JPWO2020118041A5 - - Google Patents
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- Publication number
- JPWO2020118041A5 JPWO2020118041A5 JP2021531946A JP2021531946A JPWO2020118041A5 JP WO2020118041 A5 JPWO2020118041 A5 JP WO2020118041A5 JP 2021531946 A JP2021531946 A JP 2021531946A JP 2021531946 A JP2021531946 A JP 2021531946A JP WO2020118041 A5 JPWO2020118041 A5 JP WO2020118041A5
- Authority
- JP
- Japan
- Prior art keywords
- compound
- alkylene
- alkyl
- composition
- compound according
- 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
Links
- 150000001875 compounds Chemical class 0.000 claims description 118
- 239000000203 mixture Substances 0.000 claims description 69
- 125000000217 alkyl group Chemical group 0.000 claims description 40
- 125000002947 alkylene group Chemical group 0.000 claims description 39
- 238000000034 method Methods 0.000 claims description 25
- 150000003839 salts Chemical class 0.000 claims description 20
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 19
- 108020005004 Guide RNA Proteins 0.000 claims description 19
- 150000002632 lipids Chemical class 0.000 claims description 16
- 108020004999 messenger RNA Proteins 0.000 claims description 15
- 210000004027 cell Anatomy 0.000 claims description 12
- 125000004432 carbon atom Chemical group C* 0.000 claims description 10
- 108020004707 nucleic acids Proteins 0.000 claims description 10
- 102000039446 nucleic acids Human genes 0.000 claims description 10
- 150000007523 nucleic acids Chemical class 0.000 claims description 10
- 101710163270 Nuclease Proteins 0.000 claims description 7
- 238000010362 genome editing Methods 0.000 claims description 7
- 125000006705 (C5-C7) cycloalkyl group Chemical group 0.000 claims description 6
- 229910052799 carbon Inorganic materials 0.000 claims description 6
- 229910052757 nitrogen Inorganic materials 0.000 claims description 6
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 6
- 108020004414 DNA Proteins 0.000 claims description 4
- 125000003342 alkenyl group Chemical group 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 102000053602 DNA Human genes 0.000 claims description 2
- 230000004568 DNA-binding Effects 0.000 claims description 2
- 108020004682 Single-Stranded DNA Proteins 0.000 claims description 2
- 239000011230 binding agent Substances 0.000 claims description 2
- 239000002577 cryoprotective agent Substances 0.000 claims description 2
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 2
- 230000007935 neutral effect Effects 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 239000012867 bioactive agent Substances 0.000 description 3
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 239000002773 nucleotide Substances 0.000 description 2
- 125000003729 nucleotide group Chemical group 0.000 description 2
- 125000000753 cycloalkyl group Chemical group 0.000 description 1
- 230000009977 dual effect Effects 0.000 description 1
- 238000003209 gene knockout Methods 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
- 230000003007 single stranded DNA break Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024146107A JP2024167309A (ja) | 2018-12-05 | 2024-08-28 | 修飾されたアミン脂質 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862775783P | 2018-12-05 | 2018-12-05 | |
| US62/775,783 | 2018-12-05 | ||
| PCT/US2019/064663 WO2020118041A1 (en) | 2018-12-05 | 2019-12-05 | Modified amine lipids |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024146107A Division JP2024167309A (ja) | 2018-12-05 | 2024-08-28 | 修飾されたアミン脂質 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022514214A JP2022514214A (ja) | 2022-02-10 |
| JPWO2020118041A5 true JPWO2020118041A5 (https=) | 2023-02-13 |
| JP2022514214A5 JP2022514214A5 (https=) | 2023-02-13 |
| JP7547335B2 JP7547335B2 (ja) | 2024-09-09 |
Family
ID=69006051
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021531946A Active JP7547335B2 (ja) | 2018-12-05 | 2019-12-05 | 修飾されたアミン脂質 |
| JP2024146107A Pending JP2024167309A (ja) | 2018-12-05 | 2024-08-28 | 修飾されたアミン脂質 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024146107A Pending JP2024167309A (ja) | 2018-12-05 | 2024-08-28 | 修飾されたアミン脂質 |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US12077483B2 (https=) |
| EP (1) | EP3891127A1 (https=) |
| JP (2) | JP7547335B2 (https=) |
| KR (1) | KR20210108969A (https=) |
| CN (2) | CN113260607A (https=) |
| AU (1) | AU2019392673A1 (https=) |
| BR (1) | BR112021010853A2 (https=) |
| CA (1) | CA3121621A1 (https=) |
| CO (1) | CO2021008838A2 (https=) |
| EA (1) | EA202191559A1 (https=) |
| IL (2) | IL314386A (https=) |
| MA (1) | MA54385A (https=) |
| MX (1) | MX2021006150A (https=) |
| PH (1) | PH12021551279A1 (https=) |
| SA (1) | SA521422179B1 (https=) |
| SG (1) | SG11202105367QA (https=) |
| TW (2) | TW202504886A (https=) |
| UA (1) | UA129215C2 (https=) |
| WO (1) | WO2020118041A1 (https=) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL314386A (en) * | 2018-12-05 | 2024-09-01 | Intellia Therapeutics Inc | amine modified lipids |
| WO2020219876A1 (en) * | 2019-04-25 | 2020-10-29 | Intellia Therapeutics, Inc. | Ionizable amine lipids and lipid nanoparticles |
| US12569438B2 (en) | 2019-07-29 | 2026-03-10 | Georgia Tech Research Corporation | Nanomaterials containing constrained lipids and uses thereof |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| US12435035B2 (en) | 2020-01-09 | 2025-10-07 | Guide Therapeutics, Llc | Nanomaterials |
| KR20230017783A (ko) | 2020-04-28 | 2023-02-06 | 인텔리아 테라퓨틱스, 인크. | 시험관내 세포 전달 방법 |
| JP2023546175A (ja) | 2020-10-14 | 2023-11-01 | ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド | 脂質ナノ粒子製造の方法及びそれに由来する組成物 |
| CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
| KR20230131863A (ko) * | 2021-01-20 | 2023-09-14 | 빔 테라퓨틱스, 인크. | 나노물질 |
| IL303845A (en) | 2021-01-20 | 2023-08-01 | Beam Therapeutics Inc | Nanomaterials include a degradable property |
| US20240180954A1 (en) | 2021-02-04 | 2024-06-06 | Shionogi & Co., Ltd. | Cationic lipid |
| CR20230535A (es) | 2021-04-17 | 2024-02-16 | Intellia Therapeutics Inc | Inhibidores de proteína cinasa dependiente de adn y composiciones y usos de estos |
| KR20240032013A (ko) | 2021-06-10 | 2024-03-08 | 인텔리아 테라퓨틱스, 인크. | 유전자 편집을 위한 내부 링커를 포함하는 변형된 가이드 rna |
| KR20240090727A (ko) | 2021-10-22 | 2024-06-21 | 세일 바이오메디슨스, 인크. | Mrna 백신 조성물 |
| EP4426822A2 (en) | 2021-11-03 | 2024-09-11 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
| CN118488834A (zh) | 2021-11-23 | 2024-08-13 | 赛欧生物医药股份有限公司 | 细菌源性脂质组合物和其用途 |
| WO2023122080A1 (en) | 2021-12-20 | 2023-06-29 | Senda Biosciences, Inc. | Compositions comprising mrna and lipid reconstructed plant messenger packs |
| CN114213347B (zh) * | 2022-02-22 | 2022-05-20 | 中国科学院基础医学与肿瘤研究所(筹) | 一种双价可电离脂质化合物、组合物及其应用 |
| CN114213346B (zh) * | 2022-02-22 | 2022-05-20 | 中国科学院基础医学与肿瘤研究所(筹) | 一种双价可电离脂质化合物、组合物及其应用 |
| CN116354836A (zh) * | 2022-03-25 | 2023-06-30 | 深圳市新合生物医疗科技有限公司 | 阳离子脂质化合物及其制备方法和应用、以及mRNA递送系统 |
| CN119384498A (zh) | 2022-06-16 | 2025-01-28 | 因特利亚治疗公司 | 用于对细胞进行遗传修饰的方法和组合物 |
| WO2024019936A1 (en) * | 2022-07-20 | 2024-01-25 | Beam Therapeutics Inc. | Nanomaterials comprising triols |
| EP4615424A1 (en) | 2022-11-10 | 2025-09-17 | Sail Biomedicines, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
| WO2024138189A2 (en) | 2022-12-22 | 2024-06-27 | Intellia Therapeutics, Inc. | Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies |
| WO2024138115A1 (en) | 2022-12-23 | 2024-06-27 | Intellia Theraperutics, Inc. | Systems and methods for genomic editing |
| CN121057577A (zh) | 2023-01-27 | 2025-12-02 | 赛欧生物医药股份有限公司 | 经修饰的脂质组合物及其用途 |
| EP4698150A1 (en) | 2023-04-19 | 2026-02-25 | Sail Biomedicines, Inc. | Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids |
| WO2024220712A2 (en) | 2023-04-19 | 2024-10-24 | Sail Biomedicines, Inc. | Vaccine compositions |
| WO2024220752A2 (en) | 2023-04-19 | 2024-10-24 | Sail Biomedicines, Inc. | Rna therapeutic compositions |
| CN121241046A (zh) * | 2023-05-30 | 2025-12-30 | 解放生物股份有限公司 | 脂质化合物、组合物及其用途 |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| EP4725938A1 (en) * | 2023-06-07 | 2026-04-15 | InnoVec Biotherapeutics | Ionizable lipid compound, lipid carrier comprising same and use thereof |
| WO2025038855A1 (en) * | 2023-08-16 | 2025-02-20 | Beam Therapeutics Inc. | Ionizable lipids with branched head groups |
| WO2025049481A1 (en) | 2023-08-28 | 2025-03-06 | Intellia Therapeutics, Inc. | Methods of editing an hla-a gene in vitro |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025064401A1 (en) | 2023-09-18 | 2025-03-27 | Intellia Therapeutics, Inc. | Nuclease resistant single stranded dna product for non-viral delivery to a cell and methods of production thereof |
| WO2025064396A1 (en) | 2023-09-18 | 2025-03-27 | Intellia Therapeutics, Inc. | Nuclease resistant double stranded dna product for non-viral delivery to a cell and methods of production thereof |
| WO2025106930A1 (en) | 2023-11-17 | 2025-05-22 | Sail Biomedicines, Inc. | Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof |
| WO2025106915A1 (en) | 2023-11-17 | 2025-05-22 | Sail Biomedicines, Inc. | Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025137301A1 (en) | 2023-12-20 | 2025-06-26 | Intellia Therapeutics, Inc. | Methods for rapid engineering of cells |
| WO2025159966A1 (en) * | 2024-01-23 | 2025-07-31 | Beam Therapeutics Inc. | Compositions and methods for altering a nucleobase in a transthyretin polynucleotide |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025193861A2 (en) * | 2024-03-12 | 2025-09-18 | Tessera Therapeutics, Inc. | Lipid nanoparticles for delivery of therapeutic payloads to cells |
| WO2025240946A1 (en) | 2024-05-17 | 2025-11-20 | Intellia Therapeutics, Inc. | Lipid nanoparticles and lipid nanoparticle compositions |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| CN121620500A (zh) * | 2024-06-27 | 2026-03-06 | 因诺纬克生物科技香港有限公司 | 含醚新型可电离脂质及其用途 |
| WO2026035680A1 (en) | 2024-08-06 | 2026-02-12 | Kernal Biologics, Inc. | Compositions and methods for delivery of agents |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030083272A1 (en) | 1997-09-19 | 2003-05-01 | Lahive & Cockfield, Llp | Sense mrna therapy |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| WO2011076807A2 (en) | 2009-12-23 | 2011-06-30 | Novartis Ag | Lipids, lipid compositions, and methods of using them |
| SG186085A1 (en) * | 2010-06-03 | 2013-01-30 | Alnylam Pharmaceuticals Inc | Biodegradable lipids for the delivery of active agents |
| EP3354644A1 (en) * | 2011-06-08 | 2018-08-01 | Translate Bio, Inc. | Cleavable lipids |
| DK2931897T3 (en) * | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
| EP3608308B1 (en) * | 2013-03-08 | 2021-07-21 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| EP4019506A1 (en) * | 2013-12-19 | 2022-06-29 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| RU2571176C1 (ru) * | 2014-07-14 | 2015-12-20 | Гиа Маргович Гвичия | Светодиодная матрица |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| US10125092B2 (en) | 2014-09-05 | 2018-11-13 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| JP6531983B2 (ja) * | 2015-07-31 | 2019-06-19 | パナソニックＩｐマネジメント株式会社 | 自動運転装置、自動運転支援方法及び自動運転支援プログラム |
| EP4286012A3 (en) | 2015-09-17 | 2024-05-29 | ModernaTX, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| TWI773666B (zh) | 2016-03-30 | 2022-08-11 | 美商英特利亞醫療公司 | Crispr/cas 組分之脂質奈米粒子調配物 |
| EP3538515B1 (en) * | 2016-11-08 | 2022-07-20 | Ramot at Tel-Aviv University Ltd. | Cationic lipids for nucleic acid delivery and preparation thereof |
| IL314386A (en) | 2018-12-05 | 2024-09-01 | Intellia Therapeutics Inc | amine modified lipids |
-
2019
- 2019-12-05 IL IL314386A patent/IL314386A/en unknown
- 2019-12-05 MA MA054385A patent/MA54385A/fr unknown
- 2019-12-05 TW TW113114385A patent/TW202504886A/zh unknown
- 2019-12-05 EP EP19828120.6A patent/EP3891127A1/en active Pending
- 2019-12-05 US US17/299,925 patent/US12077483B2/en active Active
- 2019-12-05 MX MX2021006150A patent/MX2021006150A/es unknown
- 2019-12-05 JP JP2021531946A patent/JP7547335B2/ja active Active
- 2019-12-05 UA UAA202103727A patent/UA129215C2/uk unknown
- 2019-12-05 AU AU2019392673A patent/AU2019392673A1/en not_active Abandoned
- 2019-12-05 KR KR1020217020770A patent/KR20210108969A/ko not_active Ceased
- 2019-12-05 WO PCT/US2019/064663 patent/WO2020118041A1/en not_active Ceased
- 2019-12-05 BR BR112021010853-0A patent/BR112021010853A2/pt unknown
- 2019-12-05 IL IL283622A patent/IL283622B2/en unknown
- 2019-12-05 CA CA3121621A patent/CA3121621A1/en active Pending
- 2019-12-05 SG SG11202105367QA patent/SG11202105367QA/en unknown
- 2019-12-05 CN CN201980086265.9A patent/CN113260607A/zh active Pending
- 2019-12-05 TW TW108144579A patent/TWI841639B/zh active
- 2019-12-05 CN CN202410977955.6A patent/CN119285480A/zh active Pending
- 2019-12-05 EA EA202191559A patent/EA202191559A1/ru unknown
-
2021
- 2021-06-01 PH PH12021551279A patent/PH12021551279A1/en unknown
- 2021-06-03 SA SA521422179A patent/SA521422179B1/ar unknown
- 2021-07-02 CO CONC2021/0008838A patent/CO2021008838A2/es unknown
-
2024
- 2024-08-06 US US18/796,047 patent/US20250074866A1/en active Pending
- 2024-08-28 JP JP2024146107A patent/JP2024167309A/ja active Pending
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