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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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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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
US14/707,876 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
US14/707,876 2015-05-08
US14/707,796 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 true JP2017535610A5 (enExample) 2018-09-27
JP6637988B2 JP6637988B2 (ja) 2020-01-29

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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)

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

Families Citing this family (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
US11028388B2 (en) 2014-03-05 2021-06-08 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
RU2019121801A (ru) 2016-12-14 2021-01-15 Лигандал, Инк. Композиции и способы для доставки полезной нагрузки в виде нуклеиновой кислоты и/или белка
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
US10383952B2 (en) * 2016-12-21 2019-08-20 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
WO2019191780A1 (en) * 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
JP7256824B2 (ja) 2018-04-25 2023-04-12 エスリス ゲーエムベーハー 粒子状製剤用の凍結保護剤
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
ES2964547T3 (es) 2018-06-08 2024-04-08 Fujifilm Corp Compuesto, sal del mismo y partículas lipídicas
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
EP3942050A4 (en) 2019-03-19 2023-02-22 Arcturus Therapeutics, Inc. PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES
CA3143865A1 (en) 2019-06-07 2020-12-10 Fujifilm Corporation Lipid composition
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
EP4059505B1 (en) 2019-11-15 2024-02-21 FUJIFILM Corporation Lipid composition
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral DNA vectors and their use for therapeutic expression of factor ix
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
US20230227826A1 (en) * 2020-06-18 2023-07-20 Arcturus Therapeutics, Inc. Una oligomers for the treatment of polyglutamine diseases
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc As a mass of large RNA lipid nanoparticles
TWI918825B (zh) 2021-02-10 2026-03-21 美商艾勒維生技公司 化合物、組合物及其使用方法
JP2024517427A (ja) 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ-α-1,6-グルコシダーゼ、4-α-グルカノトランスフェラーゼをコードするDNA分子の組成物、その作製の方法、及びその使用の方法
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
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4763287A2 (en) 2021-09-03 2026-06-24 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
WO2023081526A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
JP2024546431A (ja) 2021-11-11 2024-12-24 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 Rna送達のためのイオン化可能なカチオン性脂質
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
EP4469091A1 (en) 2022-01-28 2024-12-04 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
EP4493212A1 (en) 2022-03-14 2025-01-22 Generation Bio Co. Heterologous prime boost 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)
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
KR20250110209A (ko) 2022-09-23 2025-07-18 엔크로마 바이오, 인크. Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
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
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
US20260207525A1 (en) 2022-12-01 2026-07-23 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
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
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ,インコーポレーテッド Hbv遺伝子発現のエピジェネティック調節のための組成物および方法
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
EP4719352A1 (en) 2023-05-31 2026-04-08 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
CN117986153B (zh) 2023-09-07 2026-06-19 晟迪生物医药(苏州)有限公司 一种可电离阳离子脂质化合物及其制备方法和应用
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
EP4791878A1 (en) 2023-10-13 2026-08-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations
CN122497525A (zh) 2023-11-07 2026-07-31 欧纳医疗公司 环状rna组合物
AU2024397279A1 (en) 2023-12-13 2026-07-02 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression
IL328626A (en) 2023-12-15 2026-07-01 Capstan Therapeutics Inc Humanized anti-CD8 antibodies and their uses
AU2025217182A1 (en) 2024-02-02 2026-08-06 Editas Medicine, Inc. MODIFIED GUIDE RNAs
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
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
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
WO2026102260A1 (en) 2024-11-08 2026-05-15 Editas Medicine, Inc. Crispr-related methods and compositions targeting angiopoietin like 3 (angptl3)
WO2026142951A1 (en) 2024-12-23 2026-07-02 Genzyme Corporation Pharmaceutical system

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
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
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
KR101773643B1 (ko) * 2010-04-28 2017-08-31 교와 핫꼬 기린 가부시키가이샤 양이온성 지질
CN107043334B (zh) * 2011-06-08 2020-06-16 日东电工株式会社 用于定向药物输送和增强siRNA活性的化合物
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
TR201816986T4 (tr) * 2012-06-08 2019-01-21 Nitto Denko Corp Terapötik ajan iletim formülasyonlarına yönelik lipitler.
CN110003066B (zh) * 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质

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