JP2023549357A5 - - Google Patents
Info
- Publication number
- JP2023549357A5 JP2023549357A5 JP2023528230A JP2023528230A JP2023549357A5 JP 2023549357 A5 JP2023549357 A5 JP 2023549357A5 JP 2023528230 A JP2023528230 A JP 2023528230A JP 2023528230 A JP2023528230 A JP 2023528230A JP 2023549357 A5 JP2023549357 A5 JP 2023549357A5
- Authority
- JP
- Japan
- Prior art keywords
- acid sequence
- nucleic acid
- seq
- genetically modified
- polynucleotide
- 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 |
|---|---|---|---|
| JP2025028775A JP2025098002A (ja) | 2020-11-12 | 2025-02-26 | 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 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063113131P | 2020-11-12 | 2020-11-12 | |
| US63/113,131 | 2020-11-12 | ||
| US202163233664P | 2021-08-16 | 2021-08-16 | |
| US63/233,664 | 2021-08-16 | ||
| PCT/US2021/059146 WO2022104062A1 (en) | 2020-11-12 | 2021-11-12 | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025028775A Division JP2025098002A (ja) | 2020-11-12 | 2025-02-26 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023549357A JP2023549357A (ja) | 2023-11-24 |
| JP2023549357A5 true JP2023549357A5 (https=) | 2024-11-20 |
| JP7642809B2 JP7642809B2 (ja) | 2025-03-10 |
Family
ID=78844897
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023528230A Active JP7642809B2 (ja) | 2020-11-12 | 2021-11-12 | 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 |
| JP2025028775A Pending JP2025098002A (ja) | 2020-11-12 | 2025-02-26 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025028775A Pending JP2025098002A (ja) | 2020-11-12 | 2025-02-26 | 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 |
Country Status (9)
| Country | Link |
|---|---|
| US (4) | US11753630B2 (https=) |
| EP (1) | EP4244342B1 (https=) |
| JP (2) | JP7642809B2 (https=) |
| KR (2) | KR102826438B1 (https=) |
| CN (3) | CN120098962A (https=) |
| CA (1) | CA3172481A1 (https=) |
| IL (2) | IL317538A (https=) |
| MX (1) | MX2023005496A (https=) |
| WO (1) | WO2022104062A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4659765A3 (en) * | 2014-03-12 | 2026-02-18 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| US20250041450A1 (en) * | 2021-08-05 | 2025-02-06 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| CA3259572A1 (en) * | 2022-06-30 | 2024-01-04 | Sarepta Therapeutics, Inc. | TREATMENT METHODS FOR MUSCULAR DYSTROPHY |
| WO2024216116A1 (en) | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| WO2024216118A1 (en) | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| TW202546236A (zh) * | 2024-01-16 | 2025-12-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療第1型肌強直性營養不良之肌肉選擇性混合調節組合及其使用方法 |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
| US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
| US7404969B2 (en) | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| ES2582091T3 (es) | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| WO2009001159A1 (en) | 2007-06-25 | 2008-12-31 | Cellectis | Method for enhancing the cleavage activity of i-crei derived meganucleases |
| EP2215223B1 (en) | 2007-10-31 | 2013-05-01 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009086558A1 (en) | 2008-01-02 | 2009-07-09 | Tekmira Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| CN102177235A (zh) * | 2008-09-08 | 2011-09-07 | 赛莱克蒂斯公司 | 切割来自谷氨酰胺合成酶基因的dna靶序列的大范围核酸酶变体及其用途 |
| CA2799095A1 (en) * | 2010-05-12 | 2011-11-17 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| EP2714936B1 (en) | 2011-06-01 | 2018-12-12 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| 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 |
| EP4659765A3 (en) * | 2014-03-12 | 2026-02-18 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| TW202503057A (zh) | 2014-10-10 | 2025-01-16 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| KR102787119B1 (ko) * | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| CA3035534A1 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| KR20230010826A (ko) * | 2016-10-14 | 2023-01-19 | 프리시젼 바이오사이언시스 인코포레이티드 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| WO2018154459A1 (en) | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders |
| JP7676111B2 (ja) * | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| IL301059B2 (en) * | 2017-04-21 | 2026-04-01 | Prec Biosciences Inc | Engineered nucleosides specific for recognition sequences in the PCSK9 gene |
| MX2021001070A (es) | 2018-07-31 | 2021-05-27 | Intellia Therapeutics Inc | Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1). |
| WO2020132659A1 (en) * | 2018-12-21 | 2020-06-25 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
-
2021
- 2021-11-12 JP JP2023528230A patent/JP7642809B2/ja active Active
- 2021-11-12 KR KR1020237019155A patent/KR102826438B1/ko active Active
- 2021-11-12 CN CN202510254831.XA patent/CN120098962A/zh active Pending
- 2021-11-12 CA CA3172481A patent/CA3172481A1/en active Pending
- 2021-11-12 KR KR1020257020856A patent/KR20250099412A/ko active Pending
- 2021-11-12 CN CN202180088740.3A patent/CN116783293B/zh active Active
- 2021-11-12 MX MX2023005496A patent/MX2023005496A/es unknown
- 2021-11-12 IL IL317538A patent/IL317538A/en unknown
- 2021-11-12 IL IL302733A patent/IL302733B2/en unknown
- 2021-11-12 CN CN202510254984.4A patent/CN120098963A/zh active Pending
- 2021-11-12 EP EP21824147.9A patent/EP4244342B1/en active Active
- 2021-11-12 WO PCT/US2021/059146 patent/WO2022104062A1/en not_active Ceased
-
2022
- 2022-09-13 US US17/931,896 patent/US11753630B2/en active Active
-
2023
- 2023-07-27 US US18/360,312 patent/US12006522B2/en active Active
- 2023-07-27 US US18/360,517 patent/US11993793B2/en active Active
-
2024
- 2024-04-26 US US18/647,877 patent/US20250034536A1/en active Pending
-
2025
- 2025-02-26 JP JP2025028775A patent/JP2025098002A/ja active Pending
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