JP2017535610A5 - - Google Patents

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Publication number
JP2017535610A5
JP2017535610A5 JP2017545520A JP2017545520A JP2017535610A5 JP 2017535610 A5 JP2017535610 A5 JP 2017535610A5 JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017535610 A5 JP2017535610 A5 JP 2017535610A5
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JP
Japan
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carbons
compound according
linear
compound
item
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JP2017545520A
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English (en)
Japanese (ja)
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JP2017535610A (ja
JP6637988B2 (ja
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Priority claimed from US14/546,105 external-priority patent/US9593077B2/en
Priority claimed from US14/707,876 external-priority patent/US9365610B2/en
Application filed filed Critical
Priority claimed from PCT/US2015/030218 external-priority patent/WO2016081029A1/en
Publication of JP2017535610A publication Critical patent/JP2017535610A/ja
Publication of JP2017535610A5 publication Critical patent/JP2017535610A5/ja
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Publication of JP6637988B2 publication Critical patent/JP6637988B2/ja
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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 US9593077B2 (en) 2013-11-18 2014-11-18 Ionizable cationic lipid for RNA delivery
US14/546,105 2014-11-18
US14/707,796 2015-05-08
US14/707,796 US9567296B2 (en) 2013-11-18 2015-05-08 Ionizable cationic lipid for RNA delivery
US14/707,876 2015-05-08
US14/707,876 US9365610B2 (en) 2013-11-18 2015-05-08 Asymmetric ionizable cationic lipid for RNA delivery
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 true JP2017535610A5 (Direct) 2018-09-27
JP6637988B2 JP6637988B2 (ja) 2020-01-29

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Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017545520A Active JP6637988B2 (ja) 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

Country Status (20)

Country Link
EP (1) EP3221293B1 (Direct)
JP (1) JP6637988B2 (Direct)
KR (1) KR102380363B1 (Direct)
CN (1) CN107207428A (Direct)
AU (1) AU2015350561B2 (Direct)
CA (1) CA2968060C (Direct)
DK (1) DK3221293T3 (Direct)
ES (1) ES2946110T3 (Direct)
FI (1) FI3221293T3 (Direct)
HK (1) HK1244479A1 (Direct)
HR (1) HRP20230342T1 (Direct)
HU (1) HUE062130T2 (Direct)
IL (1) IL252291B (Direct)
LT (1) LT3221293T (Direct)
NZ (1) NZ732777A (Direct)
PL (1) PL3221293T3 (Direct)
PT (1) PT3221293T (Direct)
RS (1) RS64078B1 (Direct)
SI (1) SI3221293T1 (Direct)
WO (1) WO2016081029A1 (Direct)

Families Citing this family (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2930602C (en) 2013-11-18 2019-05-28 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2018112282A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload delivery
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
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
US20200046830A1 (en) * 2017-03-30 2020-02-13 The Government Of The United States, As Represented By The Secretary Of The Army Nucleic acid vaccine composition comprising a lipid formulation, and method of increasing the potency of nucleic acid vaccines
US11015204B2 (en) * 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
EP3773476A1 (en) 2018-03-30 2021-02-17 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
PL3784285T3 (pl) 2018-04-25 2023-12-18 Ethris Gmbh Środki krioprotekcyjne do formulacji cząstek stałych
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
KR102669334B1 (ko) 2018-06-08 2024-05-27 후지필름 가부시키가이샤 화합물 또는 그 염 및 지질 입자
EP3891274B1 (en) 2018-12-06 2023-10-18 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
KR20220018960A (ko) 2019-03-19 2022-02-15 아크투루스 쎄라퓨틱스, 인크. 지질-캡슐화된 rna 나노입자 제조 방법
EP3981435B1 (en) 2019-06-07 2023-08-16 FUJIFILM Corporation Lipid composition
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
JP7389134B2 (ja) 2019-11-15 2023-11-29 富士フイルム株式会社 脂質組成物
AU2021231795A1 (en) 2020-03-03 2022-10-06 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021257917A1 (en) * 2020-06-18 2021-12-23 Arcturus Therapeutics, Inc. Una oligomers for the treatment of polyglutamine diseases
KR20230056669A (ko) 2020-07-27 2023-04-27 안자리움 바이오사이언시스 아게 Dna 분자의 조성물, 이의 제조 방법 및 이의 사용 방법
WO2022056413A1 (en) 2020-09-13 2022-03-17 Arcturus Therapeutics, Inc. Lipid nanoparticles encapsulation of large rna
US20240173267A1 (en) * 2021-02-10 2024-05-30 Oncorus, Inc. Compounds, compositions, and methods of using thereof
KR20240012370A (ko) 2021-04-20 2024-01-29 안자리움 바이오사이언시스 아게 아밀로-알파-1, 6-글루코시다제, 4-알파-글루카노트랜스퍼라제를 인코딩하는 dna 분자의 조성물, 이를 제조하는 방법 및 이를 사용하는 방법
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
JP2024515788A (ja) 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスdnaベクター及びその使用
WO2023031394A1 (en) 2021-09-03 2023-03-09 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
JP2024542155A (ja) 2021-11-08 2024-11-13 オルナ セラピューティクス、インコーポレイテッド 環状ポリヌクレオチドを送達するための脂質ナノ粒子組成物
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
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
CA3245737A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. PRIME-BOOST HETEROLOGICAL VACCINE COMPOSITIONS AND METHODS OF USE
CA3248209A1 (en) 2022-05-25 2023-11-30 CureVac SE Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH)
AU2023283348A1 (en) 2022-06-07 2025-01-09 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
CA3268237A1 (en) 2022-09-23 2024-03-28 Nchroma Bio, Inc. COMPOSITIONS AND METHODS FOR THE 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
EP4615517A1 (en) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Circular rna compositions
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
CN120641085A (zh) 2022-12-01 2025-09-12 世代生物公司 包含核酸、可电离脂质、固醇、脂质锚定聚合物和辅助脂质的脂质纳米颗粒、其用途
EP4626401A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
CN120641465A (zh) 2022-12-01 2025-09-12 世代生物公司 新颖的聚甘油缀合脂质和包含其的脂质纳米颗粒组合物
KR20250153298A (ko) 2023-03-08 2025-10-24 큐어백 에스이 핵산 전달을 위한 신규의 지질 나노입자 제형
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
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
US12311033B2 (en) 2023-05-31 2025-05-27 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
US20250332281A2 (en) 2023-12-15 2025-10-30 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
TW202543585A (zh) 2024-03-08 2025-11-16 美商健臻公司 脂質奈米粒子
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
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR8207733A (pt) 1981-06-04 1983-05-10 Pharmasol Corp Recipiente pressurizado com bomba de suprimento
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
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
AU2011245987B2 (en) * 2010-04-28 2016-12-15 Kyowa Hakko Kirin Co., Ltd Cationic lipid
DK2718261T3 (en) * 2011-06-08 2016-03-29 Nitto Denko Corp Compounds to target drug delivery and promote siRNA activity
WO2013086373A1 (en) * 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
AU2013270685B2 (en) * 2012-06-08 2017-11-30 Nitto Denko Corporation Lipids for therapeutic agent delivery formulations
CA2930602C (en) * 2013-11-18 2019-05-28 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery

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