AU2009238175C1 - Novel lipid formulations for nucleic acid delivery - Google Patents

Novel lipid formulations for nucleic acid delivery Download PDF

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Publication number
AU2009238175C1
AU2009238175C1 AU2009238175A AU2009238175A AU2009238175C1 AU 2009238175 C1 AU2009238175 C1 AU 2009238175C1 AU 2009238175 A AU2009238175 A AU 2009238175A AU 2009238175 A AU2009238175 A AU 2009238175A AU 2009238175 C1 AU2009238175 C1 AU 2009238175C1
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AU
Australia
Prior art keywords
mol
lipid
particle
nucleic acid
sirna
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AU2009238175A
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English (en)
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AU2009238175A1 (en
AU2009238175B2 (en
Inventor
Lloyd B. Jeffs
Kieu Lam
Ian Maclachlan
Lorne R. Palmer
Edward Yaworski
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Arbutus Biopharma Corp
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Arbutus Biopharma Corp
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Application filed by Arbutus Biopharma Corp filed Critical Arbutus Biopharma Corp
Publication of AU2009238175A1 publication Critical patent/AU2009238175A1/en
Priority to AU2013202932A priority Critical patent/AU2013202932A1/en
Publication of AU2009238175B2 publication Critical patent/AU2009238175B2/en
Assigned to ARBUTUS BIOPHARMA CORPORATION reassignment ARBUTUS BIOPHARMA CORPORATION Request to Amend Deed and Register Assignors: PROTIVA BIOTHERAPEUTICS, INC.
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    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J9/00Normal steroids containing carbon, hydrogen, halogen or oxygen substituted in position 17 beta by a chain of more than two carbon atoms, e.g. cholane, cholestane, coprostane
    • 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
    • C12N15/11DNA 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
    • 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
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2009238175A 2008-04-15 2009-04-15 Novel lipid formulations for nucleic acid delivery Active AU2009238175C1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2013202932A AU2013202932A1 (en) 2008-04-15 2013-04-08 Novel lipid formulations for nucleic acid delivery

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4522808P 2008-04-15 2008-04-15
US61/045,228 2008-04-15
PCT/CA2009/000496 WO2009127060A1 (en) 2008-04-15 2009-04-15 Novel lipid formulations for nucleic acid delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2013202932A Division AU2013202932A1 (en) 2008-04-15 2013-04-08 Novel lipid formulations for nucleic acid delivery

Publications (3)

Publication Number Publication Date
AU2009238175A1 AU2009238175A1 (en) 2009-10-22
AU2009238175B2 AU2009238175B2 (en) 2013-12-12
AU2009238175C1 true AU2009238175C1 (en) 2023-11-30

Family

ID=41198741

Family Applications (1)

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AU2009238175A Active AU2009238175C1 (en) 2008-04-15 2009-04-15 Novel lipid formulations for nucleic acid delivery

Country Status (14)

Country Link
US (11) US8058069B2 (enExample)
EP (1) EP2279254B1 (enExample)
JP (1) JP5475753B2 (enExample)
CN (1) CN102119217B (enExample)
AU (1) AU2009238175C1 (enExample)
CA (1) CA2721333C (enExample)
DK (1) DK2279254T3 (enExample)
ES (1) ES2638448T3 (enExample)
HU (1) HUE034483T2 (enExample)
IL (1) IL208744A (enExample)
NZ (1) NZ588583A (enExample)
PL (1) PL2279254T3 (enExample)
PT (1) PT2279254T (enExample)
WO (1) WO2009127060A1 (enExample)

Families Citing this family (825)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10214983A1 (de) 2002-04-04 2004-04-08 TransMIT Gesellschaft für Technologietransfer mbH Vernebelbare Liposomen und ihre Verwendung zur pulmonalen Applikation von Wirkstoffen
DK1519714T3 (da) 2002-06-28 2011-01-31 Protiva Biotherapeutics Inc Fremgangsmåde og apparat til fremstilling af liposomer
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
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
ES2937245T3 (es) 2005-08-23 2023-03-27 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
CN103614375A (zh) 2006-05-11 2014-03-05 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
US9006191B2 (en) * 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering RNA
EP2245039A4 (en) * 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
JP2011518117A (ja) * 2008-03-05 2011-06-23 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 Eg5およびVEGF遺伝子の発現を阻害するための組成物および方法
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
ES2630805T3 (es) 2008-07-14 2017-08-24 Polypid Ltd. Composición de vehículo de fármaco de liberación sostenida
JP5529142B2 (ja) 2008-09-25 2014-06-25 アルナイラム ファーマシューティカルズ, インコーポレイテッド 血清アミロイドa遺伝子の発現を阻害するための脂質製剤組成物および方法
PL2350043T3 (pl) 2008-10-09 2014-09-30 Tekmira Pharmaceuticals Corp Ulepszone aminolipidy i sposoby dostarczania kwasów nukleinowych
WO2010048228A2 (en) 2008-10-20 2010-04-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
WO2010054406A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Novel lipids and compositions for the delivery of therapeutics
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
ES2572361T3 (es) * 2008-12-31 2016-05-31 Roche Innovation Ct Copenhagen As Utilización de oligómeros de ANB antisentido de ApoB para el tratamiento de síndromes coronarios agudos
JP6032724B2 (ja) * 2009-03-12 2016-11-30 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. 脂質製剤組成物およびEg5遺伝子とVEGF遺伝子の発現を阻害する方法
KR101184928B1 (ko) * 2009-05-14 2012-09-20 한국과학기술연구원 작은 올리고뉴클레오티드의 중합 방법 및 상기 중합 방법에 의하여 제조된 고분자 올리고뉴클레오티드의 용도
WO2010147992A1 (en) * 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
EA201270019A1 (ru) 2009-06-15 2012-06-29 Элнилэм Фармасьютикалз, Инк. Двуцепочечная рнк, включенная в липидный состав и мишенью которой является ген pcsk9
DE102009031274A1 (de) 2009-06-30 2011-01-13 Justus-Liebig-Universität Giessen Liposomen zur pulmonalen Applikation
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
CA2767129C (en) 2009-07-01 2015-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
IN2012DN00570A (enExample) 2009-07-14 2015-06-12 Polypid Ltd
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
CN102639115A (zh) 2009-09-15 2012-08-15 阿尔尼拉姆医药品有限公司 脂质配制的组合物及抑制eg5和vegf基因的表达的方法
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
WO2011038160A2 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
JP5723378B2 (ja) 2009-11-03 2015-05-27 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. トランスサイレチン(ttr)を阻害する脂質製剤化組成物及び方法
JP5823405B2 (ja) 2009-11-04 2015-11-25 ザ ユニバーシティ オブ ブリティッシュ コロンビア 核酸含有脂質粒子および関連方法
RS58405B1 (sr) 2009-12-01 2019-04-30 Translate Bio Inc Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima
JP5860409B2 (ja) 2010-01-19 2016-02-16 ポリピッド リミテッド 徐放性核酸マトリックス組成物
JP2013523162A (ja) 2010-04-06 2013-06-17 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 Cd274/pd−l1遺伝子の発現を阻害するための組成物および方法
EP2558074B1 (en) 2010-04-08 2018-06-06 The Trustees of Princeton University Preparation of lipid nanoparticles
WO2011139911A2 (en) * 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
JP2013527856A (ja) 2010-05-12 2013-07-04 プロチバ バイオセラピューティクス インコーポレイティッド 陽イオン性脂質およびその使用方法
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
US8865675B2 (en) 2010-05-12 2014-10-21 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
EP2575895A2 (en) 2010-05-24 2013-04-10 Merck Sharp & Dohme Corp. Novel amino alcohol cationic lipids for oligonucleotide delivery
AU2011261434B2 (en) 2010-06-02 2015-11-26 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
ES2586580T3 (es) 2010-07-06 2016-10-17 Glaxosmithkline Biologicals Sa Inmunización de mamíferos grandes con dosis bajas de ARN
HUE026646T2 (en) 2010-07-06 2016-07-28 Glaxosmithkline Biologicals Sa Preferred liposomes containing lipids of PKA value for delivery of RNA
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
RU2625546C2 (ru) * 2010-07-06 2017-07-14 Новартис Аг Катионные эмульсии "масло-в-воде"
HRP20161352T1 (hr) 2010-07-06 2016-12-02 Glaxosmithkline Biologicals Sa Čestice nalik na virione za unos samoreplicirajućih molekula rna
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
LT2606134T (lt) 2010-08-17 2019-07-25 Sirna Therapeutics, Inc. Hepatito b viruso (hbv) geno raiškos slopinimas, tarpininkaujant rnr interferencijai naudojant mažą interferuojančią nukleorūgštį (sina)
WO2012027467A1 (en) 2010-08-26 2012-03-01 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF PROLYL HYDROXYLASE DOMAIN 2 (PHD2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
KR20130100278A (ko) 2010-08-31 2013-09-10 머크 샤프 앤드 돔 코포레이션 올리고뉴클레오티드의 전달을 위한 신규 단일 화학 존재물 및 방법
EP4043040B1 (en) 2010-08-31 2023-01-11 GlaxoSmithKline Biologicals SA Small liposomes for delivery of immunogen-encoding rna
DK4226941T3 (da) 2010-08-31 2024-12-02 Glaxosmithkline Biologicals Sa Pegylerede liposomer til levering af immunogen-kodende rna
KR101223483B1 (ko) * 2010-09-10 2013-01-17 한국과학기술연구원 전하결합 및 생분해성 공유결합으로 동시에 연결된 고분자―siRNA 나노입자 전달체
WO2012040184A2 (en) 2010-09-20 2012-03-29 Merck Sharp & Dohme Corp. Novel low molecular weight cationic lipids for oligonucleotide delivery
EP2621480B1 (en) 2010-09-30 2018-08-15 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR102266691B1 (ko) 2010-10-11 2021-06-23 노파르티스 아게 항원 전달 플랫폼
US9029590B2 (en) 2010-10-21 2015-05-12 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
EP2632472B1 (en) 2010-10-29 2017-12-13 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
WO2012061259A2 (en) 2010-11-05 2012-05-10 Merck Sharp & Dohme Corp. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012078967A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (epo) production
CA3217805A1 (en) 2011-03-29 2012-10-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of tmprss6 gene
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
US20120301537A1 (en) * 2011-05-23 2012-11-29 Delta-Fly Pharma, Inc. LIPOSOME CONTAINING shRNA MOLECULE TARGETING A THYMIDYLATE SYNTHASE AND USE THEREOF
US8592572B2 (en) * 2011-05-23 2013-11-26 Delta-Fly Pharma, Inc. Liposome containing shRNA molecule targeting a thymidylate synthase and use thereof
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
EP2723351B1 (en) 2011-06-21 2018-02-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
KR102232287B1 (ko) 2011-06-21 2021-03-29 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 그 사용 방법
RU2631805C2 (ru) 2011-06-21 2017-09-26 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии генов аполипопротеина с-iii (арос3)
EP4134433A1 (en) 2011-06-23 2023-02-15 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
US11896636B2 (en) 2011-07-06 2024-02-13 Glaxosmithkline Biologicals Sa Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
HRP20220250T1 (hr) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
EP2764007A4 (en) * 2011-10-05 2015-05-06 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR DISABLING ALDEHYDE DEHYDROGENASE
WO2013059922A1 (en) 2011-10-25 2013-05-02 The University Of British Columbia Limit size lipid nanoparticles and related methods
US9320814B2 (en) * 2011-11-01 2016-04-26 Board Of Regents Of The University Of Nebraska Polyplexes of hydrophobically-modified siRNA for delivery of siRNA
AU2012340159B2 (en) 2011-11-18 2017-09-07 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
HK1200089A1 (en) 2011-12-07 2015-07-31 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
PT2817287T (pt) 2012-02-24 2018-12-28 Arbutus Biopharma Corp Lípidos catiónicos trialquílicos e seus métodos de utilização
DK2830595T3 (da) 2012-03-29 2019-12-02 Translate Bio Inc Ioniserbare kationiske lipider
AU2013243952A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
HK1206612A1 (en) * 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
JP6232416B2 (ja) 2012-04-19 2017-11-15 サーナ・セラピューティクス・インコーポレイテッドSirna Therapeutics,Inc. オリゴヌクレオチド送達のための、新規なジエステルおよびトリエステルベースの低分子量、生分解性カチオン性脂質
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
BR112014030677A2 (pt) 2012-06-08 2022-07-19 Shire Human Genetic Therapies distribuição pulmonar de mrna para células-alvo não-pulmonares
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
CN102719437A (zh) * 2012-07-11 2012-10-10 潍坊医学院 利用慢病毒载体介导PLK1 RNAi在治疗食管鳞癌转移中的应用
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
EP3336187A1 (en) 2012-12-05 2018-06-20 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
KR102605775B1 (ko) 2013-03-14 2023-11-29 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C5 iRNA 조성물 및 그 이용 방법
WO2014160243A1 (en) 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
EP2970955B1 (en) 2013-03-14 2018-11-14 Translate Bio, Inc. Methods for purification of messenger rna
WO2014153052A2 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
EP3932947A1 (en) 2013-03-14 2022-01-05 Translate Bio MA, Inc. Methods and compositions for delivering mrna coded antibodies
US20140288149A1 (en) 2013-03-15 2014-09-25 Graham Lord Mir-142 and antagonists thereof for treating disease
HUE071526T2 (hu) 2013-03-15 2025-09-28 Translate Bio Inc Nukleinsavak bejuttatásának szinergista fokozása kevert kiszerelésekkel
JP6605446B2 (ja) 2013-03-15 2019-11-13 ザ ユニバーシティ オブ ブリティッシュ コロンビア 形質移入用の脂質ナノ粒子および関連方法
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
MX379208B (es) 2013-05-22 2025-03-10 Alnylam Pharmaceuticals Inc Composiciones de arni de serpina1 y sus metodos de uso.
EP3828276A1 (en) 2013-05-22 2021-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
HUE056760T2 (hu) 2013-07-11 2022-03-28 Modernatx Inc A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik
CN105555757A (zh) * 2013-07-23 2016-05-04 普洛体维生物治疗公司 用于递送信使rna的组合物和方法
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
TWI669393B (zh) 2013-10-02 2019-08-21 艾爾妮蘭製藥公司 抑制lect2基因表現之組合物及方法
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
LT3052628T (lt) 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
BR112016008832A2 (pt) 2013-10-22 2017-10-03 Shire Human Genetic Therapies Distribuição de mrna no snc e suas utilizações
CN112656954A (zh) 2013-10-22 2021-04-16 夏尔人类遗传性治疗公司 用于递送信使rna的脂质制剂
US10202601B2 (en) 2013-11-22 2019-02-12 Mina Therapeutics Limited C/EBPα short activating RNA compositions and methods of use
AU2014362262B2 (en) 2013-12-12 2021-05-13 Alnylam Pharmaceuticals, Inc. Complement component iRNA compositions and methods of use thereof
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Hbv 및 바이러스 질병 및 질환을 위한 crispr­cas 시스템 및 조성물의 전달,용도 및 치료적 적용
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用
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
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
SG11201608725YA (en) 2014-04-25 2016-11-29 Shire Human Genetic Therapies Methods for purification of messenger rna
CN112301031A (zh) 2014-05-22 2021-02-02 阿尔尼拉姆医药品有限公司 血管紧张素原(AGT)iRNA组合物及其使用方法
EP3587409B8 (en) 2014-05-30 2022-07-13 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
JP6779866B2 (ja) 2014-06-13 2020-11-04 チルドレンズ メディカル センター コーポレイション ミトコンドリアを単離するための製品および方法
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
KR102511554B1 (ko) 2014-06-24 2023-03-16 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
IL298516B2 (en) 2014-06-25 2025-03-01 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
CN106794141B (zh) * 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
WO2016011222A2 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Circular polynucleotides
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
US9765333B2 (en) 2014-08-21 2017-09-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing marburg virus gene expression
EP3185957B1 (en) 2014-08-29 2022-06-01 Alnylam Pharmaceuticals, Inc. Patisiran for use in treating transthyretin mediated amyloidosis
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
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
RU2017114964A (ru) 2014-10-02 2018-11-07 Протива Байотерапьютикс, Инк. Композиции и способы для подавления экспрессии гена вируса гепатита в
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
AU2015350120B2 (en) 2014-11-17 2021-05-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
US9943595B2 (en) 2014-12-05 2018-04-17 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
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)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3234141A4 (en) 2014-12-18 2018-06-20 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
EP3234192B1 (en) 2014-12-19 2021-07-14 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
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016118697A1 (en) 2015-01-21 2016-07-28 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP3900702A1 (en) 2015-03-19 2021-10-27 Translate Bio, Inc. Mrna therapy for pompe disease
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2016165825A1 (en) 2015-04-13 2016-10-20 Curevac Ag Method for producing rna compositions
CN104997801A (zh) * 2015-05-28 2015-10-28 厦门成坤生物技术有限公司 包含季铵盐类化合物和siRNA的组合物及其制备方法
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的crispr酶突變
EP3666895A1 (en) 2015-06-18 2020-06-17 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
SI3313829T1 (sl) 2015-06-29 2024-09-30 Acuitas Therapeutics Inc. Lipidi in formulacije lipidnih nanodelcev za dostavo nukleinskih kislin
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
JP2018528257A (ja) * 2015-07-10 2018-09-27 ペプティノボ・バイオファーマ・リミテッド・ライアビリティ・カンパニーPeptinovo Biopharma, LLC 疎水性薬剤の効能を改善するための配合物
MA42502A (fr) 2015-07-21 2018-05-30 Modernatx Inc Vaccins contre une maladie infectieuse
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
US20180208932A1 (en) 2015-07-29 2018-07-26 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
WO2017020026A1 (en) 2015-07-30 2017-02-02 Modernatx, Inc. Concatemeric peptide epitopes rnas
DK3329002T3 (da) 2015-07-31 2021-01-11 Alnylam Pharmaceuticals Inc Transthyretin (ttr)-irna-sammensætninger og fremgangsmåder til anvendelse deraf til behandling eller forebyggelse af ttr-forbundne sygdomme
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
EP3337908A4 (en) 2015-08-18 2019-01-23 The Broad Institute, Inc. METHOD AND COMPOSITIONS FOR CHANGING THE FUNCTION AND STRUCTURE OF CHROMATIN GRINDING AND / OR DOMAINS
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
EA201890619A1 (ru) 2015-09-02 2018-09-28 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ iRNA ЛИГАНДА 1 БЕЛКА ЗАПРОГРАММИРОВАННОЙ ГИБЕЛИ КЛЕТОК 1 (PD-L1) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
WO2017048620A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
PL3350333T3 (pl) 2015-09-17 2022-03-07 Modernatx, Inc. Polinukleotydy zawierające region stabilizujący ogon
CA2998810A1 (en) 2015-09-17 2017-03-23 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
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
SG10201912568PA (en) 2015-10-16 2020-02-27 Ipsen Biopharm Ltd Stabilizing camptothecin pharmaceutical compositions
EA201891001A1 (ru) 2015-10-22 2018-11-30 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот против вируса ветряной оспы (vzv)
EP3364982A4 (en) 2015-10-22 2019-04-17 ModernaTX, Inc. VACCINES AGAINST SEXUALLY TRANSMITTED DISEASES
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
US12234454B2 (en) 2015-10-22 2025-02-25 The Broad Institute, Inc. Crispr enzymes and systems
WO2017070613A1 (en) * 2015-10-22 2017-04-27 Modernatx, Inc. Human cytomegalovirus vaccine
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
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
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
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
EP3397261B1 (en) 2015-11-30 2025-07-09 Sana Biotechnology, Inc. Sub-mitochondrial particles for use in treating cardiovascular diseases
ES2924407T3 (es) 2015-12-10 2022-10-06 Modernatx Inc Composiciones y procedimientos para el suministro de agentes terapéuticos
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
SMT202200252T1 (it) 2015-12-22 2022-07-21 Modernatx Inc Composti e composizioni per il rilascio intracellulare di agenti
PL3394093T3 (pl) 2015-12-23 2022-05-16 Modernatx, Inc. Metody stosowania polinukleotydów kodujących ligand ox40
EP3400023A1 (en) 2016-01-10 2018-11-14 ModernaTX, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
WO2017124037A1 (en) 2016-01-15 2017-07-20 The Children's Medical Center Corporation Therapeutic use of mitochondria and combined mitochondrial agents
EP3825400B1 (en) 2016-04-08 2024-12-25 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR102424476B1 (ko) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3458107B1 (en) 2016-05-18 2024-03-13 ModernaTX, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
LT3458474T (lt) 2016-05-18 2022-10-10 Modernatx, Inc. Imunitetą moduliuojančius polipeptidus koduojančių mrnr deriniai ir jų naudojimas
AU2017268394A1 (en) 2016-05-18 2019-01-03 Modernatx, Inc. Polynucleotides encoding relaxin
DK3458083T5 (da) 2016-05-18 2024-10-21 Modernatx Inc Polynukleotider, der koder for interleukin-12 (il12), og anvendelser heraf
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CA3027312A1 (en) 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
WO2017218704A1 (en) * 2016-06-14 2017-12-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
KR20230156150A (ko) 2016-06-17 2023-11-13 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
CA3034681A1 (en) 2016-06-30 2018-01-04 Arbutus Biopharma Corporation Compositions and methods for delivering messenger rna
WO2018009838A1 (en) 2016-07-07 2018-01-11 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous rna
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
CN109715205A (zh) 2016-08-19 2019-05-03 库瑞瓦格股份公司 用于癌症治疗的rna
WO2018039131A1 (en) 2016-08-22 2018-03-01 Protiva Biotherapeutics, Inc. Anti-pd-1 antibodies, or fragments thereof, for treating hepatitis b
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
EP3519578B1 (en) 2016-10-03 2021-12-22 Precision Nanosystems Inc Compositions for transfecting resistant cell types
WO2018119514A1 (en) * 2016-12-28 2018-07-05 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
MA46584A (fr) 2016-10-21 2019-08-28 Modernatx Inc Vaccin contre le cytomégalovirus humain
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3532103B1 (en) 2016-10-26 2025-12-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
US11542490B2 (en) 2016-12-08 2023-01-03 CureVac SE RNAs for wound healing
MA50335A (fr) 2016-12-08 2020-08-19 Modernatx Inc Vaccins à acide nucléique contre des virus respiratoires
JP2020501545A (ja) 2016-12-08 2020-01-23 キュアバック アーゲー 肝疾患の処置または予防のためのrna
TWI790217B (zh) 2016-12-16 2023-01-21 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白(TTR)iRNA組成物於治療或預防TTR相關疾病之方法
US11464847B2 (en) 2016-12-23 2022-10-11 Curevac Ag Lassa virus vaccine
WO2018115527A2 (en) 2016-12-23 2018-06-28 Curevac Ag Mers coronavirus vaccine
WO2018115507A2 (en) 2016-12-23 2018-06-28 Curevac Ag Henipavirus vaccine
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
US11739308B2 (en) 2017-03-15 2023-08-29 The Broad Institute, Inc. Cas13b orthologues CRISPR enzymes and systems
AU2018234814B2 (en) 2017-03-15 2022-06-30 Modernatx, Inc. Crystal forms of amino lipids
WO2018170270A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. Respiratory syncytial virus vaccine
EP3596041B1 (en) 2017-03-15 2022-11-02 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
EP3595676A4 (en) 2017-03-17 2021-05-05 Modernatx, Inc. RNA VACCINES AGAINST ZOONOSES
CA3050614A1 (en) 2017-03-17 2018-09-20 Curevac Ag Rna vaccine and immune checkpoint inhibitors for combined anticancer therapy
WO2018172556A1 (en) 2017-03-24 2018-09-27 Curevac Ag Nucleic acids encoding crispr-associated proteins and uses thereof
EP3607074A4 (en) 2017-04-05 2021-07-07 Modernatx, Inc. REDUCTION OR ELIMINATION OF IMMUNE RESPONSES TO NON-INTRAVENOUS THERAPEUTIC PROTEINS, FOR EXAMPLE SUBCUTANEOUSLY
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
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
CN118384268A (zh) 2017-04-18 2024-07-26 阿尔尼拉姆医药品有限公司 具有乙肝病毒(hbv)感染的受试者的治疗方法
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
DK3622079T3 (da) 2017-05-08 2025-11-17 Flagship Pioneering Innovations V Inc Sammensætninger til at gøre membranfusion lettere og anvendelser deraf
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis through the administration of mRNA with an optimal codon encoding ctfr
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP7284101B2 (ja) 2017-05-31 2023-05-30 ウルトラジェニクス ファーマシューティカル インク. 糖原病iii型のための治療薬
EP3630200A4 (en) 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
US11015204B2 (en) 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
KR20200038236A (ko) 2017-06-13 2020-04-10 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 큐론을 포함하는 조성물 및 그의 용도
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
AU2018298422B2 (en) 2017-07-04 2023-04-06 CureVac SE Novel nucleic acid molecules
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
JP7355731B2 (ja) 2017-08-16 2023-10-03 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US12065396B2 (en) 2017-08-17 2024-08-20 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
WO2019040516A1 (en) 2017-08-22 2019-02-28 Rubius Therapeutics, Inc. METHODS AND COMPOSITIONS OF LIPID NANOPARTICLES FOR THE PRODUCTION OF MODIFIED ERYTHROID CELLS
US11602557B2 (en) 2017-08-22 2023-03-14 Cure Vac SE Bunyavirales vaccine
US11744801B2 (en) 2017-08-31 2023-09-05 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019048631A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
EP3681514A4 (en) 2017-09-14 2021-07-14 ModernaTX, Inc. RNA VACZINE AGAINST ZIKA VIRUS
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
IL321714A (en) 2017-10-19 2025-08-01 CureVac SE Novel artificial nucleic acid molecules
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
EP3707271A1 (en) 2017-11-08 2020-09-16 CureVac AG Rna sequence adaptation
US12018080B2 (en) 2017-11-13 2024-06-25 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the CLEC2D-KLRB1 pathway
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019115635A1 (en) 2017-12-13 2019-06-20 Curevac Ag Flavivirus vaccine
WO2019118806A1 (en) 2017-12-14 2019-06-20 Solid Biosciences Inc. Non-viral production and delivery of genes
CN111727252A (zh) 2017-12-18 2020-09-29 阿尔尼拉姆医药品有限公司 高速泳动族盒-1(HMGB1)iRNA组合物及其使用方法
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
CN118634318A (zh) * 2017-12-27 2024-09-13 武田药品工业株式会社 含核酸脂质纳米粒子及其用途
EP3737408A1 (en) 2018-01-08 2020-11-18 Novartis AG Immune-enhancing rnas for combination with chimeric antigen receptor therapy
US20190330591A1 (en) 2018-01-10 2019-10-31 Rubius Therapeutics, Inc. Amplifiable rnas for therapeutic cell systems
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
US10982210B2 (en) 2018-03-02 2021-04-20 Sixfold Bioscience Ltd. Compositions for delivery of cargo to cells
US20210361761A1 (en) 2018-04-05 2021-11-25 Curevac Ag Novel yellow fever nucleic acid molecules for vaccination
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
BR112020020933A2 (pt) 2018-04-17 2021-04-06 Curevac Ag Moléculas de rna de rsv inovadoras e composições para vacinação
AU2019265904A1 (en) 2018-05-11 2020-11-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
JP7178409B2 (ja) 2018-06-08 2022-11-25 富士フイルム株式会社 化合物またはその塩および脂質粒子
US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
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
WO2020002525A1 (en) 2018-06-27 2020-01-02 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
WO2020033601A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel cas12b enzymes and systems
EP3837367A1 (en) 2018-08-16 2021-06-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法
GB2600800B (en) 2018-09-04 2023-08-16 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
AU2019336679B2 (en) 2018-09-04 2025-05-29 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3853364A1 (en) 2018-09-18 2021-07-28 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
JP7640452B2 (ja) 2018-09-19 2025-03-05 モデルナティエックス インコーポレイテッド 高純度peg脂質及びそれらの使用
WO2020061284A1 (en) 2018-09-19 2020-03-26 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
IL281615B1 (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
CA3110914A1 (en) 2018-09-28 2020-04-02 Nutcracker Therapeutics, Inc. Tertiary amino lipidated cationic peptides for nucleic acid delivery
ES2951836T3 (es) * 2018-10-01 2023-10-25 Alnylam Pharmaceuticals Inc Lípidos biodegradables para el suministro de agentes activos
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
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
US12357705B2 (en) 2018-11-09 2025-07-15 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
BR112021008953A2 (pt) 2018-11-09 2021-08-10 Arbutus Biopharma Corporation partículas de ácido nucleico-lipídio e de ácido-lipídio, composições, métodos para introduzir um ácido nucleico em uma célula, para distribuição in vivo de um ácido nucleico, para tratar uma doença ou um distúrbio e para preparar um composto, usos de uma partícula, compostos e nanopartícula de lipídio
KR20210105889A (ko) 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 지질 나노입자 제제
US20200157157A1 (en) 2018-11-21 2020-05-21 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
US11685906B2 (en) 2018-12-06 2023-06-27 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
AU2019406186A1 (en) 2018-12-20 2021-07-15 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of KCNT1 related disorders
US12268669B2 (en) 2018-12-20 2025-04-08 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
US20220040281A1 (en) 2018-12-21 2022-02-10 Curevac Ag Rna for malaria vaccines
US12042527B2 (en) 2019-01-08 2024-07-23 Modernatx, Inc. Use of mRNAs encoding OX40L, IL-23 and IL-36gamma in combination with immune checkpoint blockade for treating particular cancers
TWI852977B (zh) 2019-01-10 2024-08-21 美商健生生物科技公司 前列腺新抗原及其用途
AU2020205717B2 (en) 2019-01-11 2025-09-11 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
EP3920950A1 (en) 2019-02-08 2021-12-15 CureVac AG Coding rna administered into the suprachoroidal space in the treatment of ophtalmic diseases
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
US12070495B2 (en) 2019-03-15 2024-08-27 Modernatx, Inc. HIV RNA vaccines
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
KR102660546B1 (ko) 2019-06-07 2024-04-24 후지필름 가부시키가이샤 지질 조성물
WO2020247882A1 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Engineered casx systems
WO2020254535A1 (en) 2019-06-18 2020-12-24 Curevac Ag Rotavirus mrna vaccine
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
JP7692829B2 (ja) 2019-07-30 2025-06-16 塩野義製薬株式会社 Murf1を標的とする核酸医薬
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
CA3144902A1 (en) 2019-08-14 2022-01-19 Andreas Thess Rna combinations and compositions with decreased immunostimulatory properties
JOP20220037A1 (ar) 2019-08-14 2023-01-30 Acuitas Therapeutics Inc جسيمات نانوية دهنية محسنة لتوصيل الأحماض النووية
MX2022001896A (es) 2019-08-16 2022-06-02 Regeneron Pharma Formulaciones anti-c5 de alta concentracion.
EP4022058A2 (en) 2019-08-30 2022-07-06 Sixfold Bioscience Ltd. Compositions for transfer of cargo to cells
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CA3150452A1 (en) 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
US11066355B2 (en) 2019-09-19 2021-07-20 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
EP4048793A1 (en) 2019-10-22 2022-08-31 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2021081277A1 (en) 2019-10-25 2021-04-29 Regeneron Pharmaceuticals, Inc. Dosing regimens for treating or preventing c5-associated diseases
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
CA3155921A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN114981431A (zh) 2019-11-13 2022-08-30 阿尔尼拉姆医药品有限公司 用于治疗血管紧张素原(agt)相关病症的方法和组合物
DK4328309T3 (da) 2019-11-15 2025-08-18 Fujifilm Corp Lipidsammensætning
PH12022551208A1 (en) 2019-11-18 2023-04-24 Janssen Biotech Inc Vaccines based on mutant calr and jak2 and their uses
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
WO2021102411A1 (en) 2019-11-22 2021-05-27 Generation Bio Co. Ionizable lipids and nanoparticle compositions thereof
BR112022011417A2 (pt) 2019-12-13 2022-08-30 Alnylam Pharmaceuticals Inc Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類PATATIN磷脂酶結構域3(PNPLA3)iRNA組成物及其使用方法
EP4076647A1 (en) 2019-12-20 2022-10-26 CureVac AG Lipid nanoparticles for delivery of nucleic acids
EP4103542A4 (en) 2020-01-09 2024-02-14 Guide Therapeutics, LLC NANOMATERIALS
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
EP3886897A1 (en) 2020-02-04 2021-10-06 CureVac AG Coronavirus vaccine
CA3170377A1 (en) 2020-02-10 2021-08-19 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
JP7735288B2 (ja) 2020-02-18 2025-09-08 アルナイラム ファーマシューティカルズ, インコーポレイテッド アポリポタンパク質C3(APOC3)iRNA組成物およびその使用法
CA3169889A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
EP4114948A1 (en) 2020-03-06 2023-01-11 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
US11759515B2 (en) 2020-03-09 2023-09-19 Arcturus Therapeutics, Inc. Compositions and methods for inducing immune responses
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
JP2023519274A (ja) 2020-03-26 2023-05-10 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 コロナウイルスiRNA組成物およびその使用方法
AU2021241696A1 (en) 2020-03-27 2022-12-01 Generation Bio Co. Novel lipids and nanoparticle compositions thereof
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
IL296781A (en) 2020-03-30 2022-11-01 BioNTech SE Rna compositions targeting claudin-18.2
US11773391B2 (en) 2020-04-01 2023-10-03 University of Pittsburgh—of the Commonwealth System of Higher Education Therapeutic and diagnostic target for SARS-CoV-2 and COVID-19
US20230295622A1 (en) 2020-04-06 2023-09-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20230159933A1 (en) 2020-04-07 2023-05-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
WO2021205077A1 (en) 2020-04-09 2021-10-14 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
WO2021213924A1 (en) 2020-04-22 2021-10-28 BioNTech SE Coronavirus vaccine
EP4143319A1 (en) 2020-04-27 2023-03-08 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
WO2021220053A2 (en) 2020-04-27 2021-11-04 Sixfold Bioscience Ltd. Compositions containing nucleic acid nanoparticles with modular functionality
CN116096381A (zh) 2020-04-30 2023-05-09 阿尔尼拉姆医药品有限公司 补体因子B(CFB)iRNA组合物及其使用方法
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
CA3179444A1 (en) 2020-05-20 2021-11-25 Avak Kahvejian Immunogenic compositions and uses thereof
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
CA3182026A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
JP2023530229A (ja) 2020-05-29 2023-07-14 キュアバック エスイー 核酸ベースの混合ワクチン
MX2022015042A (es) 2020-05-29 2023-03-09 Flagship Pioneering Innovations Vi Llc Composiciones de trem y métodos relacionados con las mismas.
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
KR20230035043A (ko) 2020-06-15 2023-03-10 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 근이영양증을 위한 아데노-연관 바이러스 벡터 전달
WO2021257782A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2025056938A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
WO2022009052A2 (en) 2020-07-06 2022-01-13 Janssen Biotech, Inc. Prostate neoantigens and their uses
IL299571A (en) 2020-07-08 2023-02-01 Janssen Sciences Ireland Unlimited Co Rna replicon vaccines against hbv
CN115768438A (zh) * 2020-07-10 2023-03-07 盖纳万科学有限公司 用于向肺递送治疗剂的脂质纳米颗粒
CA3189338A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
WO2022023559A1 (en) 2020-07-31 2022-02-03 Curevac Ag Nucleic acid encoded antibody mixtures
WO2022036170A1 (en) 2020-08-14 2022-02-17 Arcturus Therapeutics, Inc. Method of lyophilizing lipid nanoparticles
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
EP4157344A2 (en) 2020-08-31 2023-04-05 CureVac SE Multivalent nucleic acid based coronavirus vaccines
TW202218669A (zh) 2020-09-03 2022-05-16 美商旗艦先鋒創新有限責任公司 免疫原性組成物及其用途
WO2022054955A1 (ja) 2020-09-14 2022-03-17 富士フイルム株式会社 脂質組成物
JP2023549011A (ja) 2020-09-15 2023-11-22 ヴァーヴ・セラピューティクス,インコーポレーテッド 遺伝子編集のための脂質製剤
TW202228784A (zh) 2020-09-23 2022-08-01 奧地利商艾柏力維亞生技有限公司 用以於一患者中螯合非預期的抗peg抗體的化合物
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
AU2021360494A1 (en) 2020-10-14 2023-05-18 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
EP4232581A1 (en) 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
US11771652B2 (en) 2020-11-06 2023-10-03 Sanofi Lipid nanoparticles for delivering mRNA vaccines
EP4244355A1 (en) 2020-11-13 2023-09-20 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
AU2021385572A1 (en) 2020-11-25 2023-06-22 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP4256054A1 (en) 2020-12-03 2023-10-11 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
MX2023007137A (es) 2020-12-21 2023-08-28 Beam Therapeutics Inc Nanomateriales que comprenden acetales unidos a ésteres.
CA3171051A1 (en) 2020-12-22 2022-06-30 Curevac Ag Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
US11918643B2 (en) 2020-12-22 2024-03-05 CureVac SE RNA vaccine against SARS-CoV-2 variants
AU2021410712A1 (en) * 2020-12-22 2023-07-06 Cornell University Zwitterionic lipid nanoparticle compositions, and methods of use
WO2022137133A1 (en) 2020-12-22 2022-06-30 Curevac Ag Rna vaccine against sars-cov-2 variants
US20240175020A1 (en) 2020-12-23 2024-05-30 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
US20220202952A1 (en) * 2020-12-28 2022-06-30 Industrial Technology Research Institute Immunostimulatory lipoplex, pharmaceutical composition including immunostimulatory lipoplex, and uses thereof
US20240316090A1 (en) 2020-12-28 2024-09-26 Arcturus Therapeutics, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
MX2023008333A (es) 2021-01-20 2023-08-21 Beam Therapeutics Inc Nanomateriales.
EP4281080A4 (en) 2021-01-20 2025-09-24 Beam Therapeutics Inc NANOMATERIALS COMPRISING A BIODEGRADABLE ELEMENT
CA3206148A1 (en) 2021-01-24 2022-07-28 Michael David FORREST Therapeutic modifiers of the reverse mode of atp synthase
CA3170747A1 (en) 2021-01-27 2022-08-04 Moritz THRAN Method of reducing the immunostimulatory properties of in vitro transcribed rna
TW202245835A (zh) 2021-02-04 2022-12-01 美商默沙東有限責任公司 用於肺炎球菌結合物疫苗之奈米乳化液佐劑組合物
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
CA3209032A1 (en) 2021-02-26 2022-09-01 Ethris Gmbh Formulations for aerosol formation and aerosols for the delivery of nucleic acid
AU2022226098A1 (en) 2021-02-26 2023-08-24 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
JP2024508896A (ja) 2021-03-04 2024-02-28 アルナイラム ファーマシューティカルズ, インコーポレイテッド アンジオポエチン様3(ANGPTL3)iRNA組成物およびその使用方法
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
JP2024511092A (ja) 2021-03-26 2024-03-12 ミナ セラピューティクス リミテッド TMEM173saRNA組成物及び使用方法
WO2022200575A1 (en) 2021-03-26 2022-09-29 Glaxosmithkline Biologicals Sa Immunogenic compositions
TW202305134A (zh) 2021-03-29 2023-02-01 美商艾拉倫製藥股份有限公司 杭丁頓蛋白(HTT)iRNA劑組成物及其使用方法
KR20230165276A (ko) 2021-03-31 2023-12-05 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 타노트랜스미션 폴리펩티드 및 암의 치료에서의 이의 용도
US20250345524A1 (en) 2021-03-31 2025-11-13 CureVac SE Syringes containing pharmaceutical compositions comprising rna
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
WO2022215036A1 (en) 2021-04-08 2022-10-13 Vaxthera Sas Coronavirus vaccine comprising a mosaic protein
CA3214538A1 (en) 2021-04-20 2022-10-27 Joel DE BEER Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
JP7656363B2 (ja) 2021-04-22 2025-04-03 インベンテージ ラボ インコーポレイテッド 脂質ナノ粒子の製造方法およびその製造装置
IL307926A (en) 2021-04-26 2023-12-01 Alnylam Pharmaceuticals Inc Transmembrane assemblies, serine 6 ((TMPRSS6 IRNA) and methods of using them
WO2022232289A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
CA3171589A1 (en) 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ, インコーポレイテッド ナトリウム-グルコース共輸送体2(sglt2)irna組成物およびその使用方法
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
WO2022244831A1 (en) 2021-05-21 2022-11-24 Okinawa Institute Of Science And Technology School Corporation Coronavirus vaccine suitable for elderly individuals
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
WO2022256395A1 (en) 2021-06-02 2022-12-08 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
TW202308663A (zh) 2021-06-04 2023-03-01 美商艾拉倫製藥股份有限公司 人類染色體9開讀框72(C9ORF72)iRNA劑組成物及其使用方法
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
US20240269302A1 (en) 2021-06-11 2024-08-15 Nibec Co., Ltd. Nanoparticle comprising peptide-lipid conjugate for delivering oligonucleotide into target cell and pharmaceutical composition comprising same
AU2022290278A1 (en) 2021-06-11 2024-01-04 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
KR20240023420A (ko) 2021-06-14 2024-02-21 제너레이션 바이오 컴퍼니 양이온성 지질 및 이의 조성물
IL309578A (en) 2021-06-22 2024-02-01 Bio Trip B V Nanoparticles containing nucleic acid
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
MX2023015489A (es) 2021-06-30 2024-01-19 Alnylam Pharmaceuticals Inc Metodos y composiciones para tratar un trastorno asociado al angiotensinogeno (agt).
US20250270562A1 (en) 2021-07-09 2025-08-28 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
US20230042860A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
WO2023288287A2 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
US20230046606A1 (en) 2021-07-15 2023-02-16 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
KR20240036041A (ko) 2021-07-21 2024-03-19 알닐람 파마슈티칼스 인코포레이티드 대사 장애-연관 표적 유전자 iRNA 조성물 및 이의 사용 방법
TW202325312A (zh) 2021-07-23 2023-07-01 美商艾拉倫製藥股份有限公司 β-鏈蛋白(CTNNB1)iRNA組成物及其使用方法
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
EP4379054A4 (en) 2021-07-27 2025-12-17 Stand Therapeutics Co Ltd PEPTIDE TAG AND CODING NUCLEOIC ACID
EP4377458A1 (en) 2021-07-29 2024-06-05 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
US20240342206A1 (en) 2021-07-30 2024-10-17 CureVac SE mRNAS FOR TREATMENT OR PROPHYLAXIS OF LIVER DISEASES
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
IL310295A (en) 2021-08-04 2024-03-01 Alnylam Pharmaceuticals Inc iRNA compositions and methods for silencing angiotensinogen (AGT)
JP2024534766A (ja) 2021-08-13 2024-09-26 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 第XII因子(F12)iRNA組成物およびその使用方法
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
EP4395790A2 (en) 2021-08-31 2024-07-10 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
US20240398933A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids comprising phosphatidylserine
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
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 (en) 2021-09-14 2024-07-24 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
TW202330916A (zh) 2021-09-17 2023-08-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸之組成物和方法
EP4401742A2 (en) 2021-09-17 2024-07-24 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CN118159654A (zh) 2021-09-20 2024-06-07 阿尔尼拉姆医药品有限公司 抑制素亚基βE(INHBE)调节剂组合物及其使用方法
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered casx repressor systems
CN118369427A (zh) 2021-10-15 2024-07-19 阿尔尼拉姆医药品有限公司 肝外递送irna组合物及其使用方法
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
EP4430024A1 (en) 2021-11-08 2024-09-18 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
KR102772764B1 (ko) 2021-11-10 2025-02-27 주식회사 에스엠엘바이오팜 트레할로즈 유도체 및 신규 구조 유지 화합물을 포함하는 핵산 의약품 전달용 지질나노입자의 약학 조성물
CA3238292A1 (en) 2021-11-16 2023-05-25 Alessandra Bartolozzi Novel ionizable lipids and lipid nanoparticles and methods of using the same
KR20240128683A (ko) 2021-11-22 2024-08-26 세일 바이오메디슨스, 인크. 신규한 이온화 가능 지질 및 지질 나노입자, 및 이를 사용하는 방법
EP4436598A2 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and their uses
EP4436984A1 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Coronavirus immunogen compositions and their uses
CN118401544A (zh) 2021-11-24 2024-07-26 旗舰创业创新六公司 水痘-带状疱疹病毒免疫原组合物及其用途
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
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
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US20240401024A1 (en) 2021-12-17 2024-12-05 Flagship Pioneering Innovations Vi, Llc Methods for enrichment of circular rna under denaturing conditions
JP2025501731A (ja) 2021-12-20 2025-01-23 ビーム セラピューティクス インク. イオン化可能なアミン脂質及び脂質ナノ粒子
JP2025500824A (ja) 2021-12-20 2025-01-15 ビーム セラピューティクス インク. 四価脂質化合物を含むナノ材料
WO2023121965A1 (en) 2021-12-20 2023-06-29 Beam Therapeutics Inc. Nanomaterial comprising diamines
WO2023121970A1 (en) 2021-12-20 2023-06-29 Beam Therapeutics Inc. Ionizable amine and ester lipids and lipid nanoparticles
EP4452942A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterials comprising disulfides
CA3241061A1 (en) 2021-12-22 2023-06-29 Alexandra Sophie DE BOER Compositions and methods for purifying polyribonucleotides
EP4452337A1 (en) 2021-12-23 2024-10-30 Flagship Pioneering Innovations VI, LLC Circular polyribonucleotides encoding antifusogenic polypeptides
US20230201817A1 (en) 2021-12-23 2023-06-29 Biontech Delivery Technologies Gmbh Modular drug production system
CA3243054A1 (en) 2021-12-23 2025-04-08 Renagade Therapeutics Management Inc. Constrained Lipids and Associated Utilization Processes
EP4463475A2 (en) 2022-01-14 2024-11-20 Anjarium Biosciences AG Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
EP4470543A1 (en) 2022-01-25 2024-12-04 Kawasaki Institute of Industrial Promotion Rna-containing composition for transdermal administration, and method for administering said composition
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
AU2023212857A1 (en) 2022-01-27 2024-07-04 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023161350A1 (en) 2022-02-24 2023-08-31 Io Biotech Aps Nucleotide delivery of cancer therapy
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
WO2023176821A1 (ja) 2022-03-15 2023-09-21 富士フイルム株式会社 脂質組成物
AU2023238223A1 (en) 2022-03-23 2024-10-10 Nanovation Therapeutics Inc. High sterol-containing lipid nanoparticles
JP2025510229A (ja) 2022-03-25 2025-04-14 セイル バイオメディシンズ インコーポレイテッド 新規のイオン化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
CN119654166A (zh) 2022-04-01 2025-03-18 内诺维什疗法股份有限公司 mRNA递送方法及其组合物
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
AU2023251104A1 (en) 2022-04-07 2024-10-17 Renagade Therapeutics Management Inc. Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
JP2025511756A (ja) 2022-04-08 2025-04-16 フラッグシップ パイオニアリング イノベーションズ セブン,エルエルシー ワクチン及び関連する方法
WO2023199943A1 (ja) 2022-04-12 2023-10-19 国立研究開発法人理化学研究所 コロナウイルス感染症を処置することに用いられる抗体
CN119365208A (zh) 2022-04-13 2025-01-24 巴塞罗那自治大学 通过表达klotho蛋白的基因疗法治疗神经肌肉疾病
CN118900914A (zh) 2022-04-27 2024-11-05 上海瑞宏迪医药有限公司 核酸构建体及其应用
CA3257006A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc TREM COMPOSITIONS AND METHODS OF USE FOR TREATING PROLIFERATIVE DISORDERS
JP2025515206A (ja) 2022-05-13 2025-05-13 上海瑞宏迪医薬有限公司 Utrを含む核酸構築物及びその使用
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
TW202409283A (zh) 2022-05-13 2024-03-01 美商旗艦先鋒創新有限責任(Vii)公司 雙股dna組合物及相關方法
CN119562966A (zh) 2022-05-13 2025-03-04 生物技术公司 靶向hiv的rna组合物
WO2023230098A1 (en) 2022-05-23 2023-11-30 Logicbio Therapeutics, Inc. Gene therapy compositions and methods of use thereof
EP4531902A1 (en) 2022-05-25 2025-04-09 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
AU2023275780A1 (en) 2022-05-25 2024-12-05 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
JP2025522297A (ja) 2022-05-25 2025-07-15 ビオンテック エスエー サルポックス抗原の送達のためのrna組成物及び関連する方法
US20250375534A1 (en) 2022-05-30 2025-12-11 BioNTech SE Complexes for delivery of nucleic acids
TW202411426A (zh) 2022-06-02 2024-03-16 美商斯奎柏治療公司 經工程化的2類v型crispr系統
CA3258109A1 (en) 2022-06-03 2023-12-07 Bio-Trip B.V. LIPID NANOPARTICLES BASED ON MULTIPURPOSE MOLECULES FOR THE ADMINISTRATION OF NUCLEIC ACIDS
IL317529A (en) 2022-06-07 2025-02-01 Scribe Therapeutics Inc Compositions and methods for targeting PCSK9
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CA3258303A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Compositions of lipid nanoparticles and their uses
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
CN120344660A (zh) 2022-07-18 2025-07-18 雷纳嘉德医疗管理公司 基因编辑组分、系统和使用方法
IL317826A (en) 2022-07-20 2025-02-01 Beam Therapeutics Inc Nanomaterials include triols
KR20250044720A (ko) 2022-08-01 2025-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 면역조절 단백질 및 관련 방법
AU2023323547A1 (en) 2022-08-12 2025-03-20 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2024035952A1 (en) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
CN120077130A (zh) 2022-08-18 2025-05-30 阿尔尼拉姆医药品有限公司 通用非靶向sirna组合物及其使用方法
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
EP4577243A1 (en) 2022-08-26 2025-07-02 Ethris GmbH Stable lipid or lipidoid nanoparticle suspensions
CN120239693A (zh) 2022-08-31 2025-07-01 赛欧生物医药股份有限公司 新型可电离脂质和脂质纳米颗粒以及其使用方法
EP4585591A1 (en) 2022-09-06 2025-07-16 Kyushu University, National University Corporation Lipid nanoparticles
MA71735A (fr) 2022-09-15 2025-05-30 Regeneron Pharmaceuticals, Inc. Compositions d'arni de 17b-hydroxystéroïde déshydrogénase de type 13 (hsd17b13) et leurs procédés d'utilisation
EP4342460A1 (en) 2022-09-21 2024-03-27 NovoArc GmbH Lipid nanoparticle with nucleic acid cargo
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
CN120283052A (zh) 2022-09-23 2025-07-08 恩科洛玛生物公司 用于表观遗传调控hbv基因表达的组合物和方法
WO2024064934A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
IL319414A (en) 2022-09-23 2025-05-01 BioNTech SE Compositions for delivering antigens at the liver level and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
CA3267272A1 (en) 2022-09-26 2024-04-04 Glaxosmithkline Biologicals Sa FLU VIRUS VACCINES
WO2024068728A1 (en) 2022-09-27 2024-04-04 Resolution Therapeutics Limited Enhanced macrophages
WO2024071319A1 (en) 2022-09-28 2024-04-04 Okinawa Institute Of Science And Technology School Corporation Composition comprising isolated or enriched phosphoenolpyruvate or a salt thereof
WO2024073588A1 (en) * 2022-09-28 2024-04-04 Board Of Regents, The University Of Texas System Respiratory dry powder delivery
US20240174732A1 (en) 2022-10-05 2024-05-30 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additional polypeptides and their use in treating cancer
EP4598954A1 (en) 2022-10-06 2025-08-13 BioNTech SE Rna compositions targeting claudin-18.2
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
EP4606372A1 (en) 2022-10-18 2025-08-27 Tipton Corp. Taylor reactor and capsule particle manufacturing method
WO2024083345A1 (en) 2022-10-21 2024-04-25 BioNTech SE Methods and uses associated with liquid compositions
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
AU2023372397A1 (en) 2022-10-31 2025-05-08 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
JP2025537178A (ja) 2022-11-08 2025-11-14 オーナ セラピューティクス, インコーポレイテッド 環状rna組成物
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
AR131008A1 (es) 2022-11-08 2025-02-05 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
WO2024119074A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
IL320870A (en) 2022-12-01 2025-07-01 Generation Bio Co Lipid nanoparticles containing nucleic acids, ionizable lipids, sterols, lipid-anchored polymers and auxiliary lipids and their uses
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
EP4626400A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
KR20240087590A (ko) 2022-12-09 2024-06-19 서울대학교산학협력단 B 세포 및 T 세포 내로 mRNA를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이의 용도
EP4631528A1 (en) 2022-12-09 2025-10-15 Nibec Co., Ltd. Nanoparticle comprising peptide-based conjugate for delivering oligonucleotide into target cell and pharmaceutical composition comprising same
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任(Vii)公司 用於將治療劑遞送至骨之組成物和方法
EP4634388A1 (en) 2022-12-14 2025-10-22 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
IL321401A (en) 2022-12-20 2025-08-01 Univ Utrecht Holding Bv Nucleic acid-lipid particles
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024141955A1 (en) 2022-12-28 2024-07-04 BioNTech SE Rna compositions targeting hiv
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
WO2024141786A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Multitarget vaccines and therapeutics
EP4628201A1 (en) 2023-01-04 2025-10-08 Inventage Lab Inc. Spiral-structured chip for preparing lipid nanoparticles, and method for preparing lipid nanoparticles by using same
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
US20240269251A1 (en) 2023-01-09 2024-08-15 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
EP4648794A2 (en) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
WO2024151685A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
WO2024155957A1 (en) 2023-01-19 2024-07-25 Altos Labs, Inc. Use of regeneration factors in organ transplantation
EP4658239A1 (en) 2023-02-03 2025-12-10 GlaxoSmithKline Biologicals S.A. Rna formulation
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫調節組合物及相關方法
WO2024168010A2 (en) 2023-02-09 2024-08-15 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
IL322468A (en) 2023-02-13 2025-09-01 Flagship Pioneering Innovations Vii Llc Ionizable lipids containing degradable crosslinkers and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
WO2024173828A1 (en) 2023-02-17 2024-08-22 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified uracil
WO2024173836A2 (en) 2023-02-17 2024-08-22 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified cytosine
DE112024001143T5 (de) 2023-03-08 2025-12-18 CureVac SE Neue lipid-nanopartikel-formeln für die abgabe von nukleinsäuren
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
AU2024237452A1 (en) 2023-03-15 2025-10-23 United Immunity, Co., Ltd. Lipid particle
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
AU2024234874A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
WO2024194767A1 (en) 2023-03-17 2024-09-26 BioNTech SE Automated drug production system
TW202444910A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 編碼casx之信使rna
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
TW202444906A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向pcsk9之組合物及方法
TW202444921A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向lpa之組合物及方法
WO2024206565A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Repressor fusion protein systems
WO2024209418A1 (en) 2023-04-07 2024-10-10 BioNTech SE Heating and cooling jacket for fluid vessels
AU2024255972A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
AU2024252590A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
WO2024213740A1 (en) 2023-04-14 2024-10-17 BioNTech SE Vaccine mixing method, syringe and system
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
CN121001738A (zh) 2023-04-27 2025-11-21 葛兰素史克生物有限公司 流感病毒疫苗
WO2024223724A1 (en) 2023-04-27 2024-10-31 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
CN121038807A (zh) 2023-05-03 2025-11-28 生物技术欧洲股份公司 优化的csp变体及相关方法
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
DE102023001946A1 (de) 2023-05-12 2024-11-14 Friedrich-Schiller-Universität Jena, Körperschaft des öffentlichen Rechts Nanopartikel für den Transport von Wirkstoffen mit anionischen Gruppen, Verfahren zu deren Herstellung und deren Verwendung
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
AU2024310564A1 (en) 2023-06-29 2025-11-27 BioNTech SE Modular multiplatform system for mrna drug production
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
TW202515531A (zh) 2023-07-07 2025-04-16 美商輝瑞股份有限公司 兩親性tlr7/8佐劑及其用途
WO2025011529A2 (en) 2023-07-07 2025-01-16 Shanghai Circode Biomed Co., Ltd. Circular rna vaccines for seasonal flu and methods of uses
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024335A2 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of 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
WO2025021083A1 (en) * 2023-07-24 2025-01-30 National Institute Of Biological Sciences, Beijing Lipid nanoparticle compositions for localized treatment of skin diseases
WO2025024486A2 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases 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
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
WO2025030154A1 (en) 2023-08-03 2025-02-06 The Trustees Of The University Of Pennsylvania Pharmaceutical compositions for delivery of herpes simplex virus glycoprotein b antigens and related methods
WO2025027576A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025030097A2 (en) 2023-08-03 2025-02-06 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
NL2035538B1 (en) 2023-08-03 2025-02-18 Univ Eindhoven Tech Lipid-like copolymers and method of producing these and the use thereof, pharmaceutical compositions comprising said copolymers, and the use of said compositions.
WO2025027579A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
US12162927B1 (en) 2023-08-09 2024-12-10 Wyvern Pharmaceuticals Inc. Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025045767A1 (en) 2023-08-25 2025-03-06 Ethris Gmbh Stabilized lipid and lipidoid nanoparticle formulations with specific surfactant properties for enhanced pharmaceutical applications
WO2025045142A1 (en) 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025046121A1 (en) 2023-09-01 2025-03-06 Novoarc Gmbh Lipid nanoparticle with nucleic acid cargo and ionizable lipid
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025051994A1 (en) 2023-09-07 2025-03-13 Coave Therapeutics Ionizable lipid nanoparticles
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025057088A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
TW202525266A (zh) 2023-09-18 2025-07-01 美商旗艦先鋒創新有限責任(Vii)公司 可電離類脂質組成物及其治療用途
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025068489A1 (en) 2023-09-27 2025-04-03 Resolution Therapeutics Limited Therapeutic macrophages
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
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
US20250162981A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
US12435338B2 (en) 2023-11-22 2025-10-07 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US20250163421A1 (en) 2023-11-22 2025-05-22 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12195747B1 (en) 2023-11-22 2025-01-14 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US20250163422A1 (en) 2023-11-22 2025-05-22 Wyvem Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
WO2025114520A1 (en) 2023-12-01 2025-06-05 Coave Therapeutics Ionizable lipid nanoparticles
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025124711A1 (en) 2023-12-13 2025-06-19 BioNTech SE Glycolipid compositions
WO2025129201A1 (en) 2023-12-15 2025-06-19 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
WO2025134062A2 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025134066A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
WO2025140551A1 (en) 2023-12-29 2025-07-03 Neuro3 Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING NaV1.7 (SCN9A) -ASSOCIATED DISORDERS
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
KR102716110B1 (ko) 2024-01-19 2024-10-15 (주) 멥스젠 다양한 크기를 갖는 지질나노입자 제조용 미세유체장치 및 이를 이용한 제조방법
WO2025160334A1 (en) 2024-01-26 2025-07-31 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
US12416020B2 (en) 2024-02-20 2025-09-16 Wyvern Pharmaceuticals Inc. Plasmid encoding a TLR3 and Fc fusion protein
US20250263707A1 (en) 2024-02-20 2025-08-21 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
WO2025184508A1 (en) 2024-03-01 2025-09-04 Acuitas Therapeutics, Inc. Materials and methods for encapsulating therapeutics in lipid nanoparticles
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025202937A1 (en) 2024-03-26 2025-10-02 BioNTech SE Cancer vaccines
WO2025202287A1 (en) 2024-03-26 2025-10-02 Resolution Therapeutics Limited Method of producing macrophages
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025219570A1 (en) 2024-04-17 2025-10-23 Johannes Kepler Universität Linz Composition for inhibiting protein translocation and methods using the same
WO2025228975A1 (en) 2024-04-29 2025-11-06 BioNTech SE Particles, compositions and methods
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
US12480129B1 (en) 2024-09-17 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12480123B1 (en) 2024-09-20 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12480121B1 (en) 2024-09-20 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12486506B1 (en) 2024-09-20 2025-12-02 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12416021B1 (en) 2024-10-11 2025-09-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12416022B1 (en) 2024-10-29 2025-09-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12428654B1 (en) 2024-10-29 2025-09-30 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12497618B1 (en) 2025-09-09 2025-12-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050064595A1 (en) * 2003-07-16 2005-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
WO2005035764A1 (en) * 2003-10-16 2005-04-21 The University Of British Columbia Autogene nucleic acids encoding a secretable rna polymerase
WO2005120152A2 (en) * 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006002538A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
WO2006053430A1 (en) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US20070042031A1 (en) * 2005-07-27 2007-02-22 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression

Family Cites Families (222)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US534484A (en) * 1895-02-19 Price and weighing scale
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
DE3068743D1 (en) * 1980-01-16 1984-08-30 Weder Hans G Process and dialysis-installation for the preparation of bilayer-vesicles and their use
US4598051A (en) * 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
US4522808A (en) 1980-08-15 1985-06-11 Societe Anonyme Dite: L'oreal Anti-sunburn compositions containing 2-phenyl-indole derivatives
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4515736A (en) * 1983-05-12 1985-05-07 The Regents Of The University Of California Method for encapsulating materials into liposomes
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
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
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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5320906A (en) * 1986-12-15 1994-06-14 Vestar, Inc. Delivery vehicles with amphiphile-associated active ingredient
JP2539665B2 (ja) * 1988-06-20 1996-10-02 日本臓器製薬株式会社 神経疾患治療剤
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
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
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
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
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
JPH06171Y2 (ja) 1990-03-30 1994-01-05 日新製鋼株式会社 圧延機のパスライン切換装置
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
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
FR2674257B1 (fr) * 1991-03-20 1993-05-28 Armines Alliages a base de niobium et de titane resistant a l'oxydation a hautes temperatures.
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
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
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
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
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)
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
WO1993017125A1 (en) * 1992-02-19 1993-09-02 Baylor College Of Medicine Oligonucleotide modulation of cell growth
JPH08500481A (ja) 1992-05-11 1996-01-23 リボザイム・ファーマシューティカルズ・インコーポレーテッド ウイルスの複製を阻害するための方法および薬剤
ATE155681T1 (de) 1992-05-18 1997-08-15 Minnesota Mining & Mfg Einrichtung zur transmucosalen wirkstoffabgabe
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
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
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
WO1995002698A1 (en) 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6075012A (en) 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
AU696881B2 (en) 1994-06-22 1998-09-24 Megabios Corporation Cationic amphiphiles
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
EP1179340A3 (en) 1994-09-30 2003-05-07 INEX Pharmaceutical Corp. 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
WO1996011266A2 (en) 1994-10-05 1996-04-18 Amgen Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5627159A (en) 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
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
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
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
JPH11507815A (ja) 1995-06-07 1999-07-13 ジンタ・インコーポレイテッド ホスホン酸基本カチオン性脂質
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
JP4338106B2 (ja) 1995-06-07 2009-10-07 ライフ テクノロジーズ コーポレーション ペプチド増強カチオン脂質トランスフェクション
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
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
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
EP0869937A4 (en) 1995-07-21 2004-07-21 Promega Biosciences Inc AMID BASED CATIONIC LIPIDS
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
US20040142895A1 (en) 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US20030216335A1 (en) 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
US6330349B1 (en) 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
US5817856A (en) 1995-12-11 1998-10-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Radiation-protective phospholipid and method
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6713069B1 (en) 1996-04-16 2004-03-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for detecting, preventing, and treating African Hemorrhagic Fever
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US5841662A (en) * 1996-07-31 1998-11-24 Coburn Optical Industries, Inc. Method and apparatus for chucked work piece recognition
US6284267B1 (en) 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US6174861B1 (en) 1996-10-22 2001-01-16 The Children's Medical Center Corporation Methods of inhibiting angiogenesis via increasing in vivo concentrations of endostatin protein
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
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
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
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
WO1998051285A2 (en) 1997-05-15 1998-11-19 Genzyme Corporation Cationic amphiphile formulations
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
AU8428998A (en) 1997-07-24 1999-02-16 Inex Pharmaceuticals Corporation Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
AU1360499A (en) 1997-10-10 1999-05-03 Pieter R. Cullis Methods for encapsulating nucleic acids in lipid bilayers
JP2993489B2 (ja) * 1997-12-15 1999-12-20 日本電気株式会社 疑似多チャンネルステレオ再生装置
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
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
US6429200B1 (en) 1998-07-17 2002-08-06 Mirus Corporation Reverse micelles for delivery of nucleic acids
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
JP4854853B2 (ja) 1998-11-12 2012-01-18 ライフ テクノロジーズ コーポレーション トランスフェクション薬剤
US6649780B1 (en) * 1998-12-22 2003-11-18 Valentis, Inc. Cationic lipids
DE60036861T2 (de) 1999-03-02 2008-07-24 Transave, Inc. Verkapselung biologisch aktiver komplexe in liposomen
CA2370690A1 (en) 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
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
DE60026030T2 (de) 1999-07-14 2006-08-10 Alza Corp., Mountain View Neutrales lipopolymer und liposomale zusammensetzungen daraus
WO2001005374A1 (en) 1999-07-15 2001-01-25 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
WO2001015726A2 (en) 1999-08-27 2001-03-08 Inex Pharmaceuticals Corp. Compositions for stimulating cytokine secretion and inducing an immune response
US6458382B1 (en) 1999-11-12 2002-10-01 Mirus Corporation Nucleic acid transfer complexes
EP2845596A3 (en) 2000-01-10 2015-07-15 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Use of lipid conjugates in the treatment of disease
US20010048040A1 (en) * 2000-02-04 2001-12-06 Sheldon Paul C. Back-pressure responsive, self-reversing, control valve
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
CN1981873A (zh) 2000-06-09 2007-06-20 莱古伦公司 制备微胶粒的方法和所述微胶粒的用途
US20030082103A1 (en) 2000-10-11 2003-05-01 Targesome, Inc. Targeted therapeutic lipid constructs having cell surface targets
WO2002034236A2 (en) 2000-10-25 2002-05-02 The University Of British Columbia Lipid formulations for target delivery
JP2004521614A (ja) 2000-10-27 2004-07-22 インヴィトロジェン コーポレーション アンチセンスオリゴヌクレオチドを真核細胞に導入する方法
US20020081736A1 (en) 2000-11-03 2002-06-27 Conroy Susan E. Nucleic acid delivery
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
DE60134421D1 (de) 2000-12-08 2008-07-24 Coley Pharmaceuticals Gmbh Cpg-artige nukleinsäuren und verfahren zu ihrer verwendung
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US20040063654A1 (en) * 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc INTERFERENCE MEDIATION INHIBITION OF GENE RNA FROM MAP KINASE
AU2003206946A1 (en) 2002-02-22 2003-09-09 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
WO2003077584A1 (en) * 2002-03-08 2003-09-18 Nokia Corporation Method and device for controlling the power in an asymmetric soft handover condition
AU2003237864B2 (en) 2002-05-15 2008-12-18 California Pacific Medical Center Delivery of nucleic acid-like compounds
DK1519714T3 (da) 2002-06-28 2011-01-31 Protiva Biotherapeutics Inc Fremgangsmåde og apparat til fremstilling af liposomer
ES2322145T3 (es) 2002-07-26 2009-06-17 Novartis Vaccines And Diagnostics, Inc. Moleculas pequeñas modificadas de adn inerferente y procedimiento de uso.
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
ATE477337T1 (de) 2003-01-16 2010-08-15 Univ Pennsylvania Zusammensetzungen und verfahren zur sirna-hemmung von icam-1
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
CA2522637C (en) 2003-04-17 2014-01-21 Alnylam Pharmaceuticals, Inc. Modified irna agents
EP1638610B1 (en) 2003-06-18 2015-03-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Sphingoid polyalkylamine conjugates for vaccination
KR100961267B1 (ko) * 2003-06-26 2010-06-03 엘지디스플레이 주식회사 콜레스테릭 액정 컬러필터를 가지는 투과형 액정표시장치및 그 제조 방법
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
JP4796062B2 (ja) 2004-06-07 2011-10-19 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 脂質封入干渉rna
WO2006031901A2 (en) 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
CA2597724A1 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
WO2006102163A2 (en) 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
CA2637931A1 (en) * 2006-01-23 2007-07-26 Abbott Laboratories Chemically modified polycation polymer for sirna delivery
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2686735A1 (en) 2007-05-04 2008-11-13 Mdrna, Inc. Amino acid lipids and uses thereof
US9006191B2 (en) * 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering RNA
CA2709875C (en) 2008-01-02 2019-07-16 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
JP2011518117A (ja) 2008-03-05 2011-06-23 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 Eg5およびVEGF遺伝子の発現を阻害するための組成物および方法
EP2281041B1 (en) 2008-04-15 2014-07-02 Protiva Biotherapeutics Inc. Silencing of csn5 gene expression using interfering rna
CA2721333C (en) * 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
PL2350043T3 (pl) 2008-10-09 2014-09-30 Tekmira Pharmaceuticals Corp Ulepszone aminolipidy i sposoby dostarczania kwasów nukleinowych
WO2010048228A2 (en) 2008-10-20 2010-04-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
WO2010088537A2 (en) 2009-01-29 2010-08-05 Alnylam Pharmaceuticals, Inc. Improved lipid formulation
JP6032724B2 (ja) 2009-03-12 2016-11-30 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. 脂質製剤組成物およびEg5遺伝子とVEGF遺伝子の発現を阻害する方法
KR102066189B1 (ko) 2009-06-10 2020-01-14 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2010147992A1 (en) * 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
CA2767129C (en) * 2009-07-01 2015-01-06 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
US9018187B2 (en) * 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
US8455455B1 (en) * 2010-03-31 2013-06-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes involved in hemorrhagic fever
WO2011141704A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
US8865675B2 (en) * 2010-05-12 2014-10-21 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8466122B2 (en) * 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
CN113214102A (zh) 2010-11-15 2021-08-06 生命技术公司 含胺的转染试剂及其制备和使用方法
US9126966B2 (en) * 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
EP2764007A4 (en) 2011-10-05 2015-05-06 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR DISABLING ALDEHYDE DEHYDROGENASE
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US10421208B2 (en) 2013-03-12 2019-09-24 Sunpower Corporation Method and device for cleaving wafers
EP3185957B1 (en) 2014-08-29 2022-06-01 Alnylam Pharmaceuticals, Inc. Patisiran for use in treating transthyretin mediated amyloidosis
RU2017114964A (ru) * 2014-10-02 2018-11-07 Протива Байотерапьютикс, Инк. Композиции и способы для подавления экспрессии гена вируса гепатита в
US20200315967A1 (en) 2016-06-24 2020-10-08 Modernatx, Inc. Lipid nanoparticles
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050064595A1 (en) * 2003-07-16 2005-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
WO2005035764A1 (en) * 2003-10-16 2005-04-21 The University Of British Columbia Autogene nucleic acids encoding a secretable rna polymerase
WO2005120152A2 (en) * 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006002538A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
WO2006053430A1 (en) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US20070042031A1 (en) * 2005-07-27 2007-02-22 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression

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