JP2017535610A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
carbons
compound according
linear
compound
item
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2017545520A
Other languages
English (en)
Japanese (ja)
Other versions
JP6637988B2 (ja
JP2017535610A (ja
Filing date
Publication date
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
Application granted granted Critical
Publication of JP6637988B2 publication Critical patent/JP6637988B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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 2015-05-08
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
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 (https=) 2018-09-27
JP6637988B2 JP6637988B2 (ja) 2020-01-29

Family

ID=56014367

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

Families Citing this family (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9593077B2 (en) 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
CN110582301A (zh) 2016-12-14 2019-12-17 利甘达尔股份有限公司 用于核酸和蛋白质有效负载递送的方法和组合物
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10383952B2 (en) * 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018183901A1 (en) * 2017-03-30 2018-10-04 The Government Of The United States Of America 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
ES3039647T3 (en) * 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
US12083224B2 (en) 2018-03-30 2024-09-10 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
MX2020011166A (es) 2018-04-25 2022-10-19 Ethris Gmbh Agentes crioprotectores para formulaciones en forma de particulas.
KR20190127277A (ko) * 2018-05-04 2019-11-13 주식회사 삼양바이오팜 mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법
PL3805198T3 (pl) * 2018-06-08 2024-02-12 Fujifilm Corporation Związek, jego sól i cząstki lipidowe
JP7445657B2 (ja) 2018-12-06 2024-03-07 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 オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
EP3942050A4 (en) * 2019-03-19 2023-02-22 Arcturus Therapeutics, Inc. PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES
WO2020246581A1 (ja) 2019-06-07 2020-12-10 富士フイルム株式会社 脂質組成物
US12544437B2 (en) 2019-10-04 2026-02-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant AAV
EP4059505B1 (en) 2019-11-15 2024-02-21 FUJIFILM Corporation Lipid composition
JP7832114B2 (ja) 2020-03-03 2026-03-17 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 オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
AU2021244559A1 (en) 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
MX2022011805A (es) 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
US20230227826A1 (en) * 2020-06-18 2023-07-20 Arcturus Therapeutics, Inc. Una oligomers for the treatment of polyglutamine diseases
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
WO2022056413A1 (en) 2020-09-13 2022-03-17 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
CN117881786A (zh) 2021-04-27 2024-04-12 世代生物公司 表达治疗性抗体的非病毒dna载体及其用途
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
MX2024002726A (es) 2021-09-03 2024-03-20 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos.
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
EP4430024A1 (en) 2021-11-08 2024-09-18 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
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
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
EP4536197A1 (en) 2022-06-07 2025-04-16 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
CN120390656A (zh) 2022-11-08 2025-07-29 欧纳医疗公司 环状rna组合物
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
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
JP2025540076A (ja) 2022-12-01 2025-12-11 ジェネレーション バイオ カンパニー 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用
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
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
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
IL324634A (en) 2023-05-15 2026-01-01 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
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
WO2025189064A1 (en) 2024-03-08 2025-09-12 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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
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
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
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
SI2858974T1 (sl) * 2012-06-08 2019-01-31 Nitto Denko Corporation Lipidi za formulacije za dostavo terapevtskega sredstva
US9593077B2 (en) * 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery

Similar Documents

Publication Publication Date Title
JP2017535610A5 (https=)
JP2015501833A5 (https=)
RU2015151202A (ru) Композиции и способы модулирования экспрессии hbv и ttr
JP2014508753A5 (https=)
JP2011517683A5 (https=)
JP2014513954A5 (https=)
JP2015504067A5 (https=)
JP2014510729A5 (https=)
JP2013507439A5 (https=)
JP2013509429A5 (https=)
ES2668045T3 (es) Compuestos, composiciones y métodos que usan antagonistas de la E-selectina para la movilización de las células hematopoyéticas
JP2015180657A5 (https=)
JP2017515901A5 (https=)
JP2018135343A5 (https=)
JP2011526924A5 (https=)
JP2017527532A5 (https=)
JP2008195730A5 (https=)
JP2017510610A5 (https=)
JP2013510120A5 (https=)
JP2012502048A5 (https=)
JP2015512943A5 (https=)
JP2009536660A5 (https=)
JP2019507111A5 (https=)
JP2010511721A5 (https=)
JP2009530398A5 (https=)