JP2021500070A5 - - Google Patents
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- Publication number
- JP2021500070A5 JP2021500070A5 JP2020542536A JP2020542536A JP2021500070A5 JP 2021500070 A5 JP2021500070 A5 JP 2021500070A5 JP 2020542536 A JP2020542536 A JP 2020542536A JP 2020542536 A JP2020542536 A JP 2020542536A JP 2021500070 A5 JP2021500070 A5 JP 2021500070A5
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- seq
- capsid protein
- protein corresponding
- aav
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574163P | 2017-10-18 | 2017-10-18 | |
| US62/574,163 | 2017-10-18 | ||
| US201862621102P | 2018-01-24 | 2018-01-24 | |
| US62/621,102 | 2018-01-24 | ||
| PCT/US2018/056271 WO2019079437A1 (en) | 2017-10-18 | 2018-10-17 | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING THE FUNCTION OF THE HBB GENE AND METHODS OF USE |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021500070A JP2021500070A (ja) | 2021-01-07 |
| JP2021500070A5 true JP2021500070A5 (https=) | 2021-12-02 |
Family
ID=64332161
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020542536A Pending JP2021500070A (ja) | 2017-10-18 | 2018-10-17 | Ｈｂｂ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物およびその使用方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12201657B2 (https=) |
| EP (1) | EP3697450A1 (https=) |
| JP (1) | JP2021500070A (https=) |
| KR (1) | KR20200095462A (https=) |
| CN (1) | CN111447954A (https=) |
| AU (1) | AU2018352855B2 (https=) |
| BR (1) | BR112020007765A2 (https=) |
| CA (1) | CA3079437A1 (https=) |
| IL (1) | IL273877A (https=) |
| MX (1) | MX2020004092A (https=) |
| SG (1) | SG11202002737PA (https=) |
| WO (1) | WO2019079437A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109536494A (zh) * | 2017-10-31 | 2019-03-29 | 广东赤萌医疗科技有限公司 | 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒 |
| EP4085143B1 (en) * | 2019-12-31 | 2025-08-13 | Swanbio Therapeutics Limited | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
| CN111363756B (zh) * | 2020-04-02 | 2023-01-17 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 一种珠蛋白基因双表达慢病毒载体及其应用 |
| WO2021207077A1 (en) * | 2020-04-06 | 2021-10-14 | Homology Medicines, Inc. | Adeno-associated virus compositions for ids gene transfer and methods of use thereof |
| WO2022221319A2 (en) * | 2021-04-12 | 2022-10-20 | Graphite Bio, Inc. | Methods and compositions for production of genetically modified primary cells |
| US20250127812A1 (en) * | 2021-08-10 | 2025-04-24 | GentiBio. Inc. | Compositions and methods for engineering stable tregs |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5449759A (en) * | 1987-05-16 | 1995-09-12 | Somatogen, Inc. | Hemoglobins with intersubunit desulfide bonds |
| US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| DE69727492T2 (de) * | 1996-12-05 | 2004-12-16 | Crucell Holland B.V. | Genetische modifikation der hämatopoietischen repopulation von stammzellen in primaten |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| CN101348786B (zh) * | 2007-07-20 | 2011-05-11 | 谭孟群 | 一种人β珠蛋白基因及其重组腺相关病毒载体 |
| WO2009013559A1 (en) | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| US8628966B2 (en) * | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| CA2960209C (en) | 2014-09-04 | 2023-08-29 | Memorial Sloan-Kettering Cancer Center | Globin gene therapy for treating hemoglobinopathies |
| BR112017005892A2 (pt) * | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| US20180135074A1 (en) * | 2015-02-19 | 2018-05-17 | University Of Florida Research Foundation, Incorporated | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
| US11136597B2 (en) * | 2016-02-16 | 2021-10-05 | Yale University | Compositions for enhancing targeted gene editing and methods of use thereof |
-
2018
- 2018-10-17 MX MX2020004092A patent/MX2020004092A/es unknown
- 2018-10-17 CN CN201880079840.8A patent/CN111447954A/zh active Pending
- 2018-10-17 AU AU2018352855A patent/AU2018352855B2/en active Active
- 2018-10-17 BR BR112020007765-8A patent/BR112020007765A2/pt not_active Application Discontinuation
- 2018-10-17 EP EP18804159.4A patent/EP3697450A1/en not_active Withdrawn
- 2018-10-17 CA CA3079437A patent/CA3079437A1/en active Pending
- 2018-10-17 KR KR1020207012240A patent/KR20200095462A/ko not_active Ceased
- 2018-10-17 WO PCT/US2018/056271 patent/WO2019079437A1/en not_active Ceased
- 2018-10-17 US US16/163,061 patent/US12201657B2/en active Active
- 2018-10-17 JP JP2020542536A patent/JP2021500070A/ja active Pending
- 2018-10-17 SG SG11202002737PA patent/SG11202002737PA/en unknown
-
2020
- 2020-04-07 IL IL273877A patent/IL273877A/en unknown
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