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 |
|---|---|---|---|---|
| WO2020132659A1 (en) * | 2018-12-21 | 2020-06-25 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| JP7642809B2 (ja) | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing involving deamination |
| US20230034581A1 (en) | 2021-06-04 | 2023-02-02 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| IL308803A (en) * | 2021-06-04 | 2024-01-01 | Arbor Biotechnologies Inc | Gene editing systems comprising guide RNA targeting hydroxy acid 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 |
| 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 |
| AU2013266968B2 (en) * | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| 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 |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| 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 |
| EP3581654B1 (en) * | 2013-12-27 | 2021-03-31 | 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 |
| CA2954791C (en) * | 2014-07-14 | 2025-11-18 | 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 |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| EP3310918B1 (en) * | 2015-06-18 | 2020-08-05 | Alnylam Pharmaceuticals, Inc. | Polynucleotide 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 |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| CA3035534A1 (en) * | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| SG10202106058WA (en) | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
-
2019
- 2019-07-30 MX MX2021001070A patent/MX2021001070A/es unknown
- 2019-07-30 WO PCT/US2019/044080 patent/WO2020028327A1/en not_active Ceased
- 2019-07-30 TW TW108127073A patent/TW202020156A/zh unknown
- 2019-07-30 AU AU2019313348A patent/AU2019313348A1/en not_active Withdrawn
- 2019-07-30 KR KR1020217005361A patent/KR20210053888A/ko not_active Withdrawn
- 2019-07-30 BR BR112021001546-9A patent/BR112021001546A2/pt not_active IP Right Cessation
- 2019-07-30 CN CN201980064321.9A patent/CN112867795A/zh active Pending
- 2019-07-30 JP JP2021504770A patent/JP2021531804A/ja active Pending
- 2019-07-30 CA CA3113190A patent/CA3113190A1/en active Pending
- 2019-07-30 EP EP19762240.0A patent/EP3830267A1/en 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