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

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special 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)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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 (566)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1519714B1 (de) 2002-06-28 2010-10-20 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
SG190613A1 (en) * 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
CN1882693B (zh) * 2003-09-15 2012-08-15 普洛体维生物治疗公司 聚乙二醇修饰的脂质化合物及其应用
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
ATE536418T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
EP1773857A4 (de) * 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc Das immunsystem stimulierende sirna-moleküle und anwendungen davon
GB0418172D0 (en) * 2004-08-13 2004-09-15 Ic Vec Ltd Vector
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
ATE514776T1 (de) * 2004-10-05 2011-07-15 California Inst Of Techn Aptamer-regulierte nukleinsäuren und verwendungen davon
US20060134189A1 (en) * 2004-11-17 2006-06-22 Protiva Biotherapeutics, Inc 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
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
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
WO2007048046A2 (en) * 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. 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.
EA029538B1 (ru) 2006-03-07 2018-04-30 Басф Энзаймс Ллк Полипептид, обладающий активностью альдолазы, полинуклеотид, кодирующий этот полипептид, и способы получения и применения полинуклеотида и полипептида
EP2112227B1 (de) 2006-03-07 2013-05-15 Cargill, Incorporated Aldolasen, Nukleinsäuren zu ihrer Kodierung sowie Verfahren zu ihrer Herstellung und Verwendung
CN101484588B (zh) 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
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
EP2057179A4 (de) * 2006-08-24 2010-11-10 British Columbia Cancer Agency Zusammensetzung und verfahren zur behandlung von myelosuppression
WO2008042973A2 (en) 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
KR100817024B1 (ko) * 2006-11-09 2008-03-26 재단법인 목암생명공학연구소 핵산 또는 약물을 간에 특이적으로 전달하는 복합체 및이를 포함하는 약학적 조성물
WO2008058291A2 (en) * 2006-11-09 2008-05-15 California Institute Of Technology Modular aptamer-regulated ribozymes
KR100825519B1 (ko) * 2007-01-05 2008-04-25 주식회사 바이오폴리메드 키토산 기재 고분자 접합체 및 그 제조방법
MX347887B (es) 2007-04-27 2017-05-16 The Regents Of The Univ Of California At San Diego Sensores de co2 de planta, acidos nucleicos que los codifican y metodos para hacerlos y usarlos.
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
WO2009011855A2 (en) * 2007-07-16 2009-01-22 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
US20120165387A1 (en) 2007-08-28 2012-06-28 Smolke Christina D General composition framework for ligand-controlled RNA regulatory systems
US8367815B2 (en) * 2007-08-28 2013-02-05 California Institute Of Technology Modular polynucleotides for ligand-controlled regulatory systems
US8865667B2 (en) 2007-09-12 2014-10-21 California Institute Of Technology Higher-order cellular information processing devices
CN110577945A (zh) 2007-10-03 2019-12-17 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
US9550827B2 (en) 2007-10-19 2017-01-24 The Regents Of The University Of California Methods for ameliorating and preventing central nervous system inflammation
CN101909581B (zh) * 2007-11-14 2012-11-14 加利福尼亚大学董事会 固醇修饰的两亲脂质
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
JP5697988B2 (ja) * 2007-12-27 2015-04-08 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 干渉rnaを使用したポロ様キナーゼ発現のサイレンシング方法
US20110097720A1 (en) * 2008-01-02 2011-04-28 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
JP5749494B2 (ja) * 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション 核酸の送達のための改善された組成物および方法
US8541220B2 (en) 2008-01-03 2013-09-24 Verenium Corporation Isomerases, nucleic acids encoding them and methods for making and using them
EP2238242B9 (de) 2008-01-03 2015-06-10 BASF Enzymes LLC Transferasen und oxidoreduktasen, dafür kodierende nukleinsäuren sowie verfahren zu ihrer herstellung und verwendung
BRPI0907008A2 (pt) * 2008-01-31 2015-07-07 Alnylam Pharmaceuticals Inc Métodos otimizados para liberação de dsrna alvejando o gene pcsk9
AU2009221775B2 (en) * 2008-03-05 2015-05-07 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
AU2009236219B8 (en) 2008-04-15 2015-06-25 Arbutus Biopharma Corporation Silencing of CSN5 gene expression using interfering RNA
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
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
EP2326331A4 (de) * 2008-08-18 2013-05-15 Merck Sharp & Dohme Neue lipidnanopartikel und neue komponenten zur freisetzung von nukleinsäuren
WO2010030730A1 (en) * 2008-09-10 2010-03-18 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
WO2010030739A1 (en) * 2008-09-10 2010-03-18 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
EP2340309A2 (de) 2008-09-22 2011-07-06 Rxi Pharmaceuticals Corporation Neutrale nanotransporter
WO2010036962A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
AU2009307677C1 (en) 2008-10-20 2022-09-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
MX2011004859A (es) 2008-11-07 2011-08-03 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
EP4241767B1 (de) * 2008-11-10 2024-10-23 Arbutus Biopharma Corporation Neuartige lipide und zusammensetzungen zur verabreichung von therapeutika
JP5600299B2 (ja) * 2008-11-26 2014-10-01 中外製薬株式会社 ベシクル製剤
EP2610343A3 (de) 2008-11-26 2013-09-25 Chugai Seiyaku Kabushiki Kaisha Verfahren zum entwurf von sirna und oligoribonukleotide zur hemmung der aktivität des hepatitis-c-virus
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
WO2010090762A1 (en) 2009-02-04 2010-08-12 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
AU2010223967B2 (en) * 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
US9145555B2 (en) 2009-04-02 2015-09-29 California Institute Of Technology Integrated—ligand-responsive microRNAs
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
AU2010260148A1 (en) * 2009-06-15 2012-02-02 Alnylam Pharmaceuticals, Inc. Lipid formulated dsRNA targeting the PCSK9 gene
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8236943B2 (en) * 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
EP2464336A4 (de) 2009-08-14 2013-11-20 Alnylam Pharmaceuticals Inc Zusammensetzungen aus lipidformulierungen und verfahren zur hemmung der expression eines gens aus dem ebolavirus
US9187746B2 (en) 2009-09-22 2015-11-17 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
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
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
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
AU2010334911A1 (en) 2009-12-23 2012-07-12 Novartis Ag Lipids, lipid compositions, and methods of using them
EP2525781A1 (de) 2010-01-22 2012-11-28 Schering Corporation Neue kationische lipide zur abgabe von oligonukleotiden
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
EP2553019A1 (de) 2010-03-26 2013-02-06 Mersana Therapeutics, Inc. Modifizierte polymere zur freisetzung von polynukleotiden, verfahren zu ihrer herstellung und verfahren zu ihrer verwendung
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
US9408914B2 (en) 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation 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
SI2591114T1 (sl) 2010-07-06 2016-10-28 Glaxosmithkline Biologicals S.A. Imunizacija velikih sesalcev z majhnimi odmerki RNA
US11291635B2 (en) 2010-07-06 2022-04-05 Glaxosmithkline Biological Sa Virion-like delivery particles for self-replicating RNA molecules
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
US9770463B2 (en) 2010-07-06 2017-09-26 Glaxosmithkline Biologicals Sa Delivery of RNA to different cell types
AU2011276234B2 (en) 2010-07-06 2016-02-25 Glaxosmithkline Biologicals S.A. Liposomes with lipids having an advantageous pKa- value for RNA delivery
EP2601212A1 (de) 2010-08-04 2013-06-12 Cizzle Biotechnology Limited Verfahren und verbindungen zur diagnose und behandlung von
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
TR201908635T4 (tr) 2010-08-31 2019-07-22 Glaxosmithkline Biologicals Sa Protein kodlayıcı rna?nın lipozomal verilmesine uygun lipitler.
BR112013004879A2 (pt) 2010-08-31 2018-04-24 Novartis Ag lipossomas pequenos para entrega de rna que codifica imunogénio
WO2012031043A1 (en) 2010-08-31 2012-03-08 Novartis Ag Pegylated liposomes for delivery of immunogen-encoding rna
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
ES3005233T3 (en) 2010-10-01 2025-03-14 Modernatx Inc Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
EP3520813B1 (de) 2010-10-11 2023-04-19 GlaxoSmithKline Biologicals S.A. Antigenfreisetzungsplattformen
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
DK3336082T3 (da) 2011-06-08 2020-04-27 Translate Bio Inc Spaltelige lipider
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
EP3854413A1 (de) 2011-07-06 2021-07-28 GlaxoSmithKline Biologicals SA Immunogene kombinationszusammensetzungen und ihre verwendung
EP4014966A1 (de) 2011-07-06 2022-06-22 GlaxoSmithKline Biologicals S.A. Liposomen mit nützlichem n:p-verhältnis zur freisetzung von rna-molekülen
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
SG11201400250UA (en) 2011-08-31 2014-03-28 Novartis Ag Pegylated liposomes for delivery of immunogen-encoding rna
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (de) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
KR20150000461A (ko) 2011-10-27 2015-01-02 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, n-말단 상에 관능화된 아미노산 유도체
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
ES3065854T3 (en) 2011-12-07 2026-05-08 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
JP6182457B2 (ja) 2011-12-12 2017-08-16 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クデリバリーシステムのための脂質ナノ粒子
AU2012353463B2 (en) 2011-12-12 2017-08-24 Kyowa Hakko Kirin Co., Ltd. Lipid nanoparticles containing combinations of cationic lipids
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
DK2817287T3 (da) 2012-02-24 2019-01-02 Arbutus Biopharma Corp Trialkyl kationisk lipid og metoder til anvendelse deraf
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
EP3865123A1 (de) 2012-03-29 2021-08-18 Translate Bio, Inc. Aus lipiden gewonnene neutrale nanopartikel
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2013185069A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013192545A1 (en) 2012-06-22 2013-12-27 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
TW201726599A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
US9913907B2 (en) 2012-07-16 2018-03-13 Kyowa Hakko Kirin Co., Ltd. RNAi pharmaceutical composition for suppressing expression of KRAS gene
EP2882706A1 (de) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Aminhaltige lipoide und ihre verwendungen
ES2921623T3 (es) 2012-11-26 2022-08-30 Modernatx Inc ARN modificado terminalmente
KR102096014B1 (ko) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA 조성물 및 그 사용 방법
EP2929035A1 (de) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidnanopartikel zur freisetzung von mrna
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014108515A1 (en) 2013-01-10 2014-07-17 Novartis Ag Influenza virus immunogenic compositions and uses thereof
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
EP2968586B1 (de) 2013-03-14 2018-07-25 Translate Bio, Inc. Cftr-mrna-zusammensetzungen und zugehörige verfahren sowie verwendungen
ES2882110T3 (es) 2013-03-14 2021-12-01 Translate Bio Inc Métodos y composiciones para administrar anticuerpos codificados con ARNm
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
EP4614148A3 (de) 2013-03-15 2025-12-17 Translate Bio, Inc. Synergistische verbesserung der abgabe von nukleinsäuren über gemischte formulierungen
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
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
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
WO2015061467A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
CA3236835A1 (en) 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
CA2932753A1 (en) 2013-12-04 2015-06-11 Rxi Pharmaceuticals Corporation Methods for treatment of wound healing utilizing chemically modified oligonucleotides
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
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
RU2016128077A (ru) 2013-12-12 2018-12-06 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
BR112016013213A2 (pt) 2013-12-12 2017-12-05 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas
BR112016013547A2 (pt) 2013-12-12 2017-10-03 Broad Inst Inc Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos
PT3122878T (pt) 2014-03-24 2019-02-01 Translate Bio Inc Terapia de arnm para o tratamento de doenças oculares
JP6571679B2 (ja) 2014-04-25 2019-09-04 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の精製方法
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
WO2015168605A1 (en) 2014-05-01 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treatment of disorders in the front of the eye utilizing nucleic acid molecules
US10022455B2 (en) 2014-05-30 2018-07-17 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
WO2015186770A1 (ja) 2014-06-04 2015-12-10 協和発酵キリン株式会社 CKAP5遺伝子発現抑制RNAi医薬組成物
EP3154694B1 (de) 2014-06-13 2025-01-29 Children's Medical Center Corporation Produkte und verfahren zur isolierung von mitochondrien
CA2952824C (en) 2014-06-24 2023-02-21 Shire Human Genetic Therapies, Inc. Stereochemically enriched compositions for delivery of nucleic acids
IL316479A (en) 2014-06-25 2024-12-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
CA2953265C (en) 2014-07-02 2023-09-26 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
EP3169309B1 (de) 2014-07-16 2023-05-10 Novartis AG Verfahren zur verkapselung einer nukleinsäure in einem lipidnanopartikelhost
GB201414464D0 (en) 2014-08-14 2014-10-01 Technion Res & Dev Foundation Compositions and methods for therapeutics prescreening
DK3180426T3 (da) 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
EP4137138A1 (de) 2014-08-29 2023-02-22 Alnylam Pharmaceuticals, Inc. Verfahren zum behandeln von transthyretin (ttr)-vermittelter amyloidose
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
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
KR20170075742A (ko) 2014-10-02 2017-07-03 프로티바 바이오쎄라퓨틱스, 인코포레이티드 B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법
EP3542825A1 (de) 2014-11-10 2019-09-25 Ethris GmbH Induktion von osteogenese durch bereitstellung von bmp-codierender rna
MA56412A (fr) 2014-12-05 2022-05-04 Translate Bio Inc Thérapie par l'arn messager pour le traitement des maladies articulaires
EP3230451B1 (de) 2014-12-12 2021-04-07 The Broad Institute, Inc. Geschützte guide-rnas (pgrnas)
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)
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
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
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
IL293323B2 (en) 2015-06-18 2024-01-01 Massachusetts Inst Technology Crispr enzyme mutations reducing off-target effects
EP3436575A1 (de) 2015-06-18 2019-02-06 The Broad Institute Inc. Neuartige crispr-enzyme und -systeme
ES2949540T3 (es) 2015-06-19 2023-09-29 Massachusetts Inst Technology 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
US11001845B2 (en) 2015-07-06 2021-05-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
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
HK1248278A1 (zh) 2015-07-29 2018-10-12 Arbutus Biopharma Corporation 用於使乙型肝炎病毒基因表达沉默的组合物及方法
JP6896703B2 (ja) 2015-07-31 2021-06-30 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. TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
US9856481B2 (en) 2015-08-13 2018-01-02 Ann & Robert H. Lurie Children's Hospital MicroRNA treatment of fibrosis
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
RS63030B1 (sr) 2015-09-17 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava
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
CN108473969B9 (zh) 2015-10-14 2024-08-23 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
CN109563509B (zh) 2015-10-19 2022-08-09 菲奥医药公司 靶向长非编码rna的减小尺寸的自递送核酸化合物
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
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
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
EP3368689B1 (de) 2015-10-28 2020-06-17 The Broad Institute, Inc. Zusammensetzungen zur modulierung von immunantworten unter verwendung von immunzellgensignaturen
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3383411B1 (de) 2015-11-30 2025-07-09 Sana Biotechnology, Inc. Sub-mitochondrielle partikel zur verwendung bei der behandlung metabolischer erkrankungen
WO2017096326A1 (en) 2015-12-02 2017-06-08 Massachusetts Institute Of Technology Method for efficient generation of neurons from non-neuronal cells
CA3007955A1 (en) 2015-12-10 2017-06-15 Modernatx, Inc. Lipid nanoparticles for delivery of therapeutic agents
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
JP7114465B2 (ja) 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
TW201726614A (zh) 2015-12-25 2017-08-01 Kyowa Hakko Kirin Co Ltd 作為陽離子性脂質之化合物
ES3057779T3 (en) 2016-01-15 2026-03-04 Childrens Medical Ct Corp Therapeutic use of mitochondria and combined mitochondrial agents
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
EP3443081A4 (de) 2016-04-13 2019-10-30 Duke University Crispr/cas9-basierte repressoren zur in-vivo-ausschaltung von gen-targets und verfahren zur verwendung
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
SG11201810179RA (en) 2016-04-19 2018-12-28 Broad Inst Inc Novel crispr enzymes and systems
AU2017253107B2 (en) 2016-04-19 2023-07-20 Massachusetts Institute Of Technology CPF1 complexes with reduced indel activity
CN109312313A (zh) 2016-06-13 2019-02-05 川斯勒佰尔公司 用于治疗鸟氨酸转氨甲酰酶缺乏症的信使rna疗法
US11788083B2 (en) 2016-06-17 2023-10-17 The Broad Institute, Inc. Type VI CRISPR orthologs and systems
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
AU2017286980B2 (en) 2016-06-30 2023-10-26 Arbutus Biopharma Corporation Compositions and methods for delivering messenger RNA
EP4275747A3 (de) 2016-07-19 2024-01-24 Duke University Therapeutische anwendungen von cpf1-basierter genomeditierung
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
MA45999A (fr) 2016-08-22 2019-06-26 Arbutus Biopharma Corp Anticorps anti-pd-1, ou leurs fragments, pour le traitement de l'hépatite b
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
WO2018064208A1 (en) 2016-09-28 2018-04-05 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
EP4714454A2 (de) 2016-10-26 2026-03-25 Acuitas Therapeutics Inc. Lipidnanopartikelformulierungen
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
MA46762A (fr) 2016-11-10 2019-09-18 Translate Bio Inc Procédé amélioré de préparation de nanoparticules lipidiques chargées d'arnm
JP7560946B2 (ja) 2016-11-10 2024-10-03 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の皮下送達
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
WO2018165257A1 (en) 2017-03-07 2018-09-13 Translate Bio, Inc. Polyanionic delivery of nucleic acids
KR20190132405A (ko) 2017-03-15 2019-11-27 모더나티엑스, 인크. 치료제의 세포내 전달을 위한 화합물 및 조성물
CA3056133A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
ES3020935T3 (en) 2017-03-15 2025-05-23 Modernatx Inc Lipid nanoparticle formulation
EP3596207B1 (de) 2017-03-15 2023-12-20 The Broad Institute, Inc. Neuartige cas13b-orthologe-crispr-enzyme und systeme
MA47800A (fr) 2017-03-17 2020-01-22 Univ Newcastle Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
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
WO2018191657A1 (en) 2017-04-13 2018-10-18 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
JP7167059B2 (ja) 2017-04-19 2022-11-08 フィオ ファーマシューティカルズ コーポレーション 核酸化合物の局所送達
ES2981244T3 (es) 2017-04-28 2024-10-07 Acuitas Therapeutics Inc Novedosas formulaciones de nanopartículas de lípidos de carbonilo y lípidos para la administración de ácidos nucleicos
WO2018204777A2 (en) 2017-05-05 2018-11-08 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
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
IL271275B1 (en) 2017-06-13 2026-04-01 Flagship Pioneering Innovations V Inc Preparations containing croons and their uses
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
EP3641834B1 (de) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger-rna-therapie zur behandlung von friedreich-ataxie
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
EP4501322A3 (de) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipide zur verwendung in lipidnanopartikelformulierungen
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
EP4219715A3 (de) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilisierte cebpa-sarna-zusammensetzungen und verfahren zur verwendung
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
KR20200089656A (ko) 2017-09-19 2020-07-27 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
WO2019089803A1 (en) 2017-10-31 2019-05-09 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
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
WO2019152802A1 (en) 2018-02-02 2019-08-08 Translate Bio, Inc. Cationic polymers
EP4242307A3 (de) 2018-04-12 2023-12-27 MiNA Therapeutics Limited Sirt1-sarna kompositionen und methoden zu deren verwendung
AU2019271132B2 (en) 2018-05-15 2026-02-05 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
US11964051B2 (en) 2018-05-16 2024-04-23 Translate Bio, Inc. Ribose cationic lipids
AU2019275068B2 (en) 2018-05-24 2024-09-26 Translate Bio, Inc. Thioester cationic lipids
AU2019277361B2 (en) 2018-05-30 2025-04-24 Translate Bio, Inc. Messenger RNA vaccines and uses thereof
WO2019232095A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Vitamin cationic lipids
US11547666B2 (en) 2018-05-30 2023-01-10 Translate Bio, Inc. Cationic lipids comprising a steroidal moiety
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
BR112020025995A2 (pt) 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
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
KR20210056331A (ko) 2018-07-23 2021-05-18 트랜슬레이트 바이오 인코포레이티드 전령 rna의 건조 분말 제형
WO2020033601A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
FI3835287T3 (fi) 2018-08-10 2024-12-03 Takeda Pharmaceuticals Co Kationinen lipidi
EP3842538A4 (de) 2018-08-10 2022-06-08 Kyoto University Verfahren zur transfektion in kardiomyozyten unter verwendung von kationischem lipid
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
KR20210091120A (ko) 2018-08-29 2021-07-21 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 공정
EP3846822A4 (de) 2018-09-04 2022-07-06 The Board Of Regents Of The University Of Texas System Zusammensetzungen und verfahren zur organspezifischen abgabe von nukleinsäuren
GB2592504B (en) 2018-09-04 2023-02-15 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
WO2020051541A1 (en) 2018-09-07 2020-03-12 Duke University Nanoparticulate drug delivery systems
US12564645B2 (en) 2018-09-14 2026-03-03 Translate Bio, Inc. Methods for treatment of methylmalonic acidemia
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US12151029B2 (en) 2018-09-19 2024-11-26 Modernatx, Inc. PEG lipids and uses thereof
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
US12583816B2 (en) 2018-10-18 2026-03-24 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
KR20210090634A (ko) 2018-10-19 2021-07-20 트랜슬레이트 바이오 인코포레이티드 전령 rna의 무펌프 캡슐화
WO2020097493A1 (en) * 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
AU2019374871B2 (en) 2018-11-09 2025-09-11 Translate Bio, Inc. PEG lipidoid compounds
KR102860605B1 (ko) 2018-11-09 2025-09-15 트랜슬레이트 바이오 인코포레이티드 에스테르, 티오에스테르, 이황화물, 및 무수물 모이어티가 삽입된 2,5-디옥소피페라진
EP3876914A2 (de) 2018-11-09 2021-09-15 Translate Bio, Inc. Messenger-rna-therapie zur behandlung von augenerkrankungen
WO2020097376A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
AU2019378763B2 (en) 2018-11-12 2025-06-26 Translate Bio, Inc. Methods for inducing immune tolerance
EP3883592A1 (de) 2018-11-21 2021-09-29 Translate Bio, Inc. Behandlung von zystischer fibrose durch verabreichung von vernebelter mrna, die cftr codiert
EP3883917B1 (de) 2018-11-21 2024-01-24 Translate Bio, Inc. Kationische lipidverbindungen und deren zusammensetzungen zur verwendung bei der verabreichung von messenger-rna
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
CN113412273B (zh) 2019-01-07 2025-08-22 川斯勒佰尔公司 用于治疗原发性纤毛运动障碍的组合物和方法
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
BR112021014528A2 (pt) 2019-02-14 2021-10-13 Ethris Gmbh Composição farmacêutica, e, método para analisar o efeito de um polirribonucleotídeo na ciliogênese
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
US20220204994A1 (en) 2019-04-05 2022-06-30 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
WO2020208361A1 (en) 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
ES2972014T3 (es) 2019-04-22 2024-06-10 Translate Bio Inc Lípidos catiónicos de tioéster
US12453759B2 (en) 2019-05-03 2025-10-28 Translate Bio, Inc. Di-thioester cationic lipids
EP3968952A1 (de) 2019-05-14 2022-03-23 Translate Bio, Inc. Verbessertes verfahren zur herstellung von mrna-beladenen lipidnanopartikeln
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
JP7686574B2 (ja) 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状rna組成物及び方法
WO2020243540A1 (en) 2019-05-31 2020-12-03 Translate Bio, Inc. Macrocyclic lipids
KR20220041080A (ko) 2019-06-18 2022-03-31 얀센 사이언시즈 아일랜드 언리미티드 컴퍼니 B형 간염 바이러스(hbv) 백신 및 항-pd-1 또는 항-pc-l1 항체의 조합
US20220305117A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and hbv-targeting rnai
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
AU2020296372A1 (en) 2019-06-18 2022-02-17 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and anti-PD-1 antibody
MA56520A (fr) 2019-06-18 2022-04-27 Janssen Sciences Ireland Unlimited Co Construction de l'interleukine 12 recombinante et ses utilisations
AU2020295807A1 (en) 2019-06-20 2022-02-17 Janssen Sciences Ireland Unlimited Company Lipid nanoparticle or liposome delivery of hepatitis B virus (HBV) vaccines
AU2020297606B2 (en) 2019-06-21 2026-04-02 Translate Bio, Inc. Tricine and citric acid lipids
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
ES2992127T3 (es) 2019-07-08 2024-12-09 Translate Bio Inc Nanopartículas lipídicas cargadas de ARNm mejoradas y sus procesos de fabricación
WO2021016430A1 (en) 2019-07-23 2021-01-28 Translate Bio, Inc. Stable compositions of mrna-loaded lipid nanoparticles and processes of making
EP4003397B1 (de) 2019-07-30 2025-02-26 Translate Bio, Inc. Behandlung von zystischer fibrose durch verabreichung von vernebelter mrna, die cftr codiert
ES2918001B2 (es) 2019-08-14 2026-05-13 Acuitas Therapeutics Inc Nanoparticulas lipidicas mejoradas para el suministro de acidos nucleicos
MX2022003269A (es) 2019-09-19 2022-07-04 Modernatx Inc Compuestos lipidicos de cola ramificada y composiciones para la administracion intracelular de agentes terapeuticos.
CA3155015A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
JP7650873B2 (ja) 2019-10-09 2025-03-25 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の組成物、方法および使用
US20210145860A1 (en) 2019-10-21 2021-05-20 Translate Bio, Inc. Compositions, methods and uses of messenger rna
CA3160657A1 (en) 2019-11-08 2021-05-14 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
KR20220142432A (ko) 2019-12-20 2022-10-21 트랜슬레이트 바이오 인코포레이티드 메신저 rna의 직장 전달
US12053551B2 (en) 2019-12-20 2024-08-06 Translate Bio, Inc. Process of preparing mRNA-loaded lipid nanoparticles
WO2021138537A1 (en) 2019-12-31 2021-07-08 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides with improved systemic delivery
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
WO2021173840A1 (en) 2020-02-25 2021-09-02 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
GB2627644B (en) 2020-03-04 2024-11-20 Verve Therapeutics Inc Method for the preparation of GalNAc-lipid nanoparticle
EP4121018A4 (de) 2020-03-17 2024-07-03 Genevant Sciences Gmbh Kationische lipide zur lipidnanopartikelabgabe von therapeutika an hepatische stellatzellen
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021195218A1 (en) 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
IL296781A (en) 2020-03-30 2022-11-01 BioNTech SE Rna compositions targeting claudin-18.2
US12605399B2 (en) 2020-05-07 2026-04-21 Translate Bio, Inc. Compositions for CFTR MRNA therapy
EP4146680A1 (de) 2020-05-07 2023-03-15 Translate Bio, Inc. Zusammensetzung und verfahren zur behandlung von primärer ziliardyskinesie
WO2021226436A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Optimized nucleotide sequences encoding sars-cov-2 antigens
US12514932B2 (en) 2020-05-14 2026-01-06 Translate Bio, Inc. PEG lipidoid compounds
EP4149425A1 (de) 2020-05-15 2023-03-22 Translate Bio, Inc. Lipidnanopartikelformulierungen zur mrna-abgabe
US20240042015A1 (en) 2020-05-19 2024-02-08 Orna Therapeutics, Inc. Circular rna compositions and methods
BR112022025586A2 (pt) 2020-06-15 2023-03-07 Res Inst Nationwide Childrens Hospital Liberação de vetor de vírus adenoassociado 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
CA3188801A1 (en) 2020-07-08 2022-01-13 Janssen Sciences Ireland Unlimited Company Rna replicon vaccines against hbv
JP7798855B2 (ja) 2020-07-16 2026-01-14 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子に使用するためのカチオン性脂質
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
WO2022076562A1 (en) 2020-10-06 2022-04-14 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022076547A1 (en) 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions
US20220133631A1 (en) 2020-10-12 2022-05-05 Translate Bio, Inc. Process of preparing ice-based lipid nanoparticles
US20220110884A1 (en) 2020-10-12 2022-04-14 Translate Bio, Inc. Process of preparing mrna-loaded lipid nanoparticles
KR20230118715A (ko) 2020-10-14 2023-08-11 조지 메이슨 리서치 파운데이션 인코포레이티드 이온화 가능 지질 및 이의 제조 및 사용 방법
AU2021373892A1 (en) 2020-11-09 2023-07-06 Translate Bio, Inc. Improved compositions for delivery of codon-optimized mrna
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
CA3200234A1 (en) 2020-11-25 2022-06-02 Daryl C. Drummond Lipid nanoparticles for delivery of nucleic acids, and related methods of use
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2022133230A1 (en) 2020-12-18 2022-06-23 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
CN117479963A (zh) 2020-12-18 2024-01-30 盖纳万科学有限公司 Peg脂质和脂质纳米粒子
WO2022146654A1 (en) 2020-12-28 2022-07-07 Janssen Pharmaceuticals, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
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
CA3214137A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
WO2022215036A1 (en) 2021-04-08 2022-10-13 Vaxthera Sas Coronavirus vaccine comprising a mosaic protein
WO2022225918A1 (en) 2021-04-19 2022-10-27 Translate Bio, Inc. Improved compositions for delivery of mrna
US20240358852A1 (en) 2021-04-20 2024-10-31 Anjarium Biosciences Ag Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
WO2022232289A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (de) 2021-04-27 2024-03-06 Generation Bio Co. Nichtvirale dna-vektoren zur expression von anti-coronavirus-antikörpern und verwendungen davon
WO2022247755A1 (en) 2021-05-24 2022-12-01 Suzhou Abogen Biosciences Co., Ltd. Lipid compounds and lipid nanoparticle compositions
AU2022290278A1 (en) 2021-06-11 2024-01-04 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
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
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
EP4377459A2 (de) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der expression von frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
TW202328445A (zh) 2021-08-03 2023-07-16 美商艾拉倫製藥股份有限公司 甲狀腺素運載蛋白(TTR)iRNA組成物及其使用方法
EP4380576A4 (de) 2021-08-03 2026-04-29 Verve Therapeutics Inc Zusammensetzungen und verfahren zur gezielten rna-abgabe
WO2023015265A2 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides
WO2023015264A1 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
EP4387597A1 (de) 2021-08-16 2024-06-26 GlaxoSmithKline Biologicals SA Gefriertrocknung von lipidnanopartikeln (lnps) zur verkapselung von rna und formulierungen davon
WO2023021421A1 (en) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Low-dose lyophilized rna vaccines and methods for preparing and using the same
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
EP4402121A1 (de) 2021-09-14 2024-07-24 Renagade Therapeutics Management Inc. Acyclische lipide und verfahren zur verwendung davon
CA3236153A1 (en) 2021-10-25 2023-05-04 Duke University Poegma-based lipid nanoparticles
EP4426832A1 (de) 2021-11-03 2024-09-11 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Genaue genomeditierung mit retrons
CN118541346A (zh) 2021-11-08 2024-08-23 奥纳治疗公司 用于递送环状多核苷酸的脂质纳米颗粒组合物
AU2022388747A1 (en) 2021-11-10 2024-06-20 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
JP2024542188A (ja) 2021-11-16 2024-11-13 セイル バイオメディシンズ インコーポレイテッド 新規イオン化可能な脂質および脂質ナノ粒子ならびにそれらを使用する方法
KR20240128683A (ko) 2021-11-22 2024-08-26 세일 바이오메디슨스, 인크. 신규한 이온화 가능 지질 및 지질 나노입자, 및 이를 사용하는 방법
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
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CA3243054A1 (en) 2021-12-23 2025-04-08 Renagade Therapeutics Management Inc. Constrained Lipids and Associated Utilization Processes
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
EP4469063A1 (de) 2022-01-27 2024-12-04 BioNTech SE Pharmazeutische zusammensetzungen zur abgabe von herpes-simplex-virus-antigenen und zugehörige verfahren
CN118922401A (zh) 2022-01-31 2024-11-08 盖纳万科学有限公司 用于脂质纳米颗粒的可电离阳离子脂质
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
AU2023235112A1 (en) 2022-03-14 2024-10-17 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
EP4499607A1 (de) 2022-03-25 2025-02-05 Sail Biomedicines, Inc. Neue ionisierbare lipide und lipidnanopartikel und verfahren zur verwendung davon
CA3255225A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California COMPOSITIONS AND METHODS OF GENETIC COMPLEMENTATION
WO2023196931A1 (en) 2022-04-07 2023-10-12 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
EP4519438A1 (de) 2022-05-05 2025-03-12 The Board of Trustees of the Leland Stanford Junior University Interferierende rna-therapie für pln-r14del-kardiomyopathie
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
WO2023218431A1 (en) 2022-05-13 2023-11-16 BioNTech SE Rna compositions targeting hiv
EP4531819A2 (de) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipidnanopartikel zur abgabe von nukleinsäuren und verfahren zur verwendung davon
WO2023230295A1 (en) 2022-05-25 2023-11-30 BioNTech SE Rna compositions for delivery of monkeypox antigens and related methods
CN119562807A (zh) 2022-05-30 2025-03-04 生物技术公司 用于递送核酸的复合物
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
AU2023283348A1 (en) 2022-06-07 2025-01-09 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN120051297A (zh) 2022-06-30 2025-05-27 因达普塔治疗公司 工程化自然杀伤(nk)细胞与抗体疗法的组合及相关方法
EP4554967A2 (de) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Zusammensetzungen, systeme und verfahren zur gezielten transkriptionsaktivierung
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
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
MA71732A (fr) 2022-08-12 2025-05-30 Life Edit Therapeutics, Inc. Nucléases guidées par arn et fragments actifs, variants associés et procédés d'utilisation
CN120435557A (zh) 2022-08-19 2025-08-05 图恩疗法股份有限公司 通过靶向基因阻遏调节乙型肝炎病毒的组合物、系统和方法
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
EP4581012A2 (de) 2022-08-31 2025-07-09 Sail Biomedicines, Inc. Neue ionisierbare lipide und lipidnanopartikel und verfahren zur verwendung davon
WO2024064642A2 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
AU2023347390A1 (en) 2022-09-23 2025-04-10 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
CN120225219A (zh) 2022-09-23 2025-06-27 生物技术欧洲股份公司 用于递送肝期抗原的组合物及相关方法
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
AU2023357320A1 (en) 2022-10-06 2025-04-17 BioNTech SE Rna compositions targeting claudin-18.2
TW202425959A (zh) 2022-11-08 2024-07-01 美商歐納醫療公司 遞送多核苷酸的脂質及奈米顆粒組合物
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid 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
IL320873A (en) 2022-12-01 2025-07-01 Generation Bio Co Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions containing them
JP2025540076A (ja) 2022-12-01 2025-12-11 ジェネレーション バイオ カンパニー 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用
JP2025541726A (ja) 2022-12-01 2025-12-23 ジェネレーション バイオ カンパニー 細胞標的化のためのステルス脂質ナノ粒子組成物
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
WO2024125597A1 (en) 2022-12-14 2024-06-20 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
EP4634390A2 (de) 2022-12-15 2025-10-22 Orna Therapeutics, Inc. Zirkuläre rna-zusammensetzungen und verfahren
WO2024126809A1 (en) 2022-12-15 2024-06-20 Sanofi Mrna encoding influenza virus-like particle
EP4637813A1 (de) 2022-12-20 2025-10-29 Sanofi Pasteur Rhinovirus-mrna-impfstoff
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
EP4642797A1 (de) 2022-12-28 2025-11-05 BioNTech SE Gegen hiv gerichtete rna-zusammensetzungen
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
JP2026504815A (ja) 2022-12-29 2026-02-10 ポップヴァックス プライベート リミテッド マルチターゲットワクチンおよび治療剤
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
MX2025012337A (es) 2023-04-17 2026-02-03 Sanofi Pasteur Inc Formulaciones de polvo seco reconstituibles y métodos de uso de las mismas
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
AU2024265158A1 (en) 2023-05-03 2025-11-06 BioNTech SE Optimized csp variants and related methods
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
IL325865A (en) 2023-07-21 2026-03-01 BioNTech SE Compositions for administering Plasmodium antigens and related methods
WO2025024324A1 (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
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025026545A1 (en) 2023-08-01 2025-02-06 BioNTech SE Ionizable thioplipids and uses thereof
WO2025027089A1 (en) 2023-08-01 2025-02-06 BioNTech SE Ionizable thiolipids and uses thereof
CN121772938A (zh) 2023-08-03 2026-03-31 百欧恩泰欧洲股份公司 用于递送单纯疱疹病毒抗原的药物组合物及相关方法
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
WO2025027579A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
CN121866336A (zh) 2023-08-03 2026-04-14 百欧恩泰欧洲股份公司 靶向hiv的rna组合物
WO2025031498A1 (zh) 2023-08-10 2025-02-13 浙江海昶生物医药技术有限公司 Toll样受体激动剂及其应用
WO2025036297A1 (zh) 2023-08-11 2025-02-20 浙江海昶生物医药技术有限公司 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
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
AU2024343011A1 (en) 2023-09-11 2026-03-26 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
AU2024357861A1 (en) 2023-10-12 2026-04-23 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
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
TW202525287A (zh) 2023-12-21 2025-07-01 德商拜恩技術運輸科技有限責任公司 可離子化脂質
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
WO2025184508A1 (en) 2024-03-01 2025-09-04 Acuitas Therapeutics, Inc. Materials and methods for encapsulating therapeutics in lipid nanoparticles
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
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
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
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
WO2026096919A1 (en) 2024-11-01 2026-05-07 Sail Biomedicines, Inc. Novel dipepetide core lipids, lipid carriers, and methods of using thereof
WO2026093616A1 (en) 2024-11-04 2026-05-07 Variant Nucleic acid encoding cone-rod homeobox (crx) protein, and expression vectors thereof, for use in treating a ciliopathy

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
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
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
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
US5225212A (en) 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
WO1993024640A2 (en) 1992-06-04 1993-12-09 The Regents Of The University Of California Methods and compositions for in vivo gene therapy
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.
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US6075012A (en) 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
JPH10506093A (ja) 1994-06-22 1998-06-16 メガバイオス・コーポレイション カチオン性両親媒性化合物
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
US5627159A (en) 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
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
CA2222328C (en) 1995-06-07 2012-01-10 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
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced 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
CA2223179A1 (en) 1995-06-07 1996-12-19 Bob Dale Brown Phosphonic acid-based cationic lipids
EP0874910A4 (de) 1995-06-07 1999-04-21 Life Technologies Inc Durch peptide erhöhte kationische lipid transfektionen
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
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
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
CA2227373A1 (en) 1995-07-21 1997-02-06 Promega Biosciences, Inc. Novel amide-based cationic lipids
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
CA2237316C (en) 1995-11-30 2012-06-26 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
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US5877220A (en) 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
WO1998051278A2 (en) 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
WO1998051285A2 (en) 1997-05-15 1998-11-19 Genzyme Corporation Cationic amphiphile formulations
DE69837274T2 (de) 1997-10-10 2007-11-08 Inex Pharmaceuticals Corp., Burnaby Verfahren zur verkapselung von nukleinsäuren in lipiddoppelschichten
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
WO2000003683A2 (en) 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
EP1829856A3 (de) 1998-11-12 2009-02-25 Invitrogen Corporation Transfektionsreagenzien
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
KR100758158B1 (ko) 1999-07-14 2007-09-12 알자 코포레이션 중성 지질중합체 및 그를 함유하는 리포솜 조성물
ES2235916T3 (es) * 1999-07-15 2005-07-16 Inex Pharmaceuticals Corp. Preparacion de agentes terapeuticos de encapsulacion lipidica.
IL150628A0 (en) 2000-01-10 2003-02-12 Yissum Res Dev Co Use of lipid conjugates in the treatment of disease
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
JP2004511572A (ja) 2000-10-25 2004-04-15 ザ ユニバーシティ オブ ブリティッシュ コロンビア 標的化送達のための脂質製剤
WO2002034879A2 (en) 2000-10-27 2002-05-02 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
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
EP1509203B1 (de) 2002-05-15 2016-04-13 California Pacific Medical Center Zuführung nukleinsäureähnlicher verbindungen
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
ATE477337T1 (de) 2003-01-16 2010-08-15 Univ Pennsylvania Zusammensetzungen und verfahren zur sirna-hemmung von 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
AU2004246904B2 (en) 2003-06-18 2010-02-04 Biolab Ltd. Sphingolipids polyalkylamine conjugates for use in transfection
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
CN1882693B (zh) 2003-09-15 2012-08-15 普洛体维生物治疗公司 聚乙二醇修饰的脂质化合物及其应用
ATE536418T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1773857A4 (de) 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc Das immunsystem stimulierende sirna-moleküle und anwendungen davon
NZ572403A (en) 2004-09-24 2010-03-26 Alnylam Pharmaceuticals Inc Rnai modulation of apob and uses thereof
US20060134189A1 (en) 2004-11-17 2006-06-22 Protiva Biotherapeutics, Inc siRNA silencing of apolipoprotein B
DE102004060863A1 (de) * 2004-12-17 2006-06-22 Dr. Johannes Heidenhain Gmbh Winkelmesseinrichtung
WO2007086883A2 (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
AU2009236219B8 (en) 2008-04-15 2015-06-25 Arbutus Biopharma Corporation Silencing of CSN5 gene expression using interfering RNA
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
EP4241767B1 (de) 2008-11-10 2024-10-23 Arbutus Biopharma Corporation Neuartige lipide und zusammensetzungen zur verabreichung von therapeutika
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
JP2014508102A (ja) 2010-11-15 2014-04-03 ライフ テクノロジーズ コーポレーション アミン含有トランスフェクション試薬ならびにそれを生成および使用するための方法

Also Published As

Publication number Publication date
EP1766035B1 (de) 2011-12-07
WO2005121348A1 (en) 2005-12-22
US20190071669A1 (en) 2019-03-07
AU2005252273B2 (en) 2011-04-28
US20160115477A1 (en) 2016-04-28
US20110060032A1 (en) 2011-03-10
US20060008910A1 (en) 2006-01-12
AU2005252273A1 (en) 2005-12-22
US7799565B2 (en) 2010-09-21
US9181545B2 (en) 2015-11-10
JP2008501730A (ja) 2008-01-24
CA2569664C (en) 2013-07-16
EP1766035A4 (de) 2009-04-22
JP4796062B2 (ja) 2011-10-19
US9926560B2 (en) 2018-03-27
EP1766035A1 (de) 2007-03-28
CA2569664A1 (en) 2005-12-22

Similar Documents

Publication Publication Date Title
EP1766035A4 (de) Lipidverkapselte interferenz-rna
WO2005007196A3 (en) Lipid encapsulated interfering rna
EP1781593A4 (de) Kationische lipide und verwendungsverfahren
BRPI0417107A (pt) fragmento de anticorpo, métodos de preparação do fragmento de anticorpo, ácido nucléico isolado, composições, célula hospedeira e métodos de fabricação e de geração de fragmento de anticorpo
EP1896073A4 (de) Anti-il-23-antikörper, zusammensetzungen, verfahren und verwendung
WO2006113679A3 (en) Delivery of sirna by neutral lipid compositions
WO2008131419A3 (en) Glycoconjugates of rna interference agents
WO2011139911A3 (en) Lipid formulated single stranded rna
MX2022013691A (es) Formulaciones que contienen lipidos.
WO2005005977A3 (en) Fluorescent phospholipase assays and compositions
MA30153B1 (fr) Anticorps anti-ox40l et methodes correspondantes
DK3181578T3 (da) Bærer til targeting af nerveceller
EP1971366A4 (de) Menschliche anti-il-23-antikörper, zusammensetzungen, verfahren und verwendungen
DK1835923T3 (da) Skemaer til intra-artikulær visco-supplering
UY3485Q (es) Envase para perfume (b9)
NO20043353L (no) Antistoff-inneholdende opplosningsfarmasoytika
WO2009058913A3 (en) Yeast cell wall protein (ycwp) encapsulated multilyered nanoparticles for nucleic acid delivery (sirna)
MX2009013084A (es) Metodos para mejorar las propiedades de la proteina.
MA32899B1 (fr) Combinaisons anticancereuses contenant des anticorps reconnaissant specifiquement cd38 et de la cytarabine
EP1819365A4 (de) Zusammensetzungen und verfahren zur induzierung einer immunantwort bei einem säugetier und verfahren zur vermeidung einer immunantwort zu oligonucleotidwirkstoffen wie etwa short interfering rnas
WO2006079048A3 (en) Databases for assessing nucleic acids
WO2007075626A3 (en) Immunostimulatory activity of palindromic immune modulatory oligonucleotides (imo tm) contiaining different lengths of palindromic segments
WO2005042692A3 (en) A method of inhibiting the expression of genes which mediate cellular cholesterol influx in animal cells and inhibiting the production of proteins resulting from the expression of such genes using cholesterol absorption inhibitors
GB0508110D0 (en) Gene delivery
WO2005049832A3 (en) FcϜRIIA-SPECIFIC NUCLEIC ACID INTERFERENCE