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
JP2017535610A (ja
JP6637988B2 (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
US14/707,796 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
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 (enExample) 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 (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 (65)

* 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
EP3957735A1 (en) 2014-03-05 2022-02-23 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
CA3045134A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload 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
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 NUCLEIC ACID VACCINE COMPOSITION WITH A LIPID FORMULATION AND METHOD FOR INCREASING THE POTENTIAL OF NUCLEIC ACID VACCINE
WO2018222890A1 (en) * 2017-05-31 2018-12-06 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
EP3784284B1 (en) 2018-04-25 2023-08-02 Ethris GmbH Lipid-based formulations for the delivery of rna
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
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
BR112021018526A2 (pt) * 2019-03-19 2021-11-23 Arcturus Therapeutics Inc Método de fabricação de nanopartículas de rna encapsuladas em lipídio
EP3981435B1 (en) 2019-06-07 2023-08-16 FUJIFILM Corporation Lipid composition
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
EP4328309B1 (en) 2019-11-15 2025-07-02 FUJIFILM Corporation Lipid composition
WO2021178510A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
CA3172591A1 (en) 2020-03-24 2021-09-30 Douglas Anthony KERR Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
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
EP4189098A1 (en) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions of dna molecules, methods of making therefor, and methods of use thereof
JP7774615B2 (ja) 2020-09-13 2025-11-21 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 大型rnaの脂質ナノ粒子封入
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
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4395748A1 (en) 2021-09-03 2024-07-10 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
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物
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
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
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
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
IL319704A (en) 2022-09-23 2025-05-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of HBV gene expression
US12186389B2 (en) 2022-10-28 2025-01-07 Glaxosmithkline Biologicals Sa Nucleic acid base vaccine against emerging SARS-CoV-2 variants
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
JP2025537178A (ja) 2022-11-08 2025-11-14 オーナ セラピューティクス, インコーポレイテッド 環状rna組成物
EP4626444A2 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
IL320873A (en) 2022-12-01 2025-07-01 Generation Bio Co Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions containing them
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
CN120615013A (zh) 2022-12-01 2025-09-09 世代生物公司 用于细胞靶向的隐形脂质纳米颗粒组合物
AU2024233180A1 (en) 2023-03-08 2025-09-25 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
EP4713453A1 (en) 2023-05-15 2026-03-25 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
CN121666232A (zh) 2023-05-31 2026-03-13 开普斯坦治疗公司 脂质纳米颗粒制剂和组合物
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
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE21633T1 (de) 1981-06-04 1986-09-15 Pharmasol Corp Druckbehaelter mit abgabepumpe.
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
US9408914B2 (en) * 2010-04-28 2016-08-09 Kyowa Hakko Kirin Co., Ltd. Cationic lipid
AU2012267467B8 (en) * 2011-06-08 2017-06-15 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
CA2876148C (en) * 2012-06-08 2021-01-12 Nitto Denko Corporation Lipids for therapeutic agent delivery formulations
US9593077B2 (en) * 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery

Similar Documents

Publication Publication Date Title
JP2017535610A5 (enExample)
JP2015501833A5 (enExample)
RU2015151202A (ru) Композиции и способы модулирования экспрессии hbv и ttr
JP2014508753A5 (enExample)
JP2011517683A5 (enExample)
JP2014513954A5 (enExample)
JP2015504067A5 (enExample)
JP2014510729A5 (enExample)
RU2013142448A (ru) Новые сульфонаминохинолиновые антагонисты гепсидина
JP2013507439A5 (enExample)
JP2013509429A5 (enExample)
WO2015081133A3 (en) Nucleotides for the treatment of liver cancer
ES2668045T3 (es) Compuestos, composiciones y métodos que usan antagonistas de la E-selectina para la movilización de las células hematopoyéticas
JP2015180657A5 (enExample)
JP2017515901A5 (enExample)
JP2018135343A5 (enExample)
JP2011526924A5 (enExample)
JP2017527532A5 (enExample)
JP2008195730A5 (enExample)
JP2017510610A5 (enExample)
JP2013510120A5 (enExample)
JP2012502048A5 (enExample)
JP2015512943A5 (enExample)
JP2012158602A5 (enExample)
JP2010511721A5 (enExample)