JP2020500539A5 - - Google Patents
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- JP2020500539A5 JP2020500539A5 JP2019530472A JP2019530472A JP2020500539A5 JP 2020500539 A5 JP2020500539 A5 JP 2020500539A5 JP 2019530472 A JP2019530472 A JP 2019530472A JP 2019530472 A JP2019530472 A JP 2019530472A JP 2020500539 A5 JP2020500539 A5 JP 2020500539A5
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- JP
- Japan
- Prior art keywords
- alkyl
- independently
- carbon
- composition
- lnp
- 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.)
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- 229910052799 carbon Inorganic materials 0.000 claims description 77
- 125000000217 alkyl group Chemical group 0.000 claims description 63
- 150000001721 carbon Chemical group 0.000 claims description 32
- 108700019146 Transgenes Proteins 0.000 claims description 22
- 238000000034 method Methods 0.000 claims description 20
- 239000008194 pharmaceutical composition Substances 0.000 claims description 20
- 108091033319 polynucleotide Proteins 0.000 claims description 19
- 102000040430 polynucleotide Human genes 0.000 claims description 19
- 239000002157 polynucleotide Substances 0.000 claims description 19
- 101710163270 Nuclease Proteins 0.000 claims description 18
- 239000011203 carbon fibre reinforced carbon Substances 0.000 claims description 18
- 125000002947 alkylene group Chemical group 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 125000004450 alkenylene group Chemical group 0.000 claims description 11
- 150000002632 lipids Chemical class 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 10
- 125000003342 alkenyl group Chemical group 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 8
- 230000007017 scission Effects 0.000 claims description 8
- 108020004414 DNA Proteins 0.000 claims description 6
- 108091023040 Transcription factor Proteins 0.000 claims description 6
- 102000040945 Transcription factor Human genes 0.000 claims description 6
- 150000001875 compounds Chemical class 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
- -1 cationic lipid Chemical class 0.000 claims description 5
- 125000005724 cycloalkenylene group Chemical group 0.000 claims description 5
- 125000002993 cycloalkylene group Chemical group 0.000 claims description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 4
- 150000003431 steroids Chemical class 0.000 claims description 4
- 108700026220 vif Genes Proteins 0.000 claims description 4
- 230000027455 binding Effects 0.000 claims description 3
- 150000003839 salts Chemical class 0.000 claims description 3
- 230000001225 therapeutic effect Effects 0.000 claims description 3
- 125000004070 6 membered heterocyclic group Chemical group 0.000 claims description 2
- 125000003341 7 membered heterocyclic group Chemical group 0.000 claims description 2
- 108010088751 Albumins Proteins 0.000 claims description 2
- 102000009027 Albumins Human genes 0.000 claims description 2
- 101150017501 CCR5 gene Proteins 0.000 claims description 2
- 230000004568 DNA-binding Effects 0.000 claims description 2
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 claims description 2
- 108010091358 Hypoxanthine Phosphoribosyltransferase Proteins 0.000 claims description 2
- 235000011449 Rosa Nutrition 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 101710185494 Zinc finger protein Proteins 0.000 claims description 2
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 2
- 125000004432 carbon atom Chemical group C* 0.000 claims description 2
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000012239 gene modification Methods 0.000 claims description 2
- 230000005017 genetic modification Effects 0.000 claims description 2
- 235000013617 genetically modified food Nutrition 0.000 claims description 2
- 125000000623 heterocyclic group Chemical group 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 230000010354 integration Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000002105 nanoparticle Substances 0.000 claims description 2
- 230000007935 neutral effect Effects 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 229920000642 polymer Polymers 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 230000002103 transcriptional effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 21
- 241001481833 Coryphaena hippurus Species 0.000 claims 1
- 241001125840 Coryphaenidae Species 0.000 claims 1
- 125000003710 aryl alkyl group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 0 CC(CCOC(C)C(N(*)*)=O)OC Chemical compound CC(CCOC(C)C(N(*)*)=O)OC 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 239000000651 prodrug Substances 0.000 description 1
- 229940002612 prodrug Drugs 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022101160A JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662432042P | 2016-12-09 | 2016-12-09 | |
| US62/432,042 | 2016-12-09 | ||
| US201762458373P | 2017-02-13 | 2017-02-13 | |
| US62/458,373 | 2017-02-13 | ||
| US201762503470P | 2017-05-09 | 2017-05-09 | |
| US62/503,470 | 2017-05-09 | ||
| US201762559186P | 2017-09-15 | 2017-09-15 | |
| US62/559,186 | 2017-09-15 | ||
| PCT/US2017/065303 WO2018107026A1 (en) | 2016-12-09 | 2017-12-08 | Delivery of target specific nucleases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022101160A Division JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020500539A JP2020500539A (ja) | 2020-01-16 |
| JP2020500539A5 true JP2020500539A5 (https=) | 2021-01-21 |
Family
ID=62492169
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019530472A Pending JP2020500539A (ja) | 2016-12-09 | 2017-12-08 | 標的特異的ヌクレアーゼの送達 |
| JP2022101160A Pending JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022101160A Pending JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20180185516A1 (https=) |
| EP (1) | EP3551169A4 (https=) |
| JP (2) | JP2020500539A (https=) |
| AU (1) | AU2017374042C1 (https=) |
| CA (1) | CA3045122A1 (https=) |
| WO (1) | WO2018107026A1 (https=) |
Families Citing this family (79)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE060907T2 (hu) | 2014-06-25 | 2023-04-28 | Acuitas Therapeutics Inc | Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére |
| PT3313829T (pt) | 2015-06-29 | 2024-07-08 | Acuitas Therapeutics Inc | Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos |
| HRP20230209T1 (hr) | 2015-10-28 | 2023-04-14 | Acuitas Therapeutics Inc. | Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina |
| EP3995574A1 (en) | 2016-08-24 | 2022-05-11 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
| KR102455249B1 (ko) | 2016-08-24 | 2022-10-17 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
| KR102712926B1 (ko) | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| WO2018191657A1 (en) | 2017-04-13 | 2018-10-18 | Acuitas Therapeutics, Inc. | Lipids for delivery of active agents |
| CN110799492B (zh) * | 2017-04-28 | 2023-06-27 | 爱康泰生治疗公司 | 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂 |
| KR20200033259A (ko) | 2017-07-31 | 2020-03-27 | 리제너론 파마슈티칼스 인코포레이티드 | 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물 |
| CN110891420B (zh) | 2017-07-31 | 2022-06-03 | 瑞泽恩制药公司 | Cas转基因小鼠胚胎干细胞和小鼠及其应用 |
| CA3073020A1 (en) | 2017-08-16 | 2019-02-21 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| EP4501322A3 (en) | 2017-08-17 | 2025-04-16 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| WO2019036030A1 (en) | 2017-08-17 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS |
| WO2019036028A1 (en) | 2017-08-17 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| CA3089587A1 (en) | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| MA52074A (fr) | 2018-03-19 | 2021-01-27 | Bayer Healthcare Llc | Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| US12479791B2 (en) | 2018-08-10 | 2025-11-25 | Takeda Pharmaceutical Company Limited | Cationic lipid |
| EP3842538A4 (en) | 2018-08-10 | 2022-06-08 | Kyoto University | METHOD OF TRANSFECTION INTO CARDIOMYOCYTES USING CATIONIC LIPID |
| KR20210060533A (ko) * | 2018-09-18 | 2021-05-26 | 상가모 테라퓨틱스, 인코포레이티드 | 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제 |
| IL281615B2 (en) | 2018-09-21 | 2026-01-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| HRP20240586T1 (hr) | 2018-10-09 | 2024-07-19 | The University Of British Columbia | Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima |
| CA3116576A1 (en) * | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| CN113710799B (zh) * | 2018-11-28 | 2024-11-12 | 克里斯珀医疗股份公司 | 用于在LNP中使用的编码CAS9的优化mRNA |
| PT3908568T (pt) | 2019-01-11 | 2024-09-30 | Acuitas Therapeutics Inc | Lípidos para a administração de agentes ativos por nanopartículas lipídicas |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| JP7630904B2 (ja) * | 2019-07-23 | 2025-02-18 | 株式会社東芝 | 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法 |
| CN114901253A (zh) * | 2019-08-14 | 2022-08-12 | 爱康泰生治疗公司 | 用于递送核酸的改进的脂质纳米颗粒 |
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| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
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| US12194157B2 (en) | 2020-04-09 | 2025-01-14 | Finncure Oy | Carrier for targeted delivery to a host |
| FI20215508A1 (en) | 2020-04-09 | 2021-10-10 | Niemelae Erik Johan | Mimetic nanoparticles to prevent the spread of new coronaviruses and reduce the rate of infection |
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| JP2023534035A (ja) * | 2020-07-15 | 2023-08-07 | ザ ジャクソン ラボラトリー | ＳＡＲＳ－ＣｏＶ－２感染のためのヒト化マウスモデル |
| ES3054438T3 (en) | 2020-07-16 | 2026-02-03 | Acuitas Therapeutics Inc | Cationic lipids for use in lipid nanoparticles |
| JP2023549011A (ja) | 2020-09-15 | 2023-11-22 | ヴァーヴ・セラピューティクス，インコーポレーテッド | 遺伝子編集のための脂質製剤 |
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| WO2022236010A1 (en) * | 2021-05-06 | 2022-11-10 | The University Of North Carolina At Chapel Hill | Use of chemical epigenetic modifiers to modulate gene expression from vectors |
| EP4367242A2 (en) | 2021-07-07 | 2024-05-15 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
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| AR127312A1 (es) * | 2021-10-08 | 2024-01-10 | Suzhou Abogen Biosciences Co Ltd | Compuestos lipídicos ycomposiciones de nanopartículas lipídicas |
| WO2023069948A1 (en) | 2021-10-18 | 2023-04-27 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| EP4422698A1 (en) | 2021-10-29 | 2024-09-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| CA3242402A1 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
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| EP4608442A1 (en) | 2022-10-28 | 2025-09-03 | GlaxoSmithKline Biologicals S.A. | Nucleic acid based vaccine |
| WO2024133160A1 (en) | 2022-12-19 | 2024-06-27 | Glaxosmithkline Biologicals Sa | Hepatitis b compositions |
| WO2024184500A1 (en) | 2023-03-08 | 2024-09-12 | CureVac SE | Novel lipid nanoparticle formulations for delivery of nucleic acids |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| EP4716741A2 (en) | 2023-05-23 | 2026-04-01 | Flagship Labs 114, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| WO2025010554A1 (en) * | 2023-07-07 | 2025-01-16 | Yoltech Therapeutics Co., Ltd. | Lipid compounds for delivery of therapeutic agents and preparation method and its use thereof |
| US20250161227A1 (en) | 2023-11-17 | 2025-05-22 | Acuitas Therapeutics, Inc. | Pegylated lipids |
| WO2025128696A1 (en) | 2023-12-12 | 2025-06-19 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025132122A1 (en) | 2023-12-13 | 2025-06-26 | Berlin Institute Of Health | Methods of delivering therapeutics using lipid nanoparticles |
| WO2025126071A1 (en) | 2023-12-14 | 2025-06-19 | Pfizer Inc. | Rna molecules |
| WO2025129128A1 (en) | 2023-12-15 | 2025-06-19 | Vivasor, Inc. | Compositions and methods for non-viral delivery of therapeutic compounds |
| WO2025174858A1 (en) | 2024-02-15 | 2025-08-21 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025184508A1 (en) | 2024-03-01 | 2025-09-04 | Acuitas Therapeutics, Inc. | Materials and methods for encapsulating therapeutics in lipid nanoparticles |
| WO2025186725A2 (en) | 2024-03-06 | 2025-09-12 | Pfizer Inc. | Improved lnp formulations and uses thereof |
| WO2025217264A1 (en) | 2024-04-10 | 2025-10-16 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025231114A1 (en) | 2024-05-01 | 2025-11-06 | Acuitas Therapeutics, Inc. | Method of using lipid nanoparticles for intramuscular delivery |
| WO2025229572A1 (en) | 2024-05-01 | 2025-11-06 | Glaxosmithkline Biologicals Sa | Epstein-barr virus antigen-encoding messenger ribonucleic acid and antigen protein vaccines |
| WO2025240833A1 (en) | 2024-05-17 | 2025-11-20 | Acuitas Therapeutics, Inc. | Galnac lipid compounds for use in lipid nanoparticles |
| WO2026018181A1 (en) | 2024-07-17 | 2026-01-22 | Pfizer Inc. | Immunogenic compositions and uses thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2848417C (en) * | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3018046A1 (en) * | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| AU2013240051B2 (en) * | 2012-03-27 | 2017-11-30 | Sirna Therapeutics, Inc. | Diether based biodegradable cationic lipids for siRNA delivery |
| US9255250B2 (en) * | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| CN105940013B (zh) * | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| HUE060907T2 (hu) * | 2014-06-25 | 2023-04-28 | Acuitas Therapeutics Inc | Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére |
| WO2016014794A1 (en) * | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
| AU2016253972B2 (en) * | 2015-04-27 | 2020-01-02 | Acuitas Therapeutics Inc. | Nucleoside-modified RNA for inducing an adaptive immune response |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
-
2017
- 2017-12-08 AU AU2017374042A patent/AU2017374042C1/en not_active Ceased
- 2017-12-08 US US15/835,957 patent/US20180185516A1/en not_active Abandoned
- 2017-12-08 JP JP2019530472A patent/JP2020500539A/ja active Pending
- 2017-12-08 EP EP17879571.2A patent/EP3551169A4/en not_active Withdrawn
- 2017-12-08 CA CA3045122A patent/CA3045122A1/en active Pending
- 2017-12-08 WO PCT/US2017/065303 patent/WO2018107026A1/en not_active Ceased
-
2022
- 2022-06-23 JP JP2022101160A patent/JP2022121531A/ja active Pending
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