ES2475065T3 - Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos - Google Patents

Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos Download PDF

Info

Publication number
ES2475065T3
ES2475065T3 ES09819991.2T ES09819991T ES2475065T3 ES 2475065 T3 ES2475065 T3 ES 2475065T3 ES 09819991 T ES09819991 T ES 09819991T ES 2475065 T3 ES2475065 T3 ES 2475065T3
Authority
ES
Spain
Prior art keywords
lipid
dma
nucleic acid
dlin
sirna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
ES09819991.2T
Other languages
English (en)
Spanish (es)
Inventor
Michael J. Hope
Sean C. Semple
Jianxin Chen
Thomas D. Madden
Pieter R. Cullis
Marco A. Ciufolini
Barbara Mui
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of British Columbia
Arbutus Biopharma Corp
Original Assignee
University of British Columbia
Tekmira Pharmaceuticals Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of British Columbia, Tekmira Pharmaceuticals Corp filed Critical University of British Columbia
Application granted granted Critical
Publication of ES2475065T3 publication Critical patent/ES2475065T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/39Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
    • 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
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • 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
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D317/00Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D317/08Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
    • C07D317/10Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings
    • C07D317/14Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D317/28Radicals substituted by nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D319/00Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D319/041,3-Dioxanes; Hydrogenated 1,3-dioxanes
    • C07D319/061,3-Dioxanes; Hydrogenated 1,3-dioxanes not condensed with other rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/555Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511Organic adjuvants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/555Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511Organic adjuvants
    • A61K2039/55555Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Mycology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Molecular Biology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Heterocyclic Compounds That Contain Two Or More Ring Oxygen Atoms (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
ES09819991.2T 2008-10-09 2009-10-09 Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos Active ES2475065T3 (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US220666P 2000-07-25
US10421908P 2008-10-09 2008-10-09
US10421208P 2008-10-09 2008-10-09
US104219P 2008-10-09
US104212P 2008-10-09
US22066609P 2009-06-26 2009-06-26
PCT/US2009/060251 WO2010042877A1 (en) 2008-10-09 2009-10-09 Improved amino lipids and methods for the delivery of nucleic acids

Publications (1)

Publication Number Publication Date
ES2475065T3 true ES2475065T3 (es) 2014-07-10

Family

ID=42100987

Family Applications (1)

Application Number Title Priority Date Filing Date
ES09819991.2T Active ES2475065T3 (es) 2008-10-09 2009-10-09 Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos

Country Status (9)

Country Link
US (2) US9139554B2 (enExample)
EP (3) EP2350043B9 (enExample)
JP (5) JP5777519B2 (enExample)
CN (2) CN102245590B (enExample)
AU (1) AU2009303345B2 (enExample)
CA (2) CA2984026C (enExample)
ES (1) ES2475065T3 (enExample)
PL (1) PL2350043T3 (enExample)
WO (1) WO2010042877A1 (enExample)

Families Citing this family (592)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
JP5324223B2 (ja) 2005-12-08 2013-10-23 トランセイブ, インク. 肺感染症を治療する抗感染薬の脂質ベースの組成物及びその使用法
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
US8324277B2 (en) 2007-08-01 2012-12-04 University of Pittsburgh—of the Commonwealth System of Higher Education Nitrated-fatty acids modulation of type II diabetes
JP5697988B2 (ja) 2007-12-27 2015-04-08 プロチバ バイオセラピューティクス インコーポレイティッド 干渉rnaを使用したポロ様キナーゼ発現のサイレンシング方法
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
WO2009134383A2 (en) 2008-05-01 2009-11-05 Complexa Inc. Vinyl substituted fatty acids
US20140024713A1 (en) 2008-06-19 2014-01-23 University Of Utah Research Foundation Use of nitrated lipids for treatment of side effects of toxic medical therapies
EP2299997A4 (en) 2008-06-19 2012-01-11 Univ Utah Res Found USE OF NITRATED LIPIDS FOR THE TREATMENT OF SIDE EFFECTS OF TOXIC MEDICAL THERAPIES
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
AU2009307677C1 (en) * 2008-10-20 2022-09-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
AU2015249072C1 (en) * 2008-10-20 2022-10-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
MX2011004859A (es) 2008-11-07 2011-08-03 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP4241767B1 (en) 2008-11-10 2024-10-23 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
US20120101148A1 (en) * 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
AU2010223967B2 (en) * 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
TR201811076T4 (tr) * 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
AU2010260148A1 (en) * 2009-06-15 2012-02-02 Alnylam Pharmaceuticals, Inc. Lipid formulated dsRNA targeting the PCSK9 gene
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
EP3045167A1 (en) 2009-07-31 2016-07-20 University of Pittsburgh - Of the Commonwealth System of Higher Education Keto fatty acids as anti-inflammatory agents
WO2011022460A1 (en) 2009-08-20 2011-02-24 Merck Sharp & Dohme Corp. Novel cationic lipids with various head groups for oligonucleotide delivery
CA2775092A1 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
CA2781276A1 (en) 2009-10-02 2011-04-07 Complexa, Inc. Heteroatom containing substituted fatty acids
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
WO2011088309A1 (en) * 2010-01-14 2011-07-21 Regulus Therapeutics Inc. Microrna compositions and methods
EP2525781A1 (en) 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011127255A1 (en) 2010-04-08 2011-10-13 Merck Sharp & Dohme Corp. Preparation of lipid nanoparticles
US9408914B2 (en) 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
JP5902617B2 (ja) * 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US20130123338A1 (en) * 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US8802863B2 (en) 2010-05-24 2014-08-12 Sirna Therapeutics, Inc. Amino alcohol cationic lipids for oligonucleotide delivery
CA2800401C (en) 2010-06-03 2020-09-15 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
SI2591114T1 (sl) 2010-07-06 2016-10-28 Glaxosmithkline Biologicals S.A. Imunizacija velikih sesalcev z majhnimi odmerki RNA
AU2011276234B2 (en) 2010-07-06 2016-02-25 Glaxosmithkline Biologicals S.A. Liposomes with lipids having an advantageous pKa- value for RNA delivery
US11291635B2 (en) 2010-07-06 2022-04-05 Glaxosmithkline Biological Sa Virion-like delivery particles for self-replicating RNA molecules
US20130323269A1 (en) * 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2606134B1 (en) 2010-08-17 2019-04-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US9233997B2 (en) 2010-08-26 2016-01-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of prolyl hydroxylase domain 2 (PHD2) gene expression using short interfering nucleic acid (siNA)
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
WO2012031043A1 (en) 2010-08-31 2012-03-08 Novartis Ag Pegylated liposomes for delivery of immunogen-encoding rna
KR20130100278A (ko) 2010-08-31 2013-09-10 머크 샤프 앤드 돔 코포레이션 올리고뉴클레오티드의 전달을 위한 신규 단일 화학 존재물 및 방법
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
MX349088B (es) 2010-09-20 2017-07-10 Merck Sharp & Dohme Lípidos catiónicos novedosos de bajo peso molecular para la entrega de oligonucleótidos.
KR20130114115A (ko) 2010-09-30 2013-10-16 머크 샤프 앤드 돔 코포레이션 올리고뉴클레오티드 전달을 위한 저분자량 양이온성 지질
ES3005233T3 (en) 2010-10-01 2025-03-14 Modernatx Inc Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
EP3520813B1 (en) 2010-10-11 2023-04-19 GlaxoSmithKline Biologicals S.A. Antigen delivery platforms
CN103153347A (zh) 2010-10-21 2013-06-12 默沙东公司 用于寡核苷酸递送的新型低分子量阳离子脂质
EP3766975A1 (en) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (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
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
DK3336082T3 (da) 2011-06-08 2020-04-27 Translate Bio Inc Spaltelige lipider
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
EP3854413A1 (en) 2011-07-06 2021-07-28 GlaxoSmithKline Biologicals SA Immunogenic combination compositions and uses thereof
EP4014966A1 (en) 2011-07-06 2022-06-22 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
EP2744491B1 (en) 2011-08-19 2020-07-29 The University of Utah Research Foundation Combination therapy with nitrated lipids and inhibitors of the renin-angiotensin-aldosterone system
US9126966B2 (en) * 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US9271952B2 (en) * 2011-10-11 2016-03-01 Complexa, Inc. Compositions and methods for treating nephropathy
KR20150000461A (ko) 2011-10-27 2015-01-02 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, n-말단 상에 관능화된 아미노산 유도체
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US9463247B2 (en) * 2011-12-07 2016-10-11 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated 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
ES3065854T3 (en) 2011-12-07 2026-05-08 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
CN104023751B (zh) 2012-01-27 2016-11-09 弗·哈夫曼-拉罗切有限公司 靶向递送至表达vla-4的细胞的整联蛋白拮抗剂结合物
MX2014008373A (es) 2012-01-27 2014-09-26 Hoffmann La Roche Conjugados antagonistas de integrina para suministro dirigido a celulas que expresan alfa-v-beta-3.
CA2857127A1 (en) 2012-01-27 2013-08-01 F. Hoffmann-La Roche Ag Integrin antagonist conjugates for targeted delivery to cells expressing lfa-1
BR112014015753A8 (pt) 2012-01-27 2017-07-04 Hoffmann La Roche derivado do polímero da quitosana, composição farmacêutica, método para o tratamento, utilização e presente invenção
DK2817287T3 (da) 2012-02-24 2019-01-02 Arbutus Biopharma Corp Trialkyl kationisk lipid og metoder til anvendelse deraf
CN102600173B (zh) * 2012-03-02 2013-05-29 海南美兰史克制药有限公司 氨氯地平/贝那普利药物组合物脂质体固体制剂
US10322089B2 (en) * 2012-03-14 2019-06-18 The Board Of Trustees Of The Leland Stanford Junior University Nanoparticles, nanoparticle delivery methods, and systems of delivery
WO2013143555A1 (en) * 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
EP3865123A1 (en) 2012-03-29 2021-08-18 Translate Bio, Inc. Lipid-derived neutral nanoparticles
ES2762873T3 (es) * 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
CN108949772A (zh) * 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9572897B2 (en) 2012-04-02 2017-02-21 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
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9402816B2 (en) 2012-04-19 2016-08-02 Sima Therapeutics, Inc. Diester and triester based low molecular weight, biodegradeable cationic lipids for oligonucleotide delivery
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
WO2013185069A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
TW201726599A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
EP2882706A1 (en) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Amine-containing lipidoids and uses thereof
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
ES2921623T3 (es) 2012-11-26 2022-08-30 Modernatx Inc ARN modificado terminalmente
MX2015006681A (es) 2012-11-29 2016-04-06 Insmed Inc Formulaciones de vancomicina estabilizadas.
EP2929035A1 (en) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
ES2882110T3 (es) 2013-03-14 2021-12-01 Translate Bio Inc Métodos y composiciones para administrar anticuerpos codificados con ARNm
EP2968586B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Cftr mrna compositions and related methods and uses
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP4614148A3 (en) 2013-03-15 2025-12-17 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
TW201534578A (zh) 2013-07-08 2015-09-16 Daiichi Sankyo Co Ltd 新穎脂質
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
WO2015061467A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
CA3236835A1 (en) 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
JP6527516B2 (ja) 2013-12-03 2019-06-05 ノースウェスタン ユニバーシティ リポソーム粒子、前述のものを作製する方法及びその使用
CN105916839B (zh) * 2014-01-09 2018-09-18 卫材R&D管理有限公司 阳离子性脂质
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
PT3122878T (pt) 2014-03-24 2019-02-01 Translate Bio Inc Terapia de arnm para o tratamento de doenças oculares
DK3134131T3 (en) 2014-04-23 2022-02-07 Modernatx Inc Nucleic acid vaccines
JP6571679B2 (ja) 2014-04-25 2019-09-04 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
SMT202000516T1 (it) 2014-05-15 2020-11-10 Insmed Inc Metodi per trattamento di infezioni micobatteriche polmonati non tubercolari
CN105085295A (zh) * 2014-05-23 2015-11-25 上海交通大学 氨甲环酸的两亲性衍生物及其用途
CN105085292B (zh) * 2014-05-23 2017-12-15 上海交通大学 3‑((2‑(二甲氨基)乙烷基)(甲基)氨基)丙酸的两亲性衍生物及其用途
US10022455B2 (en) 2014-05-30 2018-07-17 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
EP3164113B1 (en) 2014-06-04 2019-03-27 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
CA2952824C (en) 2014-06-24 2023-02-21 Shire Human Genetic Therapies, Inc. Stereochemically enriched compositions for delivery of nucleic acids
IL316479A (en) 2014-06-25 2024-12-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
EP3162794B1 (en) 2014-06-30 2020-09-16 Kyowa Kirin Co., Ltd. Alkyl substituted azetidines as cationic lipid
CA2953265C (en) 2014-07-02 2023-09-26 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
GB201414464D0 (en) 2014-08-14 2014-10-01 Technion Res & Dev Foundation Compositions and methods for therapeutics prescreening
WO2016028940A1 (en) 2014-08-19 2016-02-25 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
EP4137138A1 (en) 2014-08-29 2023-02-22 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (ttr) mediated amyloidosis
MA56412A (fr) 2014-12-05 2022-05-04 Translate Bio Inc Thérapie par l'arn messager pour le traitement des maladies articulaires
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
US11298326B2 (en) 2015-03-24 2022-04-12 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticles
CN107567497A (zh) 2015-04-17 2018-01-09 库瑞瓦格股份公司 Rna的冻干
JP2018526321A (ja) 2015-04-27 2018-09-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 適応免疫応答を誘導するためのヌクレオシド修飾rna
CA2984512A1 (en) 2015-05-06 2016-11-10 Benitec Biopharma Limited Reagents for treatment of hepatitis b virus (hbv) infection and use thereof
CN104803863B (zh) * 2015-05-08 2016-07-20 厦门成坤生物技术有限公司 阳离子类脂化合物及其制备方法
SG10201910431RA (en) 2015-05-20 2020-01-30 Curevac Ag Dry powder composition comprising long-chain rna
WO2016184576A2 (en) 2015-05-20 2016-11-24 Curevac Ag Dry powder composition comprising long-chain rna
ES2949540T3 (es) 2015-06-19 2023-09-29 Massachusetts Inst Technology 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
BR112018000254A2 (pt) 2015-07-07 2018-09-04 H Lundbeck As inibidores de pde9 com cadeia principal de imidazotriazinona e cadeia principal de imidazopirazinona para o tratamento de doenças periféricas
AU2016291228B2 (en) * 2015-07-09 2020-04-09 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
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
JP6896703B2 (ja) 2015-07-31 2021-06-30 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
RS63030B1 (sr) 2015-09-17 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava
IL258476B2 (en) 2015-10-02 2023-04-01 Complexa Inc Prevention, treatment and reversal of disease using therapeutically effective amounts of activated fatty acids
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
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
MD3718565T2 (ro) 2015-10-22 2022-09-30 Modernatx Inc Vaccinuri împotriva virusului respirator
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
WO2017070601A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (vzv)
AU2016342376A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Sexually transmitted disease vaccines
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
CA3007955A1 (en) 2015-12-10 2017-06-15 Modernatx, Inc. Lipid nanoparticles for delivery of therapeutic agents
CN106883158B (zh) * 2015-12-15 2019-08-23 中国科学院广州生物医药与健康研究院 生物可降解的氨基脂质类化合物及其制备方法和应用
JP7114465B2 (ja) 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
TW201726614A (zh) 2015-12-25 2017-08-01 Kyowa Hakko Kirin Co Ltd 作為陽離子性脂質之化合物
CN105622473B (zh) * 2016-02-06 2017-07-25 吴国球 一种阳离子氨基脂质及其合成方法和用途
US20190046638A1 (en) 2016-04-01 2019-02-14 Checkmate Pharmaceuticals, Inc. Fc RECEPTOR-MEDIATED DRUG DELIVERY
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
CN109219659B (zh) 2016-04-14 2022-09-09 贝尼泰克生物制药有限公司 用于治疗眼咽型肌营养不良(opmd)的试剂及其用途
JP7186094B2 (ja) 2016-05-06 2022-12-08 イグジキュア オペレーティング カンパニー インターロイキン17受容体mRNAの特異的ノックダウンのためのアンチセンスオリゴヌクレオチド(ASO)を提示するリポソーム系球状核酸(SNA)構築物
AU2017267634C1 (en) * 2016-05-16 2022-05-26 The Board Of Regents Of The University Of Texas System Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
US20170354672A1 (en) * 2016-05-16 2017-12-14 The Board Of Regents Of The University Of Texas System Compositions for the delivery of trna as nanoparticles and methods of use therewith
US12128113B2 (en) 2016-05-18 2024-10-29 Modernatx, Inc. Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome
JP7088911B2 (ja) 2016-05-18 2022-06-21 モデルナティエックス インコーポレイテッド リラキシンをコードするポリヌクレオチド
CN109312313A (zh) 2016-06-13 2019-02-05 川斯勒佰尔公司 用于治疗鸟氨酸转氨甲酰酶缺乏症的信使rna疗法
WO2017222016A1 (ja) 2016-06-24 2017-12-28 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
AU2017286980B2 (en) * 2016-06-30 2023-10-26 Arbutus Biopharma Corporation Compositions and methods for delivering messenger RNA
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
CA3025812A1 (en) 2016-08-19 2018-02-22 Curevac Ag Rna for cancer therapy
WO2018062233A1 (ja) 2016-09-27 2018-04-05 協和発酵キリン株式会社 カチオン性脂質としての化合物
WO2018062413A1 (ja) * 2016-09-28 2018-04-05 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
AU2017345766A1 (en) 2016-10-21 2019-05-16 Modernatx, Inc. Human cytomegalovirus vaccine
US20190314486A1 (en) 2016-10-21 2019-10-17 Merck Sharp & Dohme Corp. Influenza hemagglutinin protein vaccines
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
JP7560946B2 (ja) 2016-11-10 2024-10-03 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの皮下送達
MA46762A (fr) 2016-11-10 2019-09-18 Translate Bio Inc Procédé amélioré de préparation de nanoparticules lipidiques chargées d'arnm
CN110167587A (zh) 2016-11-11 2019-08-23 摩登纳特斯有限公司 流感疫苗
US11542490B2 (en) 2016-12-08 2023-01-03 CureVac SE RNAs for wound healing
EP3551193A4 (en) 2016-12-08 2020-08-19 Modernatx, Inc. NUCLEIC ACID VACCINES AGAINST RESPIRATORY VIRUSES
EP3808380A1 (en) 2016-12-08 2021-04-21 CureVac AG Rna for treatment or prophylaxis of a liver disease
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
US11464847B2 (en) 2016-12-23 2022-10-11 Curevac Ag Lassa virus vaccine
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
BR112019014314A2 (pt) 2017-01-11 2020-02-18 The Trustees Of The University Of Pennsylvania Composição, e, método de indução de uma resposta imune adaptativa contra vírus zika.
WO2018151816A1 (en) 2017-02-16 2018-08-23 Modernatx, Inc. High potency immunogenic compositions
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
WO2018165257A1 (en) 2017-03-07 2018-09-13 Translate Bio, Inc. Polyanionic delivery of nucleic acids
KR20190132405A (ko) 2017-03-15 2019-11-27 모더나티엑스, 인크. 치료제의 세포내 전달을 위한 화합물 및 조성물
CA3056133A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
ES3020935T3 (en) 2017-03-15 2025-05-23 Modernatx Inc Lipid nanoparticle formulation
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
MA47787A (fr) 2017-03-15 2020-01-22 Modernatx Inc Vaccin contre le virus respiratoire syncytial
WO2018170347A1 (en) 2017-03-17 2018-09-20 Modernatx, Inc. Zoonotic disease rna vaccines
US20200085944A1 (en) 2017-03-17 2020-03-19 Curevac Ag Rna vaccine and immune checkpoint inhibitors for combined anticancer therapy
CN110914433A (zh) 2017-03-24 2020-03-24 库尔维科公司 编码crispr相关蛋白质的核酸及其用途
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CA3061326A1 (en) 2017-04-27 2018-11-01 The Johns Hopkins University Nucleoside-modified mrna-lipid nanoparticle lineage vaccine for hepatitis c virus
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
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
US11939600B2 (en) 2017-05-31 2024-03-26 Arcturus Therapeutics, Inc. Compositions and methods for treating phenylketonuria
US11377643B2 (en) 2017-05-31 2022-07-05 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type III
WO2018222890A1 (en) 2017-05-31 2018-12-06 Arcturus Therapeutics, Inc. Synthesis and structure of high potency rna therapeutics
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
EP3641834B1 (en) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
BR112019028280A2 (pt) 2017-07-04 2020-07-14 Curevac Ag moléculas de ácido nucleico
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
EP3662913A4 (en) 2017-08-04 2021-06-30 Kyowa Kirin Co., Ltd. Nucleic-acid-containing lipid nanoparticles
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
EP4501322A3 (en) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
EP3673069A1 (en) 2017-08-22 2020-07-01 CureVac AG Bunyavirales vaccine
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
EP4219715A3 (en) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
US10653767B2 (en) 2017-09-14 2020-05-19 Modernatx, Inc. Zika virus MRNA vaccines
KR20200089656A (ko) 2017-09-19 2020-07-27 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
SG11202002186VA (en) 2017-10-19 2020-05-28 Curevac Ag Novel artificial nucleic acid molecules
US11692002B2 (en) 2017-11-08 2023-07-04 CureVac SE RNA sequence adaptation
EP3723796A1 (en) 2017-12-13 2020-10-21 CureVac AG Flavivirus vaccine
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
EP3733647B1 (en) 2017-12-27 2022-06-15 Eisai R&D Management Co., Ltd. Cationic lipid
MX2020006843A (es) 2017-12-27 2020-09-03 Takeda Pharmaceuticals Co Nanoparticula de lipido que contiene acido nucleico y uso de la misma.
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
WO2019148101A1 (en) 2018-01-29 2019-08-01 Modernatx, Inc. Rsv rna vaccines
JP7541482B2 (ja) 2018-01-29 2024-08-28 メルク・シャープ・アンド・ドーム・エルエルシー 安定化rsv fタンパク質およびその使用
WO2019152802A1 (en) 2018-02-02 2019-08-08 Translate Bio, Inc. Cationic polymers
WO2019155094A1 (en) * 2018-02-12 2019-08-15 Interna Technologies B.V. Anticancer microrna and lipid formulations thereof
EP3752194A4 (en) 2018-02-13 2022-03-16 Checkmate Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR TUMOR IMMUNOTHERAPY
WO2019191627A1 (en) 2018-03-30 2019-10-03 Insmed Incorporated Methods for continuous manufacture of liposomal drug products
WO2019193183A2 (en) 2018-04-05 2019-10-10 Curevac Ag Novel yellow fever nucleic acid molecules for vaccination
SG11202009647VA (en) 2018-04-09 2020-10-29 Checkmate Pharmaceuticals Packaging oligonucleotides into virus-like particles
EP4242307A3 (en) 2018-04-12 2023-12-27 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
DE202019006199U1 (de) 2018-04-17 2026-01-12 CureVac SE Neue RSV-RNA-Moleküle und Zusammensetzungen für die Impfung
WO2019213398A1 (en) 2018-05-02 2019-11-07 Insmed Incorporated Methods for the manufacture of liposomal drug formulations
AU2019271132B2 (en) 2018-05-15 2026-02-05 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
US11964051B2 (en) 2018-05-16 2024-04-23 Translate Bio, Inc. Ribose cationic lipids
AU2019275068B2 (en) 2018-05-24 2024-09-26 Translate Bio, Inc. Thioester cationic lipids
EP3801526B1 (en) 2018-05-25 2023-12-27 Imara Inc. Monohydrate and crystalline forms of 6-[(3s,4s)-4-methyl-1- (pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3-tetrahydropyran-4-yl- 7h-imid azo [1,5- a] pyrazin-8-one
WO2019232095A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Vitamin cationic lipids
US11547666B2 (en) 2018-05-30 2023-01-10 Translate Bio, Inc. Cationic lipids comprising a steroidal moiety
AU2019277361B2 (en) 2018-05-30 2025-04-24 Translate Bio, Inc. Messenger RNA vaccines and uses thereof
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
US20210260178A1 (en) 2018-06-27 2021-08-26 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
KR20210056331A (ko) 2018-07-23 2021-05-18 트랜슬레이트 바이오 인코포레이티드 전령 rna의 건조 분말 제형
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
KR20210091120A (ko) 2018-08-29 2021-07-21 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 공정
CN112996512B (zh) 2018-08-31 2025-01-03 卡都瑞恩医药公司 用于治疗镰状细胞病的pde9抑制剂
US12564645B2 (en) 2018-09-14 2026-03-03 Translate Bio, Inc. Methods for treatment of methylmalonic acidemia
US12151029B2 (en) 2018-09-19 2024-11-26 Modernatx, Inc. PEG lipids and uses thereof
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
EP3860561B1 (en) 2018-10-01 2023-06-21 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
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
CN119876276A (zh) 2018-10-18 2025-04-25 武田药品工业株式会社 T细胞的活化/增殖方法
US12583816B2 (en) 2018-10-18 2026-03-24 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
KR20210090634A (ko) 2018-10-19 2021-07-20 트랜슬레이트 바이오 인코포레이티드 전령 rna의 무펌프 캡슐화
EA202191315A1 (ru) * 2018-11-09 2021-09-08 Арбутус Биофарма Корпорэйшн Катионные липиды, содержащие кремний
EP3876914A2 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
AU2019374871B2 (en) 2018-11-09 2025-09-11 Translate Bio, Inc. PEG lipidoid compounds
WO2020097376A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
KR102860605B1 (ko) 2018-11-09 2025-09-15 트랜슬레이트 바이오 인코포레이티드 에스테르, 티오에스테르, 이황화물, 및 무수물 모이어티가 삽입된 2,5-디옥소피페라진
AU2019378763B2 (en) 2018-11-12 2025-06-26 Translate Bio, Inc. Methods for inducing immune tolerance
EP3883917B1 (en) 2018-11-21 2024-01-24 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
EP3883592A1 (en) 2018-11-21 2021-09-29 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
PL3891274T3 (pl) 2018-12-06 2024-03-25 Arcturus Therapeutics, Inc. Kompozycje i sposoby leczenia niedoboru transkarbamylazy ornityny
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
CN113453707A (zh) 2018-12-21 2021-09-28 库瑞瓦格股份公司 用于疟疾疫苗的rna
CN113412273B (zh) 2019-01-07 2025-08-22 川斯勒佰尔公司 用于治疗原发性纤毛运动障碍的组合物和方法
JP7523449B2 (ja) 2019-01-11 2024-07-26 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
MX2021009245A (es) * 2019-01-31 2021-11-12 Modernatx Inc Metodos de preparacion de nanoparticulas lipidicas.
EP4491229A3 (en) 2019-02-08 2025-05-14 CureVac SE 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
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
US20220204994A1 (en) 2019-04-05 2022-06-30 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
WO2020208361A1 (en) 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
ES2972014T3 (es) 2019-04-22 2024-06-10 Translate Bio Inc Lípidos catiónicos de tioéster
US12453759B2 (en) 2019-05-03 2025-10-28 Translate Bio, Inc. Di-thioester cationic lipids
EP3968952A1 (en) 2019-05-14 2022-03-23 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
JP7686574B2 (ja) 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状rna組成物及び方法
WO2020243540A1 (en) 2019-05-31 2020-12-03 Translate Bio, Inc. Macrocyclic lipids
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
AU2020297606B2 (en) 2019-06-21 2026-04-02 Translate Bio, Inc. Tricine and citric acid lipids
JP7699552B2 (ja) * 2019-07-03 2025-06-27 ファクター バイオサイエンス インコーポレイテッド カチオン性脂質及びその使用
ES2992127T3 (es) 2019-07-08 2024-12-09 Translate Bio Inc Nanopartículas lipídicas cargadas de ARNm mejoradas y sus procesos de fabricación
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
JP7630904B2 (ja) * 2019-07-23 2025-02-18 株式会社東芝 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法
WO2021016430A1 (en) 2019-07-23 2021-01-28 Translate Bio, Inc. Stable compositions of mrna-loaded lipid nanoparticles and processes of making
EP4003397B1 (en) 2019-07-30 2025-02-26 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
JP2022544412A (ja) 2019-08-14 2022-10-18 キュアバック アーゲー 免疫賦活特性が減少したrna組み合わせおよび組成物
MX2022003269A (es) 2019-09-19 2022-07-04 Modernatx Inc Compuestos lipidicos de cola ramificada y composiciones para la administracion intracelular de agentes terapeuticos.
CA3155015A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
AU2020352552A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
CN114391040A (zh) 2019-09-23 2022-04-22 欧米茄治疗公司 用于调节载脂蛋白b(apob)基因表达的组合物和方法
JP7650873B2 (ja) 2019-10-09 2025-03-25 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの組成物、方法および使用
US20220396556A1 (en) * 2019-10-18 2022-12-15 The Trustees Of The University Of Pennsylvania Lipid and Lipid Nanoparticle Formulation for Drug Delivery
US20210145860A1 (en) 2019-10-21 2021-05-20 Translate Bio, Inc. Compositions, methods and uses of messenger rna
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
CN114901360A (zh) 2019-12-20 2022-08-12 库瑞瓦格股份公司 用于递送核酸的新型脂质纳米颗粒
US12053551B2 (en) 2019-12-20 2024-08-06 Translate Bio, Inc. Process of preparing mRNA-loaded lipid nanoparticles
KR20220142432A (ko) 2019-12-20 2022-10-21 트랜슬레이트 바이오 인코포레이티드 메신저 rna의 직장 전달
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
EP3886897A1 (en) 2020-02-04 2021-10-06 CureVac AG Coronavirus vaccine
BR112022015313A2 (pt) 2020-02-14 2022-09-27 Merck Sharp & Dohme Llc Vacina de hpv
WO2021173840A1 (en) 2020-02-25 2021-09-02 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
WO2021178510A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
CA3171231A1 (en) 2020-03-09 2021-09-16 Sean Michael Sullivan Compositions and methods for inducing immune responses
WO2021183720A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021195218A1 (en) 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
AU2021252164A1 (en) 2020-04-09 2022-12-15 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
CA3176844A1 (en) 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
WO2021226436A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Optimized nucleotide sequences encoding sars-cov-2 antigens
US12605399B2 (en) 2020-05-07 2026-04-21 Translate Bio, Inc. Compositions for CFTR MRNA therapy
EP4146680A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
US12514932B2 (en) 2020-05-14 2026-01-06 Translate Bio, Inc. PEG lipidoid compounds
EP4149425A1 (en) 2020-05-15 2023-03-22 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
US20240042015A1 (en) 2020-05-19 2024-02-08 Orna Therapeutics, Inc. Circular rna compositions and methods
TW202206097A (zh) 2020-05-20 2022-02-16 美商旗艦先鋒創新有限責任公司 冠狀病毒抗原組成物及其用途
EP4153223A1 (en) 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
CN116322758A (zh) 2020-05-29 2023-06-23 库尔维科欧洲股份公司 基于核酸的组合疫苗
CA3182026A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
IL298539A (en) 2020-05-29 2023-01-01 Flagship Pioneering Innovations Vi Llc Trem compositions and methods relating thereto
US20240307556A1 (en) 2020-07-02 2024-09-19 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
CA3188801A1 (en) 2020-07-08 2022-01-13 Janssen Sciences Ireland Unlimited Company Rna replicon vaccines against hbv
JP7798855B2 (ja) 2020-07-16 2026-01-14 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子に使用するためのカチオン性脂質
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
EP4172194A1 (en) 2020-07-31 2023-05-03 CureVac SE Nucleic acid encoded antibody mixtures
TW202220637A (zh) * 2020-08-06 2022-06-01 美商現代公司 製備脂質奈米顆粒之方法
CN116171150A (zh) 2020-08-14 2023-05-26 阿克丘勒斯治疗公司 冻干脂质纳米颗粒的方法
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
CA3170743A1 (en) 2020-08-31 2022-03-03 Susanne RAUCH Multivalent nucleic acid based coronavirus vaccines
EP4208196A1 (en) 2020-09-03 2023-07-12 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
EP4211247A4 (en) 2020-09-13 2024-11-13 Arcturus Therapeutics, Inc. ENCAPSULATION OF LARGE RNA IN LIPID NANOPARTICLES
WO2022076562A1 (en) 2020-10-06 2022-04-14 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022076547A1 (en) 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions
US20220133631A1 (en) 2020-10-12 2022-05-05 Translate Bio, Inc. Process of preparing ice-based lipid nanoparticles
US20220110884A1 (en) 2020-10-12 2022-04-14 Translate Bio, Inc. Process of preparing mrna-loaded lipid nanoparticles
KR20230118715A (ko) 2020-10-14 2023-08-11 조지 메이슨 리서치 파운데이션 인코포레이티드 이온화 가능 지질 및 이의 제조 및 사용 방법
AU2021373892A1 (en) 2020-11-09 2023-07-06 Translate Bio, Inc. Improved compositions for delivery of codon-optimized mrna
CA3200234A1 (en) 2020-11-25 2022-06-02 Daryl C. Drummond Lipid nanoparticles for delivery of nucleic acids, and related methods of use
CN116723829A (zh) 2020-11-25 2023-09-08 翻译生物公司 稳定的液体脂质纳米颗粒配制品
PH12023500013A1 (en) 2020-12-04 2024-03-11 Tidal Therapeutics Inc Ionizable cationic lipids and lipi nanoparticles, and methods of synthesis and use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
US20240299309A1 (en) 2020-12-22 2024-09-12 CureVac SE Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
US20240156946A1 (en) 2020-12-22 2024-05-16 CureVac SE Rna vaccine against sars-cov-2 variants
BR112023012377A2 (pt) 2020-12-23 2023-10-24 Flagship Pioneering Innovations Vi Llc Composições de trems modificadas e usos das mesmas
WO2022146654A1 (en) 2020-12-28 2022-07-07 Janssen Pharmaceuticals, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
JP2024504614A (ja) 2021-01-14 2024-02-01 トランスレイト バイオ, インコーポレイテッド mRNAがコードする抗体を送達する方法および組成物
EP4087938A2 (en) 2021-01-27 2022-11-16 CureVac AG 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
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022204549A1 (en) 2021-03-25 2022-09-29 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
CA3214137A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
MX2023011400A (es) 2021-03-26 2023-10-09 Glaxosmithkline Biologicals Sa Composiciones inmunogenicas.
US20220325287A1 (en) 2021-03-31 2022-10-13 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
WO2022207862A2 (en) 2021-03-31 2022-10-06 Curevac Ag Syringes containing pharmaceutical compositions comprising rna
EP4323770A1 (en) * 2021-04-12 2024-02-21 Institut Curie Folr2+ macrophages and anti-tumor immunity
WO2022225918A1 (en) 2021-04-19 2022-10-27 Translate Bio, Inc. Improved compositions for delivery of mrna
US20240358852A1 (en) 2021-04-20 2024-10-31 Anjarium Biosciences Ag Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022232289A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4334446A1 (en) 2021-05-03 2024-03-13 CureVac SE Improved nucleic acid sequence for cell type specific expression
EP4347577A4 (en) * 2021-05-28 2025-09-03 Nanovation Therapeutics Inc KC2-TYPE LIPIDS
KR20220167770A (ko) 2021-06-11 2022-12-21 주식회사 나이벡 타겟 세포 내로 올리고뉴클레오티드를 전달하기 위한 펩타이드-지질의 결합체를 포함하는 나노입자 및 이를 포함하는 약학적 조성물
JP2024527541A (ja) 2021-07-01 2024-07-25 トランスレイト バイオ, インコーポレイテッド Mrnaの送達のための組成物
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
US20240342206A1 (en) 2021-07-30 2024-10-17 CureVac SE mRNAS FOR TREATMENT OR PROPHYLAXIS OF LIVER DISEASES
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
TW202328445A (zh) 2021-08-03 2023-07-16 美商艾拉倫製藥股份有限公司 甲狀腺素運載蛋白(TTR)iRNA組成物及其使用方法
CA3229064A1 (en) 2021-08-19 2023-02-23 Andrew Bett Thermostable lipid nanoparticle and methods of use thereof
CN113461577B (zh) * 2021-09-01 2021-12-14 中山大学附属第七医院(深圳) 一种氨基脂质及其应用
CN115745942B (zh) * 2021-09-03 2025-06-10 广州谷森制药有限公司 阳离子脂质化合物
IL309505A (en) 2021-09-03 2024-02-01 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
CN117940158A (zh) 2021-09-03 2024-04-26 库瑞瓦格欧洲公司 用于核酸递送的包含磷脂酰丝氨酸的新型脂质纳米颗粒
CN115745941B (zh) * 2021-09-03 2025-07-01 广州谷森制药有限公司 阳离子脂质化合物
CN115772089B (zh) * 2021-09-07 2025-06-10 广州谷森制药有限公司 阳离子脂质化合物
DK4271818T3 (da) 2021-09-17 2024-11-04 Flagship Pioneering Innovations Vi Llc Sammensætninger og fremgangsmåder til fremstilling af cirkulære polyribonukleotider
US20240417714A1 (en) 2021-10-18 2024-12-19 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
CN118541346A (zh) 2021-11-08 2024-08-23 奥纳治疗公司 用于递送环状多核苷酸的脂质纳米颗粒组合物
AU2022388747A1 (en) 2021-11-10 2024-06-20 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
CN118900845A (zh) 2021-11-24 2024-11-05 旗舰创业创新六公司 冠状病毒免疫原组合物及其用途
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
CA3238370A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Varicella-zoster virus immunogen compositions and their uses
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
MX2024007307A (es) 2021-12-17 2024-08-06 Flagship Pioneering Innovations Vi Llc Métodos de enriquecimiento de arn circular en condiciones desnaturalizantes.
AU2022417517A1 (en) 2021-12-22 2024-06-27 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
MX2024007870A (es) 2021-12-23 2024-08-15 Flagship Pioneering Innovations Vi Llc Polirribonucleotidos circulares que codifican polipeptidos antifusogenicos.
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Viii因子をコードするdna分子の組成物、それを作製する方法、及びその使用方法
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
AU2023235112A1 (en) 2022-03-14 2024-10-17 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
EP4499607A1 (en) 2022-03-25 2025-02-05 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
US20250205168A1 (en) * 2022-03-28 2025-06-26 Nof Corporation Method for producing nucleic acid-encapsulated lipid nanoparticles, method for producing pharmaceutical composition containing said lipid nanoparticles, and method for introducing nucleic acid into cell or target cell
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
WO2023196634A2 (en) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
WO2023214405A1 (en) 2022-05-01 2023-11-09 Yeda Research And Development Co. Ltd. Reexpression of hnf4a to alleviate cancer-associated cachexia
US20250295687A1 (en) 2022-05-09 2025-09-25 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
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
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
CA3248209A1 (en) 2022-05-25 2023-11-30 CureVac SE Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH)
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
AU2023283348A1 (en) 2022-06-07 2025-01-09 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
AR129560A1 (es) 2022-06-08 2024-09-04 Tidal Therapeutics Inc Lípidos catiónicos ionizables y nanopartículas lipídicas, y métodos de síntesis y uso de los mismos
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN120051297A (zh) 2022-06-30 2025-05-27 因达普塔治疗公司 工程化自然杀伤(nk)细胞与抗体疗法的组合及相关方法
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
AU2023320333A1 (en) 2022-08-01 2025-01-16 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
WO2024035952A1 (en) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024040195A1 (en) 2022-08-17 2024-02-22 Capstan Therapeutics, Inc. Conditioning for in vivo immune cell engineering
CN120435557A (zh) 2022-08-19 2025-08-05 图恩疗法股份有限公司 通过靶向基因阻遏调节乙型肝炎病毒的组合物、系统和方法
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
EP4581012A2 (en) 2022-08-31 2025-07-09 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
JPWO2024053648A1 (enExample) 2022-09-06 2024-03-14
WO2024064642A2 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
CA3267272A1 (en) 2022-09-26 2024-04-04 Glaxosmithkline Biologicals Sa FLU VIRUS VACCINES
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
CA3266203A1 (en) * 2022-10-25 2024-05-02 Nanovation Therapeutics Inc. LIPID NANOPARTICLE FORMULATIONS FOR ADMINISTRATION OF ANTISENSE OLIGONUCLEOTIDES
EP4608442A1 (en) 2022-10-28 2025-09-03 GlaxoSmithKline Biologicals S.A. Nucleic acid based vaccine
EP4612296A1 (en) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
TW202434728A (zh) 2022-11-08 2024-09-01 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的組成物和方法
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
TW202425959A (zh) 2022-11-08 2024-07-01 美商歐納醫療公司 遞送多核苷酸的脂質及奈米顆粒組合物
EP4622630A1 (en) 2022-11-21 2025-10-01 Translate Bio, Inc. Compositions of dry powder formulations of messenger rna and methods of use thereof
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
IL320873A (en) 2022-12-01 2025-07-01 Generation Bio Co Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions containing them
JP2025541726A (ja) 2022-12-01 2025-12-23 ジェネレーション バイオ カンパニー 細胞標的化のためのステルス脂質ナノ粒子組成物
JP2025540076A (ja) 2022-12-01 2025-12-11 ジェネレーション バイオ カンパニー 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用
WO2024123134A1 (ko) 2022-12-09 2024-06-13 서울대학교산학협력단 B 세포 및 t 세포 내로 mrna를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이의 용도
KR20240087591A (ko) 2022-12-09 2024-06-19 주식회사 나이벡 타겟 세포 내로 올리고뉴클레오티드를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이를 포함하는 약학적 조성물
WO2024121814A1 (en) 2022-12-09 2024-06-13 Takeda Pharmaceutical Company Limited Modified immunomodulators
WO2024129988A1 (en) 2022-12-14 2024-06-20 Flagship Pioneering Innovations Vii, Llc Compositions and methods for delivery of therapeutic agents to bone
WO2024125597A1 (en) 2022-12-14 2024-06-20 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
WO2024126809A1 (en) 2022-12-15 2024-06-20 Sanofi Mrna encoding influenza virus-like particle
EP4634390A2 (en) 2022-12-15 2025-10-22 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4637813A1 (en) 2022-12-20 2025-10-29 Sanofi Pasteur Rhinovirus mrna vaccine
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
JP2026504815A (ja) 2022-12-29 2026-02-10 ポップヴァックス プライベート リミテッド マルチターゲットワクチンおよび治療剤
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
US12410394B2 (en) * 2023-01-06 2025-09-09 Athergen, Inc. Composition comprising nicotinamide mononucleotide and leucomethylene blue
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
TW202438673A (zh) 2023-01-09 2024-10-01 美商旗艦先鋒創新有限責任(Vii)公司 疫苗及相關方法
US20240269251A1 (en) 2023-01-09 2024-08-15 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151685A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
EP4658239A1 (en) 2023-02-03 2025-12-10 GlaxoSmithKline Biologicals S.A. Rna formulation
WO2024163905A1 (en) 2023-02-03 2024-08-08 Genzyme Corporation Hsc-specific antibody conjugated lipid nanoparticles and uses thereof
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
CN120813562A (zh) * 2023-02-09 2025-10-17 斯奇络斯公司 可离子化阳离子化合物
EP4665736A2 (en) 2023-02-13 2025-12-24 Flagship Pioneering Innovations VII, LLC Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
JP2026507514A (ja) 2023-02-17 2026-03-04 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 修飾されたシトシンを含むdna組成物
AU2024222598A1 (en) 2023-02-17 2025-07-31 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified uracil
JP2026509258A (ja) 2023-03-08 2026-03-17 キュアバック エスイー 核酸送達のための新規な脂質ナノ粒子製剤
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
JP2026512425A (ja) 2023-03-30 2026-04-16 オーエスイー・イミュノセラピューティクス 免疫細胞増強分子の発現のための活性化免疫細胞に標的化された脂質ベースのナノ粒子及びその使用
EP4687990A1 (en) 2023-03-30 2026-02-11 Ose Immunotherapeutics Method of synthesis of targeted lipid nanoparticle and uses thereof
AR132276A1 (es) 2023-04-07 2025-06-11 Takeda Pharmaceuticals Co Complejo de conjugación
JP2026514004A (ja) 2023-04-12 2026-05-01 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー 修飾されたtrem、組成物及びその関連方法
EP4695391A1 (en) 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Trems for use in correction of missense mutations
MX2025012337A (es) 2023-04-17 2026-02-03 Sanofi Pasteur Inc Formulaciones de polvo seco reconstituibles y métodos de uso de las mismas
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024223728A1 (en) 2023-04-27 2024-10-31 Glaxosmithkline Biologicals Sa Influenza virus vaccines
EP4701657A1 (en) 2023-04-27 2026-03-04 GlaxoSmithKline Biologicals S.A. Influenza virus vaccines
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
JP2026505939A (ja) 2023-05-08 2026-02-20 メルク・シャープ・アンド・ドーム・エルエルシー ノロウイルスvp1抗原をコードするポリヌクレオチドおよびその使用
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
WO2024236504A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Sequences and methods for delivery of dna and rna
WO2024236361A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Compositions and methods for delivery of nucleic acids to cells
CN116270543B (zh) * 2023-05-19 2023-09-26 清华大学 脂质纳米颗粒在制备通过雾吸或鼻滴给药递送核酸的药物中的用途
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
TW202513091A (zh) 2023-06-09 2025-04-01 美商默沙東有限責任公司 用於人類乳突病毒疫苗之奈米乳液佐劑組合物
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006799A1 (en) 2023-06-27 2025-01-02 Capstan Therapeutics, Inc. Extracorporeal and ex vivo engineering of select cell populations from peripheral blood
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
TW202517669A (zh) 2023-07-05 2025-05-01 日商武田藥品工業股份有限公司 用於A型血友病之基因療法的編碼表現增加之重組FVlll變異體的病毒載體
WO2025012756A1 (en) 2023-07-07 2025-01-16 Pfizer Inc. Amphiphilic tlr7/8 adjuvants and uses thereof
CN121532514A (zh) 2023-07-07 2026-02-13 上海环码生物医药有限公司 用于季节性流感的环状rna疫苗及使用方法
US20250092426A1 (en) 2023-07-25 2025-03-20 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
US20250243274A1 (en) * 2023-07-31 2025-07-31 Jibe Therapeutics, Inc. Compositions for Redirecting Immunoglobulins to Immune Cells
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
CN121620589A (zh) 2023-08-01 2026-03-06 法国居里学院 包含编码环gmp-amp合酶的核酸序列的纳米粒子
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025045142A1 (en) 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
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
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
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
CN121909024A (zh) * 2023-09-22 2026-04-21 国立大学法人东北大学 包封有核酸的配体修饰脂质纳米颗粒的制造方法
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025106670A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任(Vii)公司 用於治療非酒精性脂肪性肝病之方法及組成物
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025133115A1 (en) 2023-12-21 2025-06-26 Ose Immunotherapeutics Lipid-based nanoparticles comprising il-35
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫受體抑制蛋白及相關方法
TW202543581A (zh) 2024-02-12 2025-11-16 美商艾伊拉醫療公司 遞送組成物
WO2025180874A1 (en) * 2024-02-27 2025-09-04 Basf Se Substituted 1,3-dioxolane sulfates and their use
WO2025187760A1 (ja) * 2024-03-07 2025-09-12 日東電工株式会社 脂質ナノ粒子
WO2025190300A1 (zh) * 2024-03-13 2025-09-18 仁景(苏州)生物科技有限公司 脂质纳米颗粒及脂质纳米颗粒组合物
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025196065A1 (en) 2024-03-20 2025-09-25 Sanofi Novel homocysteine based lipids and their use for delivery of nucleic acids
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
CN118388360B (zh) 2024-04-17 2025-04-04 北京悦康科创医药科技股份有限公司 含有苯环结构的长效脾靶向阳离子脂质化合物、包含其的组合物及用途
WO2025240680A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2025252759A1 (en) 2024-06-03 2025-12-11 Bio-Sourcing Transient expression by lipid nanoparticle formulations
WO2025259931A1 (en) 2024-06-14 2025-12-18 Orbital Therapeutics, Inc. Compositions and methods for rna circularization
WO2026006203A2 (en) 2024-06-24 2026-01-02 Orbital Therapeutics, Inc. Compositions and methods for making circular rna
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026020171A2 (en) 2024-07-19 2026-01-22 Aera Therapeutics, Inc. Chimeric antigen receptor constructs
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026033123A1 (en) 2024-08-08 2026-02-12 Sanofi Pasteur Inc. Lipid nanoparticle formulations for mrna delivery
WO2026055543A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Modified dna compositions and related methods
WO2026055547A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2026064313A1 (en) 2024-09-17 2026-03-26 Flagship Pioneering Innovations Vii, Llc Rna compositions and related methods
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026072557A2 (en) 2024-09-24 2026-04-02 Genzyme Corporation Ionizable cationic lipids and lipid nanoparticles, and methods of synthesis and use thereof
WO2026068705A1 (en) 2024-09-26 2026-04-02 Ose Immunotherapeutics Lipid-based nanoparticles comprising non-glycosylated fc domains and uses thereof
WO2026085300A1 (en) 2024-10-16 2026-04-23 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
WO2026088119A1 (en) 2024-10-24 2026-04-30 Pfizer Inc. Lipidated tlr7/8 modulators as adjuvants and uses thereof
WO2026088120A1 (en) 2024-10-24 2026-04-30 Pfizer Inc. Lipidated tlr7/8 modulators as adjuvants and uses thereof
WO2026093414A1 (en) 2024-10-30 2026-05-07 Ose Immunotherapeutics Lipid-based nanoparticles comprising membrane il-35
WO2026093616A1 (en) 2024-11-04 2026-05-07 Variant Nucleic acid encoding cone-rod homeobox (crx) protein, and expression vectors thereof, for use in treating a ciliopathy

Family Cites Families (230)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4394448A (en) 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
EP0032578B1 (de) 1980-01-16 1984-07-25 Hans Georg Prof. Dr. Weder Verfahren und Dialysiereinrichtung zur Herstellung von Bilayer-Vesikeln und Verwendung der Bilayer-Vesikel
US4598051A (en) 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
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
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5545412A (en) 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
WO1988000824A1 (en) * 1986-07-28 1988-02-11 Liposome Technology, Inc. Liposomes with enhanced retention on mucosal tissue
US4839175A (en) * 1986-07-28 1989-06-13 Liposome Technology, Inc. Liposomes with enhanced retention on mucosal tissue
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
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
DE3851889T2 (de) 1987-06-24 1995-04-13 Florey Howard Inst Nukleosid-derivate.
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
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
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
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
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5225212A (en) 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
DK0942000T3 (da) 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US6465188B1 (en) 1990-06-11 2002-10-15 Gilead Sciences, Inc. Nucleic acid ligand complexes
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
DE69126530T2 (de) 1990-07-27 1998-02-05 Isis Pharmaceutical, Inc., Carlsbad, Calif. Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
JPH06502300A (ja) 1990-08-03 1994-03-17 サノフィ 遺伝子発現の抑制のための化合物及び方法
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
EP0549686A4 (en) 1990-09-20 1995-01-18 Gilead Sciences Inc Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
WO1992007065A1 (en) 1990-10-12 1992-04-30 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modified ribozymes
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
DE4120916A1 (de) * 1991-06-25 1993-01-07 Basf Ag Omega-3 mehrfach ungesaettigte fettsaeurederivate, ihre herstellung und verwendung
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
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
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
WO1993017125A1 (en) 1992-02-19 1993-09-02 Baylor College Of Medicine Oligonucleotide modulation of cell growth
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
WO1993023569A1 (en) 1992-05-11 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
WO1993024640A2 (en) 1992-06-04 1993-12-09 The Regents Of The University Of California Methods and compositions for in vivo gene therapy
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
WO1994022864A1 (en) 1993-03-30 1994-10-13 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
CA2159629A1 (en) 1993-03-31 1994-10-13 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5578475A (en) 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5532130A (en) 1993-07-20 1996-07-02 Dyad Pharmaceutical Corporation Methods and compositions for sequence-specific hybridization of RNA by 2'-5' oligonucleotides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
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
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
JPH10506093A (ja) 1994-06-22 1998-06-16 メガバイオス・コーポレイション カチオン性両親媒性化合物
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
WO1996010585A1 (en) 1994-09-30 1996-04-11 Inex Pharmaceuticals Corp. Glycosylated protein-liposome conjugates and methods for their preparation
AU3889595A (en) 1994-10-05 1996-05-02 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
JP3583505B2 (ja) * 1995-05-10 2004-11-04 花王株式会社 新規な第4級アンモニウム塩及びそれを含有する柔軟剤組成物
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
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
CA2223179A1 (en) 1995-06-07 1996-12-19 Bob Dale Brown Phosphonic acid-based cationic lipids
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
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
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
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
EP0874910A4 (en) 1995-06-07 1999-04-21 Life Technologies Inc ENHANCED CATIONIC LIPID TRANSFECTION BY PEPTIDES
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
CA2227373A1 (en) 1995-07-21 1997-02-06 Promega Biosciences, Inc. Novel amide-based cationic lipids
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
US6743171B1 (en) 1997-04-02 2004-06-01 Henry M. Bowles Relocatable mental function testing gateway
US6235310B1 (en) * 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
WO1998051278A2 (en) 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
WO1999005303A1 (en) 1997-07-24 1999-02-04 Inex Pharmaceuticals Corporation Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
EP1829856A3 (en) 1998-11-12 2009-02-25 Invitrogen Corporation Transfection reagents
US6656498B1 (en) * 1998-11-25 2003-12-02 Vanderbilt University Cationic liposomes for gene transfer
US6649780B1 (en) 1998-12-22 2003-11-18 Valentis, Inc. Cationic lipids
RU2148349C1 (ru) * 1999-01-05 2000-05-10 Всероссийский научно-исследовательский институт консервной и овощесушильной промышленности Способ производства комбикормового премикса
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
US7094423B1 (en) 1999-07-15 2006-08-22 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
ES2235916T3 (es) 1999-07-15 2005-07-16 Inex Pharmaceuticals Corp. Preparacion de agentes terapeuticos de encapsulacion lipidica.
CN100368020C (zh) 1999-08-27 2008-02-13 不列颠哥伦比亚大学 用于刺激细胞因子分泌和诱导免疫应答的组合物
US20030229037A1 (en) * 2000-02-07 2003-12-11 Ulrich Massing Novel cationic amphiphiles
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US6543484B1 (en) 2000-08-15 2003-04-08 Roderick Highsmith Waste water disposal system
JP2004511572A (ja) 2000-10-25 2004-04-15 ザ ユニバーシティ オブ ブリティッシュ コロンビア 標的化送達のための脂質製剤
WO2002034879A2 (en) 2000-10-27 2002-05-02 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
ES2307568T3 (es) 2000-12-08 2008-12-01 Coley Pharmaceutical Gmbh Acidos nucleicos de tipo cpg y metodos de uso de los mismos.
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20040142892A1 (en) 2001-04-30 2004-07-22 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
US7223887B2 (en) 2001-12-18 2007-05-29 The University Of British Columbia Multivalent cationic lipids and methods of using same in the production of lipid particles
EP1509203B1 (en) 2002-05-15 2016-04-13 California Pacific Medical Center Delivery of nucleic acid-like compounds
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
ATE477337T1 (de) 2003-01-16 2010-08-15 Univ Pennsylvania Zusammensetzungen und verfahren zur sirna-hemmung von icam-1
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
AU2004246904B2 (en) 2003-06-18 2010-02-04 Biolab Ltd. Sphingolipids polyalkylamine conjugates for use in transfection
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
CN1882693B (zh) 2003-09-15 2012-08-15 普洛体维生物治疗公司 聚乙二醇修饰的脂质化合物及其应用
ATE536418T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1773857A4 (en) 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc THE IMMUNE SYSTEM STIMULATING SIRNA MOLECULES AND APPLICATIONS THEREOF
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
NZ572403A (en) 2004-09-24 2010-03-26 Alnylam Pharmaceuticals Inc Rnai modulation of apob and uses thereof
US20060134189A1 (en) 2004-11-17 2006-06-22 Protiva Biotherapeutics, Inc siRNA silencing of apolipoprotein B
WO2006074546A1 (en) 2005-01-13 2006-07-20 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US20060228406A1 (en) 2005-03-17 2006-10-12 Invitrogen Corporation Transfection reagent for non-adherent suspension cells
EP2476756A1 (en) * 2005-06-15 2012-07-18 Massachusetts Institute of Technology Amine-containing lipids and uses thereof
CN103989633A (zh) 2005-07-27 2014-08-20 普洛体维生物治疗公司 制造脂质体的系统和方法
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
WO2008042973A2 (en) * 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
JP5749494B2 (ja) * 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション 核酸の送達のための改善された組成物および方法
US20110097720A1 (en) 2008-01-02 2011-04-28 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
NZ588583A (en) * 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
AU2009236219B8 (en) * 2008-04-15 2015-06-25 Arbutus Biopharma Corporation Silencing of CSN5 gene expression using interfering RNA
WO2009132131A1 (en) * 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
WO2010036962A1 (en) * 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
JP5777519B2 (ja) * 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
EP4241767B1 (en) 2008-11-10 2024-10-23 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
CA2760706C (en) 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
EP2506879A4 (en) 2009-12-01 2014-03-19 Protiva Biotherapeutics Inc PREPARATIONS OF SNALP CONTAINING ANTIOXIDANTS
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141703A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
JP2014508102A (ja) 2010-11-15 2014-04-03 ライフ テクノロジーズ コーポレーション アミン含有トランスフェクション試薬ならびにそれを生成および使用するための方法

Also Published As

Publication number Publication date
EP3225621A1 (en) 2017-10-04
PL2350043T3 (pl) 2014-09-30
EP2350043A1 (en) 2011-08-03
CN102245590A (zh) 2011-11-16
WO2010042877A1 (en) 2010-04-15
CN104119242A (zh) 2014-10-29
JP2015028054A (ja) 2015-02-12
EP2743265B1 (en) 2017-03-15
JP2016196508A (ja) 2016-11-24
HK1160459A1 (en) 2012-08-17
EP2743265A1 (en) 2014-06-18
JP5777519B2 (ja) 2015-09-09
CA2984026A1 (en) 2010-04-15
US20110256175A1 (en) 2011-10-20
AU2009303345A1 (en) 2010-04-15
CN104119242B (zh) 2017-07-07
JP6505912B2 (ja) 2019-04-24
CA2984026C (en) 2020-02-11
EP2350043B9 (en) 2014-08-20
JP2012505250A (ja) 2012-03-01
CA2740000C (en) 2017-12-12
AU2009303345B2 (en) 2015-08-20
EP2350043B1 (en) 2014-03-26
JP2019137688A (ja) 2019-08-22
CA2740000A1 (en) 2010-04-15
US20160095924A1 (en) 2016-04-07
CN102245590B (zh) 2014-03-19
EP2350043A4 (en) 2012-06-13
US10653780B2 (en) 2020-05-19
JP6077509B2 (ja) 2017-02-08
US9139554B2 (en) 2015-09-22
JP2018119016A (ja) 2018-08-02

Similar Documents

Publication Publication Date Title
JP6505912B2 (ja) 改良されたアミノ脂質および核酸の送達方法
JP5749494B2 (ja) 核酸の送達のための改善された組成物および方法
AU2015210364B2 (en) Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) Processes for preparing lipids
AU2019250141A1 (en) Methods of delivering oligonucleotides to immune cells
WO2009132131A1 (en) Amino lipid based improved lipid formulation
HK1160459B (en) Improved amino lipids and methods for the delivery of nucleic acids