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
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021531804A JP2021531804A (ja) | 2021-11-25 |
| JPWO2020028327A5 JPWO2020028327A5 (https=) | 2022-09-05 |
| JP2021531804A5 true JP2021531804A5 (https=) | 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 (https=) |
| EP (1) | EP3830267A1 (https=) |
| JP (1) | JP2021531804A (https=) |
| KR (1) | KR20210053888A (https=) |
| CN (1) | CN112867795A (https=) |
| AU (1) | AU2019313348A1 (https=) |
| BR (1) | BR112021001546A2 (https=) |
| CA (1) | CA3113190A1 (https=) |
| CO (1) | CO2021002691A2 (https=) |
| MX (1) | MX2021001070A (https=) |
| TW (1) | TW202020156A (https=) |
| WO (1) | WO2020028327A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220090047A1 (en) * | 2018-12-21 | 2022-03-24 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| CA3172481A1 (en) | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
| 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 |
| CA3222159A1 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof |
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | 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 |
| KR940703846A (ko) | 1991-12-24 | 1994-12-12 | 비. 린네 파샬 | 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides) |
| US6169169B1 (en) | 1994-05-19 | 2001-01-02 | Dako A/S | PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis |
| EP3241902B1 (en) * | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| DK2931898T3 (en) | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS |
| CA2895155C (en) | 2012-12-17 | 2021-07-06 | President And Fellows Of Harvard College | Rna-guided human genome engineering |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| WO2015100436A1 (en) * | 2013-12-27 | 2015-07-02 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| ES3047792T3 (en) * | 2014-07-14 | 2025-12-04 | Univ California | Crispr/cas transcriptional modulation |
| EP3169309B1 (en) | 2014-07-16 | 2023-05-10 | Novartis AG | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
| EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | 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 |
| CN109310784B (zh) * | 2015-12-07 | 2022-08-19 | 阿克生物公司 | 用于制备和使用指导核酸的方法和组合物 |
| US11845933B2 (en) | 2016-02-03 | 2023-12-19 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide RNA and its applications |
| 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 |
| WO2018049009A2 (en) * | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
-
2019
- 2019-07-30 BR BR112021001546-9A patent/BR112021001546A2/pt not_active IP Right Cessation
- 2019-07-30 AU AU2019313348A patent/AU2019313348A1/en not_active Withdrawn
- 2019-07-30 CA CA3113190A patent/CA3113190A1/en active Pending
- 2019-07-30 MX MX2021001070A patent/MX2021001070A/es unknown
- 2019-07-30 KR KR1020217005361A patent/KR20210053888A/ko not_active Withdrawn
- 2019-07-30 CN CN201980064321.9A patent/CN112867795A/zh active Pending
- 2019-07-30 EP EP19762240.0A patent/EP3830267A1/en active Pending
- 2019-07-30 TW TW108127073A patent/TW202020156A/zh unknown
- 2019-07-30 WO PCT/US2019/044080 patent/WO2020028327A1/en not_active Ceased
- 2019-07-30 JP JP2021504770A patent/JP2021531804A/ja active Pending
-
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