active ingredient
US5703055A (en) * 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
JPH03126211A (ja) 1989-10-12 1991-05-29 Nippon Chemicon Corp 電解コンデンサ用電解液
US5013556A (en) * 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5225212A (en) * 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5279833A (en) * 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US6465188B1 (en) * 1990-06-11 2002-10-15 Gilead Sciences, Inc. Nucleic acid ligand complexes
JP2774417B2 (ja) 1991-08-07 1998-07-09 株式会社ディ・ディ・エス研究所 ペプチド骨格を有する分枝鎖型糖複合体及び微粒子キャリヤー
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
WO1993012240A1 (en) 1991-12-17 1993-06-24 The Regents Of The University Of California Gene therapy for cystic fibrosis transmembrane conductance regulator activity (cftr)
US5858784A (en) * 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
WO1993017125A1 (en) * 1992-02-19 1993-09-02 Baylor College Of Medicine Oligonucleotide modulation of cell growth
WO1993025673A1 (en) 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
JP2854203B2 (ja) 1992-09-03 1999-02-03 株式会社ディ・ディ・エス研究所 リポソームの製造法
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5674908A (en) * 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6075012A (en) * 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US6989434B1 (en) * 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
EP0807116A4 (de) 1994-06-22 1998-01-07 Megabios Corp Kationische amphiphile
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
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
CA2200952C (en) 1994-09-30 2006-04-11 Inex Pharmaceuticals Corp. Novel compositions comprising quaternary ammonium compounds and neutral lipids for the introduction of polyanionic materials into cells
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5627159A (en) * 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
AU5979296A (en) * 1995-06-07 1996-12-30 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5705385A (en) * 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
JPH11507352A (ja) * 1995-06-07 1999-06-29 ジンタ・インコーポレイテッド 新規カルバメート基本カチオン性脂質
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
ES2231819T3 (es) * 1995-06-07 2005-05-16 Inex Pharmaceuticals Corp Particulas de lipido-acido nucleico preparadas a traves de un intermedio complejo de lipido-acido nucleico hidrofobo y uso para transferir genes.
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
CA2223179A1 (en) * 1995-06-07 1996-12-19 Bob Dale Brown Phosphonic acid-based cationic lipids
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
US7422902B1 (en) * 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
EP0869937A4 (de) * 1995-07-21 2004-07-21 Promega Biosciences Inc Amidbasierende kationische lipide
US6339173B1 (en) * 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
US6330349B1 (en) * 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
WO1997019675A2 (en) * 1995-11-30 1997-06-05 Vical Incorporated Complex cationic lipids
US5817856A (en) * 1995-12-11 1998-10-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Radiation-protective phospholipid and method
US6284267B1 (en) * 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
US5877220A (en) * 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
US6034135A (en) * 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
DE69841002D1 (de) * 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
AU7490098A (en) 1997-05-15 1998-12-08 Genzyme Corporation Cationic amphiphile formulations
AU1360499A (en) 1997-10-10 1999-05-03 Pieter R. Cullis Methods for encapsulating nucleic acids in lipid bilayers
US6410328B1 (en) * 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
CA2271582A1 (en) 1998-05-14 1999-11-14 Sean C. Semple Method for administration of therapeutic agents, including antisense, with repeat dosing
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
PT1129064E (pt) * 1998-11-12 2008-01-31 Invitrogen Corp Reagentes de transfecção
US6649780B1 (en) * 1998-12-22 2003-11-18 Valentis, Inc. Cationic lipids
EP1173600A2 (de) 1999-04-20 2002-01-23 The University Of British Columbia Kationische peg-lipide und verfahren zu ihrer verwendung
US6696424B1 (en) * 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
US20010048940A1 (en) * 1999-06-18 2001-12-06 Jennifer D. Tousignant Cationic amphiphile micellar complexes
RU2250911C2 (ru) 1999-07-14 2005-04-27 Элзэ Копэрейшн Липосомальная композиция, нейтральный липополимер и способ увеличения времени циркуляции липосомы
CA2378430A1 (en) * 1999-07-15 2001-01-25 Inex Pharmaceuticals Corp. Methods and apparatus for preparation of lipid vesicles
CA2397016C (en) 2000-01-10 2011-03-29 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of disease
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
AU2002214854A1 (en) 2000-10-25 2002-05-06 Inex Pharmaceuticals Corporation Lipid formulations for target delivery
CA2427068A1 (en) * 2000-10-27 2002-05-02 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
DE60130583T3 (de) * 2000-12-01 2018-03-22 Europäisches Laboratorium für Molekularbiologie Kleine rns moleküle, die rns-interferenz vermitteln
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20040142892A1 (en) * 2001-04-30 2004-07-22 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
US20040063654A1 (en) * 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
US8496961B2 (en) 2002-05-15 2013-07-30 Sutter West Bay Hospital Delivery of nucleic acid-like compounds
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US20040220129A1 (en) 2003-01-16 2004-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for siRNA inhibition of ICAM-1
US7803781B2 (en) * 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US8673285B2 (en) 2003-06-18 2014-03-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Sphingoid polyalkylamine conjugates for vaccination
KR101168440B1 (ko) * 2003-07-16 2012-07-27 프로티바 바이오쎄라퓨틱스, 인코포레이티드 지질 캡슐화된 간섭 ｒｎａ
NZ581166A (en) * 2003-09-15 2011-06-30 Protiva Biotherapeutics Inc Polyethyleneglycol-modified lipid compounds and uses thereof
ATE536418T1 (de) * 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
CA2569645C (en) * 2004-06-07 2014-10-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
AU2005259799A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
JP5883205B2 (ja) * 2004-09-24 2016-03-09 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． ＡｐｏＢのＲＮＡｉ調節およびその使用
AU2005306533B2 (en) 2004-11-17 2012-05-31 Arbutus Biopharma Corporation siRNA silencing of apolipoprotein B
DE102004060863A1 (de) * 2004-12-17 2006-06-22 Dr. Johannes Heidenhain Gmbh Winkelmesseinrichtung
CA2597724A1 (en) * 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
US20060228406A1 (en) * 2005-03-17 2006-10-12 Invitrogen Corporation Transfection reagent for non-adherent suspension cells
EP2281041B1 (de) * 2008-04-15 2014-07-02 Protiva Biotherapeutics Inc. Abdämpfen der csn5 genexpression unter verwendung von interferierender rns
WO2009129387A2 (en) 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
CA3029724A1 (en) 2008-11-10 2010-05-14 Muthiah Manoharan Lipids and compositions for the delivery of therapeutics
US8569256B2 (en) * 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US20120136073A1 (en) * 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same

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US20110060032A1 (en) 2011-03-10
CA2569664A1 (en) 2005-12-22
AU2005252273A1 (en) 2005-12-22
US20060008910A1 (en) 2006-01-12
US9181545B2 (en) 2015-11-10
US20160115477A1 (en) 2016-04-28
EP1766035B1 (de) 2011-12-07
AU2005252273B2 (en) 2011-04-28
US9926560B2 (en) 2018-03-27
WO2005121348A1 (en) 2005-12-22
JP2008501730A (ja) 2008-01-24
EP1766035A4 (de) 2009-04-22
JP4796062B2 (ja) 2011-10-19
US7799565B2 (en) 2010-09-21
US20190071669A1 (en) 2019-03-07
CA2569664C (en) 2013-07-16

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