JPWO2020106916A5 - - Google Patents
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- Publication number
- JPWO2020106916A5 JPWO2020106916A5 JP2021529002A JP2021529002A JPWO2020106916A5 JP WO2020106916 A5 JPWO2020106916 A5 JP WO2020106916A5 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP WO2020106916 A5 JPWO2020106916 A5 JP WO2020106916A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- nucleic acid
- seq
- nos
- aav vector
- 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
- 108020004707 nucleic acids Proteins 0.000 claims 24
- 150000007523 nucleic acids Chemical class 0.000 claims 24
- 102000003869 Frataxin Human genes 0.000 claims 6
- 108090000217 Frataxin Proteins 0.000 claims 6
- 230000001402 polyadenylating Effects 0.000 claims 4
- 210000003169 Central Nervous System Anatomy 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 239000003623 enhancer Substances 0.000 claims 2
- 230000002708 enhancing Effects 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000271566 Aves Species 0.000 claims 1
- 241000283690 Bos taurus Species 0.000 claims 1
- 102000004040 Capsid Proteins Human genes 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 206010017374 Friedreich's ataxia Diseases 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 210000001519 tissues Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862770202P | 2018-11-21 | 2018-11-21 | |
| US62/770,202 | 2018-11-21 | ||
| PCT/US2019/062531 WO2020106916A1 (en) | 2018-11-21 | 2019-11-21 | Recombinant viral vectors and nucleic acids for producing the same |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022508182A JP2022508182A (ja) | 2022-01-19 |
| JPWO2020106916A5 true JPWO2020106916A5 (ru) | 2022-11-30 |
Family
ID=70773595
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021529002A Pending JP2022508182A (ja) | 2018-11-21 | 2019-11-21 | 組換えウイルスベクター及びそれの産生のための核酸 |
Country Status (20)
| Country | Link |
|---|---|
| US (1) | US20210324418A1 (ru) |
| EP (1) | EP3883954A4 (ru) |
| JP (1) | JP2022508182A (ru) |
| KR (1) | KR20210103469A (ru) |
| CN (1) | CN113302201A (ru) |
| AR (1) | AR117145A1 (ru) |
| AU (1) | AU2019385506A1 (ru) |
| BR (1) | BR112021009733A2 (ru) |
| CA (1) | CA3120289A1 (ru) |
| CL (1) | CL2021001327A1 (ru) |
| CO (1) | CO2021008120A2 (ru) |
| EA (1) | EA202191418A1 (ru) |
| EC (1) | ECSP21044840A (ru) |
| IL (1) | IL283344A (ru) |
| MX (1) | MX2021005997A (ru) |
| PE (1) | PE20211419A1 (ru) |
| PH (1) | PH12021551155A1 (ru) |
| SG (1) | SG11202105326WA (ru) |
| TW (1) | TW202039533A (ru) |
| WO (1) | WO2020106916A1 (ru) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN107686842A (zh) * | 2016-08-03 | 2018-02-13 | 南京大学 | 一种靶多核苷酸编辑方法及其应用 |
| SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease |
| US20220010332A1 (en) * | 2020-07-08 | 2022-01-13 | Neuracle Genetics Inc. | Intron fragments |
| US20230285596A1 (en) * | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| US20220098255A1 (en) * | 2020-09-29 | 2022-03-31 | NeuExcell Therapeutics Inc. | Neurod1 combination vector |
| CA3197321A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Neurod1 vector |
| WO2022072309A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Ascl1 vector |
| WO2023221942A1 (en) * | 2022-05-16 | 2023-11-23 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating glutaric aciduria type i |
| WO2023240162A1 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2024074143A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20090221620A1 (en) * | 2008-02-20 | 2009-09-03 | Celera Corporation | Gentic polymorphisms associated with stroke, methods of detection and uses thereof |
| US20160256571A1 (en) * | 2013-09-30 | 2016-09-08 | Sanofi | Invention |
| CA2970730A1 (en) * | 2015-01-16 | 2016-07-21 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| WO2016150964A1 (en) * | 2015-03-23 | 2016-09-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia |
| IL296365A (en) * | 2015-05-16 | 2022-11-01 | Genzyme Corp | Genetic editing of deep intronic mutations |
| KR102200642B1 (ko) * | 2015-11-05 | 2021-01-12 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
| AU2016372035B2 (en) * | 2015-12-15 | 2023-03-30 | Genzyme Corporation | Adeno-associated viral vectors for treating mucolipidosis type ii |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
-
2019
- 2019-11-21 EP EP19887003.2A patent/EP3883954A4/en active Pending
- 2019-11-21 BR BR112021009733A patent/BR112021009733A2/pt unknown
- 2019-11-21 SG SG11202105326WA patent/SG11202105326WA/en unknown
- 2019-11-21 MX MX2021005997A patent/MX2021005997A/es unknown
- 2019-11-21 AU AU2019385506A patent/AU2019385506A1/en active Pending
- 2019-11-21 JP JP2021529002A patent/JP2022508182A/ja active Pending
- 2019-11-21 CN CN201980088932.7A patent/CN113302201A/zh active Pending
- 2019-11-21 AR ARP190103423A patent/AR117145A1/es unknown
- 2019-11-21 PE PE2021000747A patent/PE20211419A1/es unknown
- 2019-11-21 TW TW108142416A patent/TW202039533A/zh unknown
- 2019-11-21 CA CA3120289A patent/CA3120289A1/en active Pending
- 2019-11-21 KR KR1020217017068A patent/KR20210103469A/ko unknown
- 2019-11-21 EA EA202191418A patent/EA202191418A1/ru unknown
- 2019-11-21 WO PCT/US2019/062531 patent/WO2020106916A1/en unknown
-
2021
- 2021-05-20 CL CL2021001327A patent/CL2021001327A1/es unknown
- 2021-05-20 IL IL283344A patent/IL283344A/en unknown
- 2021-05-20 PH PH12021551155A patent/PH12021551155A1/en unknown
- 2021-05-21 US US17/326,884 patent/US20210324418A1/en active Pending
- 2021-06-21 CO CONC2021/0008120A patent/CO2021008120A2/es unknown
- 2021-06-21 EC ECSENADI202144840A patent/ECSP21044840A/es unknown
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