JP2022502055A5 - - Google Patents
Info
- Publication number
- JP2022502055A5 JP2022502055A5 JP2021517350A JP2021517350A JP2022502055A5 JP 2022502055 A5 JP2022502055 A5 JP 2022502055A5 JP 2021517350 A JP2021517350 A JP 2021517350A JP 2021517350 A JP2021517350 A JP 2021517350A JP 2022502055 A5 JP2022502055 A5 JP 2022502055A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- guide
- composition
- seq
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862738956P | 2018-09-28 | 2018-09-28 | |
| US62/738,956 | 2018-09-28 | ||
| US201962834334P | 2019-04-15 | 2019-04-15 | |
| US62/834,334 | 2019-04-15 | ||
| US201962841740P | 2019-05-01 | 2019-05-01 | |
| US62/841,740 | 2019-05-01 | ||
| PCT/US2019/053423 WO2020069296A1 (en) | 2018-09-28 | 2019-09-27 | Compositions and methods for lactate dehydrogenase (ldha) gene editing |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022502055A JP2022502055A (ja) | 2022-01-11 |
| JP2022502055A5 true JP2022502055A5 (enExample) | 2022-09-30 |
Family
ID=68296656
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021517350A Pending JP2022502055A (ja) | 2018-09-28 | 2019-09-27 | 乳酸デヒドロゲナーゼ（ｌｄｈａ）遺伝子編集のための組成物及び方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20210222173A1 (enExample) |
| EP (1) | EP3856901A1 (enExample) |
| JP (1) | JP2022502055A (enExample) |
| KR (1) | KR20210086621A (enExample) |
| CN (1) | CN113056559A (enExample) |
| AU (1) | AU2019347517A1 (enExample) |
| BR (1) | BR112021005718A2 (enExample) |
| CA (1) | CA3114425A1 (enExample) |
| CO (1) | CO2021005231A2 (enExample) |
| IL (1) | IL281529A (enExample) |
| MX (1) | MX2021003457A (enExample) |
| PH (1) | PH12021550686A1 (enExample) |
| SG (1) | SG11202102660RA (enExample) |
| TW (1) | TW202028460A (enExample) |
| WO (1) | WO2020069296A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP4228658A4 (en) | 2020-10-14 | 2025-02-19 | George Mason Research Foundation, Inc. | IONIZABLE LIPIDS AND METHODS FOR THE PRODUCTION AND USE THEREOF |
| 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 |
| US20240415980A1 (en) | 2021-10-28 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| WO2023122433A1 (en) * | 2021-12-22 | 2023-06-29 | Arbor Biotechnologies, Inc. | Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha) |
| CA3242731A1 (en) | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease |
| CN114657181B (zh) * | 2022-04-01 | 2023-08-25 | 安徽大学 | 一种靶向H1.4的sgRNA以及H1.4基因编辑方法 |
| WO2023212677A2 (en) | 2022-04-29 | 2023-11-02 | Regeneron Pharmaceuticals, Inc. | Identification of tissue-specific extragenic safe harbors for gene therapy approaches |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| JP2025521154A (ja) | 2022-05-31 | 2025-07-08 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法 |
| CN120659627A (zh) | 2022-07-29 | 2025-09-16 | 瑞泽恩制药公司 | 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法 |
| KR20250075694A (ko) | 2022-09-28 | 2025-05-28 | 리제너론 파마슈티칼스 인코포레이티드 | 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체 |
| EP4612184A1 (en) | 2022-11-04 | 2025-09-10 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| KR20250116795A (ko) | 2022-11-14 | 2025-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 별아교세포로의 섬유아세포 성장 인자 수용체 3-매개된 전달을 위한 조성물 및 방법 |
| IL326018A (en) | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
| CN121693566A (zh) | 2023-07-28 | 2026-03-17 | 瑞泽恩制药公司 | 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达 |
| US20250049896A1 (en) | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
| WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
| CN121752723A (zh) * | 2023-08-31 | 2026-03-27 | 正基基因科技有限公司 | 编码crispr相关蛋白的核酸及其应用 |
| 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 |
| WO2025162435A1 (en) * | 2024-02-02 | 2025-08-07 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of liver disease |
| 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 |
| US20250276092A1 (en) | 2024-03-01 | 2025-09-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response |
| WO2025235388A1 (en) | 2024-05-06 | 2025-11-13 | Regeneron Pharmaceuticals, Inc. | Transgene genomic identification by nuclease-mediated long read sequencing |
| WO2025265017A1 (en) | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
Family Cites Families (16)
| 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 |
| 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 |
| 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 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| 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 |
| AU2015330670B2 (en) * | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| 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 |
| EP3452080A4 (en) * | 2016-05-02 | 2019-10-09 | The Regents of the University of California | MAMMALIAN CELLS WITHOUT LACTATE DEHYDROGENASE ACTIVITY |
| SG10202106058WA (en) | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
-
2019
- 2019-09-27 TW TW108135340A patent/TW202028460A/zh unknown
- 2019-09-27 CA CA3114425A patent/CA3114425A1/en active Pending
- 2019-09-27 KR KR1020217012025A patent/KR20210086621A/ko not_active Withdrawn
- 2019-09-27 MX MX2021003457A patent/MX2021003457A/es unknown
- 2019-09-27 WO PCT/US2019/053423 patent/WO2020069296A1/en not_active Ceased
- 2019-09-27 EP EP19790921.1A patent/EP3856901A1/en active Pending
- 2019-09-27 BR BR112021005718-8A patent/BR112021005718A2/pt not_active IP Right Cessation
- 2019-09-27 AU AU2019347517A patent/AU2019347517A1/en not_active Withdrawn
- 2019-09-27 CN CN201980076179.XA patent/CN113056559A/zh active Pending
- 2019-09-27 JP JP2021517350A patent/JP2022502055A/ja active Pending
- 2019-09-27 SG SG11202102660RA patent/SG11202102660RA/en unknown
-
2021
- 2021-03-16 IL IL281529A patent/IL281529A/en unknown
- 2021-03-25 US US17/212,901 patent/US20210222173A1/en not_active Abandoned
- 2021-03-26 PH PH12021550686A patent/PH12021550686A1/en unknown
- 2021-04-23 CO CONC2021/0005231A patent/CO2021005231A2/es unknown
-
2025
- 2025-05-16 US US19/210,426 patent/US20260035697A1/en active Pending
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