CN116348147A - 用于将有效载荷分子递送至气道上皮的组合物 - Google Patents

用于将有效载荷分子递送至气道上皮的组合物 Download PDF

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CN116348147A
CN116348147A CN202180068470.XA CN202180068470A CN116348147A CN 116348147 A CN116348147 A CN 116348147A CN 202180068470 A CN202180068470 A CN 202180068470A CN 116348147 A CN116348147 A CN 116348147A
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nanoparticle
alkyl
lipid
membered heterocycloalkyl
cell
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A·卡德特皮雷斯
J·C·宋
M·科恩比斯
J·赫卡奇
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ModernaTx Inc
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1273Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • C07J41/0055Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
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CN202180068470.XA 2020-08-06 2021-08-06 用于将有效载荷分子递送至气道上皮的组合物 Pending CN116348147A (zh)

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US202063062367P 2020-08-06 2020-08-06
US63/062367 2020-08-06
PCT/US2021/045038 WO2022032154A2 (en) 2020-08-06 2021-08-06 Compositions for the delivery of payload molecules to airway epithelium

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US (1) US20230285310A1 (https=)
EP (1) EP4192433A2 (https=)
JP (1) JP2023538260A (https=)
KR (1) KR20230087443A (https=)
CN (1) CN116348147A (https=)
AU (1) AU2021320426A1 (https=)
BR (1) BR112023001955A2 (https=)
CA (1) CA3189854A1 (https=)
IL (1) IL300111A (https=)
MA (1) MA71659A (https=)
MX (1) MX2023001461A (https=)
TW (1) TW202214215A (https=)
WO (1) WO2022032154A2 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117919200A (zh) * 2024-01-19 2024-04-26 晟迪生物医药(苏州)有限公司 含有可电离脂质的肺部递送系统、其制备方法和应用
WO2025180481A1 (zh) * 2024-03-01 2025-09-04 北京剂泰医药科技有限公司 聚乙二醇化脂质化合物、其脂质纳米颗粒、其组合物及其用途
CN120718086A (zh) * 2024-06-13 2025-09-30 仁景(苏州)生物科技有限公司 用于递送的脂质化合物和脂质纳米颗粒
US12617752B2 (en) 2023-06-16 2026-05-05 Beijing Jitai Life Sciences Ltd Rapidly metabolized lipid compound

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
MA42543A (fr) 2015-07-30 2018-06-06 Modernatx Inc Arn épitope peptidiques concatémériques
AU2016341309A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Cancer vaccines
EP4011451A1 (en) 2015-10-22 2022-06-15 ModernaTX, Inc. Metapneumovirus mrna vaccines
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
US11905525B2 (en) 2017-04-05 2024-02-20 Modernatx, Inc. Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
EP3668979A4 (en) 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF HPLC ANALYSIS
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
EP3746090A4 (en) 2018-01-29 2021-11-17 ModernaTX, Inc. RSV RNA Vaccines
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061295A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. High-purity peg lipids and uses thereof
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
CN113874502A (zh) 2019-03-11 2021-12-31 摩登纳特斯有限公司 补料分批体外转录方法
US12070495B2 (en) 2019-03-15 2024-08-27 Modernatx, Inc. HIV RNA vaccines
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
EP4274607A1 (en) 2021-01-11 2023-11-15 ModernaTX, Inc. Seasonal rna influenza virus vaccines
JP2024511463A (ja) 2021-03-22 2024-03-13 リコード セラピューティクス,インク. 細胞への標的送達のための組成物および方法
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
WO2023076598A1 (en) * 2021-10-29 2023-05-04 Modernatx, Inc. Lipid amines
US20250281419A1 (en) * 2021-10-29 2025-09-11 Modernatx, Inc. Lipid amines
CN118829423A (zh) * 2021-11-12 2024-10-22 摩登纳特斯有限公司 用于将有效载荷分子递送至气道上皮的组合物
EP4475882A1 (en) 2022-02-09 2024-12-18 ModernaTX, Inc. Mucosal administration methods and formulations
EP4479082A2 (en) * 2022-02-15 2024-12-25 The Broad Institute, Inc. Cell-type specific targeting contractile injection system
US20250188028A1 (en) * 2022-03-14 2025-06-12 Nanovation Therapeutics Inc. Synthetic Method for Producing Ionizable Amino Lipids
EP4509544A4 (en) * 2022-04-12 2025-07-02 Xiamen Sinopeg Biotech Co Ltd NONLINEAR PEGYLATED LIPID CONTAINING TERTIARY AMINE AND ITS APPLICATION
CN117003808A (zh) * 2022-04-28 2023-11-07 北京科兴中维生物技术有限公司 一种阳离子脂质化合物及其制备方法和用途
CN119816292A (zh) * 2022-08-08 2025-04-11 先进核糖核酸疫苗(Arv)技术股份有限公司 基于甾醇的可电离脂质以及包含其的脂质纳米颗粒
IL320459A (en) 2022-10-27 2025-06-01 Pfizer Rna molecules encoding rsv-f and vaccines containing them
US20250367129A1 (en) 2022-11-04 2025-12-04 Pfizer Inc. Lipid Compounds and Uses Thereof
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
AU2023409158A1 (en) * 2022-12-19 2025-07-03 Fujifilm Corporation Methods of delivering therapeutic agents, and lipid compositions
EP4658636A1 (en) 2023-01-31 2025-12-10 Pfizer Inc. Lipid compounds and uses thereof
CN121263425A (zh) * 2023-02-13 2026-01-02 旗舰创业创新第七有限责任公司 含可裂解接头的可电离脂质和用于治疗性组合物的脂质载体
CN121487722A (zh) * 2023-04-14 2026-02-06 北京剂泰生命科技有限公司 靶向脾的脂质纳米颗粒
EP4704905A1 (en) * 2023-05-02 2026-03-11 Sage Therapeutics, LLC Lymphatic system-targeting compounds
EP4704816A1 (en) * 2023-05-03 2026-03-11 ModernaTX, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2025027492A1 (en) 2023-07-31 2025-02-06 Pfizer Inc. Coronavirus antigen variants
WO2025034764A1 (en) * 2023-08-07 2025-02-13 Life Technologies Corporation Lipids for delivery of nucleic acids to eukaryotic cells
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2025126019A1 (en) 2023-12-13 2025-06-19 Pfizer Inc. Rna molecules
WO2025126071A1 (en) 2023-12-14 2025-06-19 Pfizer Inc. Rna molecules
WO2025126113A1 (en) 2023-12-15 2025-06-19 Pfizer Inc. Lipid compounds and uses thereof
WO2025126118A1 (en) 2023-12-15 2025-06-19 Pfizer Inc. Lipid compounds and uses thereof
WO2025186725A2 (en) 2024-03-06 2025-09-12 Pfizer Inc. Improved lnp formulations and uses thereof
WO2025207803A1 (en) * 2024-03-26 2025-10-02 General Therapeutics, Inc. Lipid compounds, compositions and methods of use thereof
WO2026018181A1 (en) 2024-07-17 2026-01-22 Pfizer Inc. Immunogenic compositions and uses thereof
WO2026029597A1 (ko) * 2024-07-31 2026-02-05 주식회사 메디치바이오 신규한 스테롤 지질 또는 이의 유도체를 함유하는 지질 나노입자 조성물
WO2026030275A1 (en) 2024-08-01 2026-02-05 Pfizer Inc. Rna molecules

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5783565A (en) * 1994-12-09 1998-07-21 Genzyme Corporation Cationic amphiphiles containing spermine or spermidine cationic group for intracellular delivery of therapeutic molecules
JPH10510813A (ja) * 1994-12-09 1998-10-20 ジェンザイム・コーポレイション 治療分子の細胞内送達のためのカチオン性両親媒性化合物およびプラスミド
US5925628A (en) * 1997-03-31 1999-07-20 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5942634A (en) * 1997-05-09 1999-08-24 Genzyme Corporation Cationic amphiphiles for cell transfections
WO2009051451A2 (en) * 2007-10-17 2009-04-23 Korea Advanced Institute Of Science And Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
WO2014058179A2 (ko) * 2012-10-11 2014-04-17 포항공과대학교 산학협력단 저밀도 지단백질 유사 나노입자 및 이를 포함하는 간 표적 진단 및 치료용 조성물
WO2019094405A1 (en) * 2017-11-10 2019-05-16 Board Of Regents, The University Of Texas System Lipid-based nanoparticles for encapsulation and sustained release of therapeutic agents
CN110520409A (zh) * 2017-03-15 2019-11-29 摩登纳特斯有限公司 用于细胞内递送治疗剂的化合物和组合物
US20200129445A1 (en) * 2017-03-15 2020-04-30 Modernatx, Inc. Lipid nanoparticle formulation

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
CA2689042A1 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
WO2010021865A1 (en) 2008-08-18 2010-02-25 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
WO2010080724A1 (en) 2009-01-12 2010-07-15 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
EP2485770A4 (en) 2009-10-08 2013-04-10 Merck Sharp & Dohme Novel cationic lipids with short lipid chains for oligonucleotide delivery
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
EP2525781A1 (en) 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011143230A1 (en) 2010-05-10 2011-11-17 Alnylam Pharmaceuticals Methods and compositions for delivery of active agents
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
DK2575767T3 (en) 2010-06-04 2017-03-13 Sirna Therapeutics Inc HOWEVER UNKNOWN LOW MOLECULAR CATIONIC LIPIDS TO PROCESS OIGONUCLEOTIDES
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
CA2809858C (en) 2010-09-20 2019-11-12 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
CA2811430A1 (en) 2010-09-30 2012-04-05 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
EP3485913A1 (en) 2010-10-21 2019-05-22 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
WO2012061259A2 (en) 2010-11-05 2012-05-10 Merck Sharp & Dohme Corp. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2013103659A1 (en) 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
CN105143456A (zh) 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
CN118697900A (zh) 2019-01-31 2024-09-27 摩登纳特斯有限公司 制备脂质纳米颗粒的方法

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5783565A (en) * 1994-12-09 1998-07-21 Genzyme Corporation Cationic amphiphiles containing spermine or spermidine cationic group for intracellular delivery of therapeutic molecules
JPH10510813A (ja) * 1994-12-09 1998-10-20 ジェンザイム・コーポレイション 治療分子の細胞内送達のためのカチオン性両親媒性化合物およびプラスミド
US5925628A (en) * 1997-03-31 1999-07-20 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5942634A (en) * 1997-05-09 1999-08-24 Genzyme Corporation Cationic amphiphiles for cell transfections
WO2009051451A2 (en) * 2007-10-17 2009-04-23 Korea Advanced Institute Of Science And Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
WO2014058179A2 (ko) * 2012-10-11 2014-04-17 포항공과대학교 산학협력단 저밀도 지단백질 유사 나노입자 및 이를 포함하는 간 표적 진단 및 치료용 조성물
CN110520409A (zh) * 2017-03-15 2019-11-29 摩登纳特斯有限公司 用于细胞内递送治疗剂的化合物和组合物
US20200129445A1 (en) * 2017-03-15 2020-04-30 Modernatx, Inc. Lipid nanoparticle formulation
WO2019094405A1 (en) * 2017-11-10 2019-05-16 Board Of Regents, The University Of Texas System Lipid-based nanoparticles for encapsulation and sustained release of therapeutic agents

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
EMA ROBINSON等: "Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis", 《MOLECULAR THERAPY》, vol. 26, no. 08, 15 June 2018 (2018-06-15), pages 2035 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12617752B2 (en) 2023-06-16 2026-05-05 Beijing Jitai Life Sciences Ltd Rapidly metabolized lipid compound
CN117919200A (zh) * 2024-01-19 2024-04-26 晟迪生物医药(苏州)有限公司 含有可电离脂质的肺部递送系统、其制备方法和应用
CN117919200B (zh) * 2024-01-19 2025-09-05 晟迪生物医药(苏州)有限公司 含有可电离脂质的肺部递送系统、其制备方法和应用
WO2025180481A1 (zh) * 2024-03-01 2025-09-04 北京剂泰医药科技有限公司 聚乙二醇化脂质化合物、其脂质纳米颗粒、其组合物及其用途
CN120718086A (zh) * 2024-06-13 2025-09-30 仁景(苏州)生物科技有限公司 用于递送的脂质化合物和脂质纳米颗粒

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