CN119072464A - 新型可电离脂质和脂质纳米颗粒以及其使用方法 - Google Patents

新型可电离脂质和脂质纳米颗粒以及其使用方法 Download PDF

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Publication number
CN119072464A
CN119072464A CN202380035765.6A CN202380035765A CN119072464A CN 119072464 A CN119072464 A CN 119072464A CN 202380035765 A CN202380035765 A CN 202380035765A CN 119072464 A CN119072464 A CN 119072464A
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independently
lipid
alkyl
compound according
unsubstituted
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Chinese (zh)
Inventor
A·巴尔托洛齐
J·普劳德富特
A·阿迪卡里
S·帕特尔
A·豪
D·萨勒诺
J·尤尼恩
S·阿迪卡里
R·埃德曼
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Sail Biopharmaceutical Co ltd
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Sail Biopharmaceutical Co ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/04—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D207/10—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D207/12—Oxygen or sulfur atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/04—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D207/10—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D207/16—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/60—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings linked by a chain containing hetero atoms as chain links

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202380035765.6A 2022-03-25 2023-03-24 新型可电离脂质和脂质纳米颗粒以及其使用方法 Pending CN119072464A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263323948P 2022-03-25 2022-03-25
US63/323,948 2022-03-25
PCT/US2023/016300 WO2023183616A1 (en) 2022-03-25 2023-03-24 Novel ionizable lipids and lipid nanoparticles and methods of using the same

Publications (1)

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CN119072464A true CN119072464A (zh) 2024-12-03

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Country Status (9)

Country Link
US (1) US20250205167A1 (https=)
EP (1) EP4499607A1 (https=)
JP (1) JP2025510229A (https=)
KR (1) KR20240166554A (https=)
CN (1) CN119072464A (https=)
AU (1) AU2023239151A1 (https=)
CA (1) CA3245624A1 (https=)
MX (1) MX2024011629A (https=)
WO (1) WO2023183616A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121057577A (zh) * 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN119285518A (zh) * 2023-07-10 2025-01-10 台湾生物医药制造股份有限公司 脂质及其应用
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025088570A1 (en) * 2023-10-26 2025-05-01 Crispr Therapeutics Ag Ionizable lipids and ionizable lipid nanoparticles
WO2025129201A1 (en) 2023-12-15 2025-06-19 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025134066A1 (en) * 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025189064A1 (en) 2024-03-08 2025-09-12 Genzyme Corporation Lipid nanoparticles
WO2025250729A1 (en) * 2024-05-29 2025-12-04 Liberate Bio, Inc. Lipid compounds, compositions, and uses thereof
CN118512419B (zh) * 2024-06-14 2025-03-14 江西济民可信集团有限公司 一种金水宝微丸胶囊及其制备方法
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof
CN119193934B (zh) * 2024-11-29 2025-03-14 聚芯堂(成都)生物科技有限公司 一种高危型hpv一锅法检测冻干试剂、试剂盒及其用途

Family Cites Families (99)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US553A (en) 1838-01-09 Machine eoe
US907699A (en) 1906-04-24 1908-12-22 William D Moore Building-block.
US4511069A (en) 1981-06-04 1985-04-16 The Pharmasol Corporation Dispensing system
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
US5965542A (en) 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
JP2002502831A (ja) 1998-02-03 2002-01-29 イネックス ファーマシューティカルズ コーポレイション 癌の治療に用いる血清に対して安定なプラスミド脂質粒子の全身供与
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
US7780882B2 (en) 1999-02-22 2010-08-24 Georgetown University Simplified and improved method for preparing an antibody or an antibody fragment targeted immunoliposome for systemic administration of a therapeutic or diagnostic agent
US9034329B2 (en) 1999-02-22 2015-05-19 Georgetown University Preparation of antibody or an antibody fragment-targeted immunoliposomes for systemic administration of therapeutic or diagnostic agents and uses thereof
US6211140B1 (en) 1999-07-26 2001-04-03 The Procter & Gamble Company Cationic charge boosting systems
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US6927663B2 (en) 2003-07-23 2005-08-09 Cardiac Pacemakers, Inc. Flyback transformer wire attach method to printed circuit board
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
JP4380411B2 (ja) 2004-04-30 2009-12-09 澁谷工業株式会社 滅菌方法
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
CN101287497B (zh) 2004-12-27 2013-03-06 赛伦斯治疗公司 涂层脂质复合体和它们的用途
US7451428B2 (en) 2005-02-24 2008-11-11 Texas Instruments Incorporated Merging sub-resolution assist features of a photolithographic mask through the use of a merge bar
US9005654B2 (en) 2005-07-27 2015-04-14 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
WO2008008230A2 (en) 2006-07-10 2008-01-17 Memsic, Inc. A system for sensing yaw rate using a magnetic field sensor and portable electronic devices using the same
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
WO2010054384A1 (en) * 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2467357B1 (en) 2009-08-20 2016-03-30 Sirna Therapeutics, Inc. Novel cationic lipids with various head groups for oligonucleotide delivery
EP2506879A4 (en) 2009-12-01 2014-03-19 Protiva Biotherapeutics Inc PREPARATIONS OF SNALP CONTAINING ANTIOXIDANTS
WO2011071860A2 (en) * 2009-12-07 2011-06-16 Alnylam Pharmaceuticals, Inc. Compositions for nucleic acid delivery
CA2784568A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Lipid particles for delivery of nucleic acids
US20130116419A1 (en) 2010-01-22 2013-05-09 Daniel Zewge Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
JP4782232B1 (ja) 2010-04-09 2011-09-28 シャープ株式会社 光源モジュール、及びそれを備えた電子機器
JP5902616B2 (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
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
SG186085A1 (en) 2010-06-03 2013-01-30 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
US8748667B2 (en) 2010-06-04 2014-06-10 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
ES2727583T3 (es) 2010-08-31 2019-10-17 Glaxosmithkline Biologicals Sa Lípidos adecuados para la administración liposómica de ARN que codifica proteínas
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
ES2888231T3 (es) 2010-09-20 2022-01-03 Sirna Therapeutics Inc Lípidos catiónicos de bajo peso molecular para el suministro de oligonucleótidos
EP2621480B1 (en) 2010-09-30 2018-08-15 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
JP2013545727A (ja) 2010-10-21 2013-12-26 メルク・シャープ・アンド・ドーム・コーポレーション オリゴヌクレオチド送達用の新規低分子量カチオン性脂質
EP2635265B1 (en) * 2010-11-05 2018-04-04 Sirna Therapeutics, Inc. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
US9617461B2 (en) 2010-12-06 2017-04-11 Schlumberger Technology Corporation Compositions and methods for well completions
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
WO2012162210A1 (en) 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
EP2760477B1 (en) 2011-09-27 2018-08-08 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
US8762704B2 (en) 2011-09-29 2014-06-24 Apple Inc. Customized content for electronic devices
KR102451116B1 (ko) 2011-10-27 2022-10-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
JP6093710B2 (ja) 2011-11-18 2017-03-08 日油株式会社 細胞内動態を改善したカチオン性脂質
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
EP2788006B1 (en) 2011-12-07 2026-01-07 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
EP2788316B1 (en) 2011-12-07 2019-04-24 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
TWI594767B (zh) 2011-12-12 2017-08-11 協和醱酵麒麟有限公司 含有陽離子性脂質之藥物傳遞系統用脂質奈米粒子
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
WO2013148541A1 (en) 2012-03-27 2013-10-03 Merck Sharp & Dohme Corp. DIETHER BASED BIODEGRADABLE CATIONIC LIPIDS FOR siRNA DELIVERY
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
EP3608308B1 (en) * 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
US9593077B2 (en) 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
EP4019506A1 (en) 2013-12-19 2022-06-29 Novartis AG Lipids and lipid compositions for the delivery of active agents
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
SMT202200502T1 (it) 2014-06-25 2023-01-13 Acuitas Therapeutics Inc Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici
US10125092B2 (en) * 2014-09-05 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US20180303925A1 (en) * 2015-04-27 2018-10-25 The Trustees Of The University Of Pennsylvania Nucleoside-Modified RNA For Inducing an Adaptive Immune Response
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
LT3313829T (lt) 2015-06-29 2024-08-12 Acuitas Therapeutics Inc. Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
HRP20220652T1 (hr) 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
HUE057877T2 (hu) * 2015-12-22 2022-06-28 Modernatx Inc Vegyületek és készítmények terápiás szerek intracelluláris bejuttatására
EP3397613A1 (en) 2015-12-30 2018-11-07 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
US20190167811A1 (en) * 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
EP3458106A4 (en) * 2016-05-18 2020-03-18 Modernatx, Inc. POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA
ES3063077T3 (en) 2016-10-26 2026-04-15 Acuitas Therapeutics Inc Lipid nanoparticle formulations
EP3630076A1 (en) * 2017-05-30 2020-04-08 GlaxoSmithKline Biologicals SA Methods for manufacturing a liposome encapsulated rna
AU2018330208B2 (en) 2017-09-08 2025-04-17 Generation Bio Co. Lipid nanoparticle formulations of non-viral, capsid-free DNA vectors
JP7543259B2 (ja) 2018-10-18 2024-09-02 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
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
WO2020219876A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
AU2020350759A1 (en) * 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
CN114391008B (zh) * 2020-08-20 2024-05-03 苏州艾博生物科技有限公司 脂质化合物和脂质纳米颗粒组合物
CN112961065B (zh) * 2021-02-05 2023-03-14 嘉晨西海(杭州)生物技术有限公司 一种可电离脂质分子及其制备方法及其在制备脂质纳米颗粒的应用
TW202328067A (zh) * 2021-09-14 2023-07-16 美商雷納嘉德醫療管理公司 環狀脂質及其使用方法

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