JP6637988B2 - Rna送達のためのイオン化可能カチオン性脂質 - Google Patents

Rna送達のためのイオン化可能カチオン性脂質 Download PDF

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JP6637988B2
JP6637988B2 JP2017545520A JP2017545520A JP6637988B2 JP 6637988 B2 JP6637988 B2 JP 6637988B2 JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017545520 A JP2017545520 A JP 2017545520A JP 6637988 B2 JP6637988 B2 JP 6637988B2
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JP2017535610A5 (OSRAM
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ジョセフ イー. ペイン,
ジョセフ イー. ペイン,
パッドマナブ チブクラ,
パッドマナブ チブクラ,
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アークトゥルス セラピューティクス, インコーポレイテッド
アークトゥルス セラピューティクス, インコーポレイテッド
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JP2017545520A 2014-11-18 2015-05-11 Rna送達のためのイオン化可能カチオン性脂質 Active JP6637988B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US14/546,105 2014-11-18
US14/546,105 US9593077B2 (en) 2013-11-18 2014-11-18 Ionizable cationic lipid for RNA delivery
US14/707,876 US9365610B2 (en) 2013-11-18 2015-05-08 Asymmetric ionizable cationic lipid for RNA delivery
US14/707,796 US9567296B2 (en) 2013-11-18 2015-05-08 Ionizable cationic lipid for RNA delivery
US14/707,796 2015-05-08
US14/707,876 2015-05-08
PCT/US2015/030218 WO2016081029A1 (en) 2014-11-18 2015-05-11 Ionizable cationic lipid for rna delivery

Publications (3)

Publication Number Publication Date
JP2017535610A JP2017535610A (ja) 2017-11-30
JP2017535610A5 JP2017535610A5 (OSRAM) 2018-09-27
JP6637988B2 true JP6637988B2 (ja) 2020-01-29

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JP (1) JP6637988B2 (OSRAM)
KR (1) KR102380363B1 (OSRAM)
CN (1) CN107207428A (OSRAM)
AU (1) AU2015350561B2 (OSRAM)
CA (1) CA2968060C (OSRAM)
DK (1) DK3221293T3 (OSRAM)
ES (1) ES2946110T3 (OSRAM)
FI (1) FI3221293T3 (OSRAM)
HK (1) HK1244479A1 (OSRAM)
HR (1) HRP20230342T1 (OSRAM)
HU (1) HUE062130T2 (OSRAM)
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PT (1) PT3221293T (OSRAM)
RS (1) RS64078B1 (OSRAM)
SI (1) SI3221293T1 (OSRAM)
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Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102095085B1 (ko) 2013-11-18 2020-03-31 아크투루스 쎄라퓨틱스, 인크. Rna 전달을 위한 이온화가능한 양이온성 지질
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
KR20210035022A (ko) 2016-12-14 2021-03-31 리간달 인코포레이티드 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
US10383952B2 (en) * 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
EP3600396A4 (en) * 2017-03-30 2021-01-13 The Government of the United States of America as represented by the Secretary of the Army VACCINE COMPOSITION OF NUCLEIC ACID INCLUDING A LIPIDIC FORMULATION, AND PROCESS FOR INCREASING THE EFFECTIVENESS OF NUCLEIC ACID VACCINES
US12083224B2 (en) 2018-03-30 2024-09-10 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
ES2960936T3 (es) 2018-04-25 2024-03-07 Ethris Gmbh Formulaciones a base de lípidos para el suministro de ARN
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
CN116172975B (zh) * 2018-06-08 2025-03-28 富士胶片株式会社 脂质颗粒、包含该脂质颗粒的核酸药物以及核酸递送用组合物
WO2020118115A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
WO2020191103A1 (en) 2019-03-19 2020-09-24 Arcturus Therapeutics, Inc. Method of making lipid-encapsulated rna nanoparticles
CA3143865A1 (en) 2019-06-07 2020-12-10 Fujifilm Corporation Lipid composition
JP2022550435A (ja) 2019-10-04 2022-12-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えaavの改善された治療的使用のための方法
CA3158293C (en) 2019-11-15 2024-06-25 Fujifilm Corporation Lipid composition
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
JP2023520763A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性dnaベクター及びその使用
JP2023530487A (ja) * 2020-06-18 2023-07-18 アークトゥラス・セラピューティクス・インコーポレイテッド ポリグルタミン病の治療のためのunaオリゴマー
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Dna分子の組成物、その作製方法、及びその使用方法
AU2021340876A1 (en) 2020-09-13 2023-04-13 Arcturus Therapeutics, Inc. Lipid nanoparticles encapsulation of large RNA
MX2023009345A (es) 2021-02-10 2023-10-24 Oncorus Inc Compuestos, composiciones y metodos de uso de estos.
AU2022260111A1 (en) 2021-04-20 2023-11-30 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
EP4329885A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
AU2022336209B2 (en) 2021-09-03 2025-10-16 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
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
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN114213295B (zh) * 2022-02-22 2022-05-20 中国科学院基础医学与肿瘤研究所(筹) 一种阳离子化合物、制备方法及其复合物和用途
JP2025509607A (ja) 2022-03-14 2025-04-11 ジェネレーション バイオ カンパニー 異種プライムブーストワクチン組成物及び使用の方法
CN119212720A (zh) 2022-05-25 2024-12-27 库瑞瓦格欧洲股份公司 编码大肠杆菌FimH抗原性多肽的基于核酸的疫苗
WO2023239756A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
AU2023347320A1 (en) 2022-09-23 2025-04-10 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
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
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
AU2023375897A1 (en) 2022-11-08 2025-06-12 Orna Therapeutics, Inc. Circular rna compositions
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
KR20250129819A (ko) 2022-12-01 2025-08-29 제너레이션 바이오 컴퍼니 핵산, 이온화 가능한 지질, 스테롤, 지질에 고정된 중합체, 및 헬퍼 지질을 포함하는 지질 나노입자 및 이의 용도
KR20250131271A (ko) 2022-12-01 2025-09-02 제너레이션 바이오 컴퍼니 신규한 폴리글리세롤-접합된 지질 및 이를 포함하는 지질 나노입자 조성물
KR20250114116A (ko) 2022-12-01 2025-07-28 제너레이션 바이오 컴퍼니 세포 표적화를 위한 스텔스 지질 나노입자 조성물
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
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
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025080939A1 (en) 2023-10-13 2025-04-17 Ultragenyx Pharmaceutical, Inc. Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025128853A2 (en) 2023-12-13 2025-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
WO2025129201A1 (en) 2023-12-15 2025-06-19 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
US20250381150A1 (en) 2024-03-08 2025-12-18 Genzyme Corporation Lipid nanoparticles
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0079963B1 (en) 1981-06-04 1986-08-27 Delaware Chemicals Corporation Pressurized container with dispensing pump
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
PL169576B1 (pl) 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US9408914B2 (en) * 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
EP2998289B1 (en) * 2011-06-08 2019-08-07 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing sirna activity
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
BR112014030714B1 (pt) * 2012-06-08 2020-12-22 Nitto Denko Corporation composto de lipídeo ionizável, composição, veículo de fármaco e formulação farmacêutica
KR102095085B1 (ko) * 2013-11-18 2020-03-31 아크투루스 쎄라퓨틱스, 인크. Rna 전달을 위한 이온화가능한 양이온성 지질

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