JP2023029966A5 - - Google Patents
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- JP2023029966A5 JP2023029966A5 JP2022194564A JP2022194564A JP2023029966A5 JP 2023029966 A5 JP2023029966 A5 JP 2023029966A5 JP 2022194564 A JP2022194564 A JP 2022194564A JP 2022194564 A JP2022194564 A JP 2022194564A JP 2023029966 A5 JP2023029966 A5 JP 2023029966A5
- Authority
- JP
- Japan
- Prior art keywords
- guide
- seq
- nucleotides
- sequence
- rna
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025112164A JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566236P | 2017-09-29 | 2017-09-29 | |
| US62/566,236 | 2017-09-29 | ||
| US201862671902P | 2018-05-15 | 2018-05-15 | |
| US62/671,902 | 2018-05-15 | ||
| JP2020517500A JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| PCT/US2018/053382 WO2019067872A1 (en) | 2017-09-29 | 2018-09-28 | COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Division JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025112164A Division JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023029966A JP2023029966A (ja) | 2023-03-07 |
| JP2023029966A5 true JP2023029966A5 (https=) | 2023-06-02 |
| JP7707140B2 JP7707140B2 (ja) | 2025-07-14 |
Family
ID=63858214
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Pending JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2022194564A Active JP7707140B2 (ja) | 2017-09-29 | 2022-12-06 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020517500A Pending JP2021500864A (ja) | 2017-09-29 | 2018-09-28 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025112164A Pending JP2025163016A (ja) | 2017-09-29 | 2025-07-02 | Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法 |
Country Status (16)
| Country | Link |
|---|---|
| US (4) | US20200248180A1 (https=) |
| EP (1) | EP3688161A1 (https=) |
| JP (3) | JP2021500864A (https=) |
| KR (1) | KR20200058509A (https=) |
| CN (2) | CN118530993A (https=) |
| AU (2) | AU2018338787C1 (https=) |
| BR (1) | BR112020005287A2 (https=) |
| CA (1) | CA3077251A1 (https=) |
| CO (1) | CO2020005116A2 (https=) |
| IL (3) | IL311170B1 (https=) |
| MX (2) | MX2020003608A (https=) |
| MY (1) | MY203849A (https=) |
| PH (1) | PH12020550364A1 (https=) |
| SG (1) | SG11202002565YA (https=) |
| TW (1) | TWI904069B (https=) |
| WO (1) | WO2019067872A1 (https=) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| US20190098879A1 (en) | 2017-09-29 | 2019-04-04 | Regeneron Pharmaceuticals, Inc. | Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| MX2021011757A (es) * | 2019-03-28 | 2021-12-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la expresión de polipéptidos. |
| JP7631215B2 (ja) * | 2019-03-28 | 2025-02-18 | インテリア セラピューティクス，インコーポレイテッド | Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
| AU2020248337A1 (en) * | 2019-03-28 | 2021-11-04 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof |
| US11891618B2 (en) | 2019-06-04 | 2024-02-06 | Regeneron Pharmaceuticals, Inc. | Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| EP4073249A1 (en) * | 2019-12-11 | 2022-10-19 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
| JP2023548005A (ja) * | 2020-10-28 | 2023-11-15 | ノヴォ ノルディスク アー／エス | 抗トランスサイレチン抗体およびその使用方法 |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing involving deamination |
| CN112795595A (zh) * | 2020-12-24 | 2021-05-14 | 中山大学 | 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统 |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| KR20240038705A (ko) | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| US20250127927A1 (en) * | 2021-08-23 | 2025-04-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells |
| AU2022345881A1 (en) | 2021-09-20 | 2024-03-21 | Alnylam Pharmaceuticals, Inc. | Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof |
| CN116064598B (zh) * | 2021-10-08 | 2024-03-12 | 苏州艾博生物科技有限公司 | 冠状病毒的核酸疫苗 |
| WO2023185697A2 (en) * | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| WO2024003810A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Guide rna with chemical modifications |
| CN118853659A (zh) * | 2023-04-14 | 2024-10-29 | 深圳瑞吉生物科技有限公司 | 提高mRNA翻译蛋白的输出量的核酸及其应用 |
| WO2024259135A1 (en) | 2023-06-13 | 2024-12-19 | Intellia Therapeutics, Inc. | Assays for analysis of ribonucleic acid (rna) molecules |
| CN120225668A (zh) * | 2023-10-25 | 2025-06-27 | 石药集团中奇制药技术（石家庄）有限公司 | 一种高效低脱靶的基因编辑工具 |
| 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 |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| JP3131222B2 (ja) | 1991-12-24 | 2001-01-31 | アイシス・ファーマシューティカルス・インコーポレーテッド | ギャップを有する２′修飾オリゴヌクレオチド |
| JPH10500310A (ja) | 1994-05-19 | 1998-01-13 | ダコ アクティーゼルスカブ | 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ |
| US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US7244764B2 (en) * | 2003-06-23 | 2007-07-17 | Neurochem (International) Limited | Methods and compositions for treating amyloid-related diseases |
| US20060051405A1 (en) | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| EP2323667A4 (en) | 2008-08-07 | 2012-07-25 | Isis Pharmaceuticals Inc | MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES |
| MX360460B (es) | 2008-10-20 | 2018-11-05 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de transtiretina. |
| US9101643B2 (en) | 2009-11-03 | 2015-08-11 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR) |
| PL2563920T3 (pl) | 2010-04-29 | 2017-08-31 | Ionis Pharmaceuticals, Inc. | Modulacja ekspresji transtyretyny |
| EP3730618A1 (en) | 2011-11-18 | 2020-10-28 | Alnylam Pharmaceuticals, Inc. | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| EP3031921B1 (en) * | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CN107879960B (zh) | 2013-03-08 | 2021-06-22 | 诺华股份有限公司 | 用于传递活性成分的脂质和脂质组合物 |
| DK3456831T3 (da) * | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| DK2992098T3 (da) | 2013-05-01 | 2019-06-17 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression |
| CN105683379A (zh) * | 2013-06-17 | 2016-06-15 | 布罗德研究所有限公司 | 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| WO2015183025A1 (ko) * | 2014-05-28 | 2015-12-03 | 주식회사 툴젠 | 표적 특이적 뉴클레아제를 이용한 표적 dna의 민감한 검출 방법 |
| SI3161128T1 (sl) * | 2014-06-26 | 2019-02-28 | Regeneron Pharmaceuticals, Inc. | Postopki in sestavki za ciljano genetsko spremembo in postopki uporabe |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| CN120842296A (zh) | 2015-09-21 | 2025-10-28 | 垂林克生物技术有限公司 | 用于合成5’-加帽rna的组合物和方法 |
| JP6799058B2 (ja) * | 2015-09-21 | 2020-12-09 | 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Ａｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | アレル選択的な遺伝子編集およびその使用 |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| CN109715801B (zh) * | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
| EP3429632B1 (en) * | 2016-03-16 | 2023-01-04 | CRISPR Therapeutics AG | Materials and methods for treatment of hereditary haemochromatosis |
| LT3436077T (lt) * | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| SG10202106058WA (en) | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
-
2018
- 2018-09-28 EP EP18786638.9A patent/EP3688161A1/en active Pending
- 2018-09-28 JP JP2020517500A patent/JP2021500864A/ja active Pending
- 2018-09-28 CN CN202410646325.0A patent/CN118530993A/zh active Pending
- 2018-09-28 MX MX2020003608A patent/MX2020003608A/es unknown
- 2018-09-28 IL IL311170A patent/IL311170B1/en unknown
- 2018-09-28 CN CN201880076628.6A patent/CN111417728A/zh active Pending
- 2018-09-28 KR KR1020207012163A patent/KR20200058509A/ko active Pending
- 2018-09-28 MY MYPI2020001489A patent/MY203849A/en unknown
- 2018-09-28 WO PCT/US2018/053382 patent/WO2019067872A1/en not_active Ceased
- 2018-09-28 CA CA3077251A patent/CA3077251A1/en active Pending
- 2018-09-28 AU AU2018338787A patent/AU2018338787C1/en active Active
- 2018-09-28 TW TW107134336A patent/TWI904069B/zh active
- 2018-09-28 BR BR112020005287-6A patent/BR112020005287A2/pt unknown
- 2018-09-28 SG SG11202002565YA patent/SG11202002565YA/en unknown
-
2020
- 2020-03-15 IL IL273317A patent/IL273317A/en unknown
- 2020-03-24 US US16/828,573 patent/US20200248180A1/en not_active Abandoned
- 2020-03-27 PH PH12020550364A patent/PH12020550364A1/en unknown
- 2020-04-24 CO CONC2020/0005116A patent/CO2020005116A2/es unknown
- 2020-07-13 MX MX2025014378A patent/MX2025014378A/es unknown
-
2022
- 2022-12-06 JP JP2022194564A patent/JP7707140B2/ja active Active
- 2022-12-09 US US18/064,086 patent/US11795460B2/en active Active
- 2022-12-09 US US18/063,972 patent/US11965165B2/en active Active
-
2024
- 2024-11-07 US US18/940,080 patent/US20250207130A1/en active Pending
-
2025
- 2025-04-17 AU AU2025202742A patent/AU2025202742A1/en active Pending
- 2025-07-02 JP JP2025112164A patent/JP2025163016A/ja active Pending
- 2025-12-18 IL IL325460A patent/IL325460A/en unknown
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