JP2021531804A5 - - Google Patents
Info
- Publication number
- JP2021531804A5 JP2021531804A5 JP2021504770A JP2021504770A JP2021531804A5 JP 2021531804 A5 JP2021531804 A5 JP 2021531804A5 JP 2021504770 A JP2021504770 A JP 2021504770A JP 2021504770 A JP2021504770 A JP 2021504770A JP 2021531804 A5 JP2021531804 A5 JP 2021531804A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- sequence
- guide
- inventions
- 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.)
- Pending
Links
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862712904P | 2018-07-31 | 2018-07-31 | |
| US62/712,904 | 2018-07-31 | ||
| US201862738936P | 2018-09-28 | 2018-09-28 | |
| US62/738,936 | 2018-09-28 | ||
| US201962834328P | 2019-04-15 | 2019-04-15 | |
| US62/834,328 | 2019-04-15 | ||
| US201962841734P | 2019-05-01 | 2019-05-01 | |
| US62/841,734 | 2019-05-01 | ||
| PCT/US2019/044080 WO2020028327A1 (en) | 2018-07-31 | 2019-07-30 | Compositions and methods for hydroxyacid oxidase 1 ( hao1) gene editing for treating primary hyperoxaluria type 1 (ph1) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021531804A JP2021531804A (ja) | 2021-11-25 |
| JP2021531804A5 true JP2021531804A5 (enExample) | 2022-09-05 |
Family
ID=67810993
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021504770A Pending JP2021531804A (ja) | 2018-07-31 | 2019-07-30 | 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20210163943A1 (enExample) |
| EP (1) | EP3830267A1 (enExample) |
| JP (1) | JP2021531804A (enExample) |
| KR (1) | KR20210053888A (enExample) |
| CN (1) | CN112867795A (enExample) |
| AU (1) | AU2019313348A1 (enExample) |
| BR (1) | BR112021001546A2 (enExample) |
| CA (1) | CA3113190A1 (enExample) |
| CO (1) | CO2021002691A2 (enExample) |
| MX (1) | MX2021001070A (enExample) |
| TW (1) | TW202020156A (enExample) |
| WO (1) | WO2020028327A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3898661A1 (en) * | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| KR20250099412A (ko) | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| EP4259792A1 (en) | 2020-12-11 | 2023-10-18 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing involving deamination |
| WO2022256655A2 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| KR20240034746A (ko) * | 2021-06-04 | 2024-03-14 | 아버 바이오테크놀로지스, 인크. | 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도 |
| EP4771154A2 (en) | 2023-09-01 | 2026-07-08 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| 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 |
| 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 |
| CN118086297B (zh) * | 2024-02-28 | 2025-10-03 | 复旦大学附属儿科医院 | 用于人HAO1基因编辑的sgRNA、组合物及其应用 |
| CN117925623B (zh) * | 2024-03-20 | 2024-06-07 | 北京引正基因科技有限公司 | 用于hao1基因编辑和治疗ph1的组合物 |
Family Cites Families (21)
| 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 |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| ES2728782T3 (es) * | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| PT2931898E (pt) | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | Rna-guided human genome engineering |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| SI3581654T1 (sl) * | 2013-12-27 | 2021-08-31 | Dicerna Pharmaceuticals, Inc. | Postopki in sestavki za specifično inhibicijo glikolat-oksidaze (HA01) z dvoverižno RNA |
| WO2015200555A2 (en) | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
| EP3169776B1 (en) * | 2014-07-14 | 2025-09-03 | The Regents of The University of California | Crispr/cas transcriptional modulation |
| 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 |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| WO2016205323A1 (en) * | 2015-06-18 | 2016-12-22 | Alnylam Pharmaceuticals, Inc. | Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof |
| AU2016365720B2 (en) * | 2015-12-07 | 2020-11-26 | Arc Bio, Llc | Methods and compositions for the making and using of guide nucleic acids |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| JP7256739B2 (ja) * | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス， インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
-
2019
- 2019-07-30 MX MX2021001070A patent/MX2021001070A/es unknown
- 2019-07-30 CA CA3113190A patent/CA3113190A1/en active Pending
- 2019-07-30 TW TW108127073A patent/TW202020156A/zh unknown
- 2019-07-30 AU AU2019313348A patent/AU2019313348A1/en not_active Withdrawn
- 2019-07-30 CN CN201980064321.9A patent/CN112867795A/zh active Pending
- 2019-07-30 WO PCT/US2019/044080 patent/WO2020028327A1/en not_active Ceased
- 2019-07-30 JP JP2021504770A patent/JP2021531804A/ja active Pending
- 2019-07-30 BR BR112021001546-9A patent/BR112021001546A2/pt not_active IP Right Cessation
- 2019-07-30 EP EP19762240.0A patent/EP3830267A1/en active Pending
- 2019-07-30 KR KR1020217005361A patent/KR20210053888A/ko not_active Withdrawn
-
2021
- 2021-01-29 US US17/162,377 patent/US20210163943A1/en not_active Abandoned
- 2021-02-26 CO CONC2021/0002691A patent/CO2021002691A2/es unknown
-
2025
- 2025-06-23 US US19/246,289 patent/US20260043026A1/en active Pending
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