JP2022508182A5 - - Google Patents
Info
- Publication number
- JP2022508182A5 JP2022508182A5 JP2021529002A JP2021529002A JP2022508182A5 JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- nucleic acid
- aav vector
- acid according
- recombinant 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025034621A JP2025102783A (ja) | 2018-11-21 | 2025-03-05 | 組換えウイルスベクター及びそれの産生のための核酸 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025034621A Division JP2025102783A (ja) | 2018-11-21 | 2025-03-05 | 組換えウイルスベクター及びそれの産生のための核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022508182A JP2022508182A (ja) | 2022-01-19 |
| JP2022508182A5 true JP2022508182A5 (https=) | 2022-11-30 |
| JP7704998B2 JP7704998B2 (ja) | 2025-07-09 |
Family
ID=70773595
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021529002A Active JP7704998B2 (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 |
| JP2025034621A Pending JP2025102783A (ja) | 2018-11-21 | 2025-03-05 | 組換えウイルスベクター及びそれの産生のための核酸 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025034621A Pending JP2025102783A (ja) | 2018-11-21 | 2025-03-05 | 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 (https=) |
| EP (1) | EP3883954A4 (https=) |
| JP (2) | JP7704998B2 (https=) |
| KR (1) | KR20210103469A (https=) |
| CN (1) | CN113302201A (https=) |
| AR (1) | AR117145A1 (https=) |
| AU (1) | AU2019385506A1 (https=) |
| BR (1) | BR112021009733A2 (https=) |
| CA (1) | CA3120289A1 (https=) |
| CL (1) | CL2021001327A1 (https=) |
| CO (1) | CO2021008120A2 (https=) |
| EA (1) | EA202191418A1 (https=) |
| EC (1) | ECSP21044840A (https=) |
| IL (1) | IL283344A (https=) |
| MX (1) | MX2021005997A (https=) |
| PE (1) | PE20211419A1 (https=) |
| PH (1) | PH12021551155A1 (https=) |
| SG (1) | SG11202105326WA (https=) |
| TW (1) | TW202039533A (https=) |
| WO (1) | WO2020106916A1 (https=) |
Families Citing this family (28)
| 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 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| US12173304B2 (en) * | 2020-07-08 | 2024-12-24 | Neuracle Genetics Inc. | Intron fragments |
| WO2022026410A2 (en) * | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| EP4222270A4 (en) * | 2020-09-29 | 2025-02-26 | Neuexcell Therapeutics Inc. | NEUROD1-VECTOR |
| JP2023543360A (ja) * | 2020-09-29 | 2023-10-13 | ニューエクセル・セラピューティクス・インコーポレイテッド | Ｄｌｘ２ベクター |
| WO2022072309A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Ascl1 vector |
| WO2022072325A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Neurod1 combination vector |
| WO2022087321A1 (en) * | 2020-10-21 | 2022-04-28 | Duke University | Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| CA3203975A1 (en) | 2020-12-03 | 2022-06-09 | Battelle Memorial Institute | Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery |
| WO2022147575A1 (en) * | 2021-01-04 | 2022-07-07 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| CA3216359A1 (en) | 2021-04-07 | 2022-10-13 | Battelle Memorial Institute | Rapid design, build, test, and learn technologies for identifying and using non-viral carriers |
| IL311499A (en) * | 2021-09-17 | 2024-05-01 | Astellas Gene Therapies Inc | Frataxin gene therapy |
| CN119213133B (zh) | 2022-05-16 | 2026-01-06 | 上海天泽云泰生物医药有限公司 | 用于治疗i型戊二酸尿症的重组aav载体 |
| US20250361525A1 (en) * | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| EP4598590A1 (en) * | 2022-10-08 | 2025-08-13 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| WO2024163678A2 (en) * | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| AU2024353375A1 (en) | 2023-09-29 | 2026-04-09 | Battelle Memorial Institute | Polymer nanoparticle compositions for in vivo expression of polypeptides |
| WO2025122954A1 (en) | 2023-12-08 | 2025-06-12 | Battelle Memorial Institute | Use of dna origami nanostructures for molecular information based data storage systems |
| CN117701638B (zh) * | 2023-12-18 | 2024-10-18 | 苏州新芽基因生物技术有限公司 | 一种抑制残留aav载体质粒和质粒骨架转录活性的方法 |
| WO2026019230A1 (ko) * | 2024-07-15 | 2026-01-22 | 삼성바이오에피스 주식회사 | 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002081632A2 (en) * | 2001-04-04 | 2002-10-17 | The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | Nucleic acids for transgene expression |
| US20090221620A1 (en) * | 2008-02-20 | 2009-09-03 | Celera Corporation | Gentic polymorphisms associated with stroke, methods of detection and uses thereof |
| AU2014255665B2 (en) * | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| US20160256571A1 (en) * | 2013-09-30 | 2016-09-08 | Sanofi | Invention |
| US10947596B2 (en) | 2014-05-06 | 2021-03-16 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators |
| US11697825B2 (en) * | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| MX2017009336A (es) * | 2015-01-16 | 2017-11-15 | Voyager Therapeutics Inc | Polinucleótidos dirigidos al sistema nervioso central. |
| CR20170406A (es) * | 2015-02-10 | 2017-11-14 | Genzyme Corp | Arni variante |
| US11040113B2 (en) * | 2015-03-23 | 2021-06-22 | 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 |
| WO2016164642A1 (en) * | 2015-04-08 | 2016-10-13 | The United States Of America, As Represented By The Secretary Of Health And Human Services | Viral gene therapy as treatment for cholesterol storage disease or disorder |
| BR112017024514A2 (pt) * | 2015-05-16 | 2018-07-24 | Genzyme Corporation | edição genética de mutações intrônicas profundas |
| MX390848B (es) * | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| WO2017023708A1 (en) * | 2015-07-31 | 2017-02-09 | California Institute Of Technology | Activity-dependent expression of nucleic acids |
| SG11201802408RA (en) * | 2015-11-05 | 2018-05-30 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| MX2018007330A (es) * | 2015-12-15 | 2019-03-14 | Genzyme Corp | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. |
| EP4484443A3 (en) * | 2016-06-29 | 2025-03-26 | CRISPR Therapeutics AG | Materials and methods for treatment of friedreich ataxia and other related disorders |
| CA3045335A1 (en) * | 2016-12-01 | 2018-06-07 | Universite Laval | Crispr-based treatment of friedreich ataxia |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| AU2018338728B2 (en) * | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
-
2019
- 2019-11-21 WO PCT/US2019/062531 patent/WO2020106916A1/en not_active Ceased
- 2019-11-21 EP EP19887003.2A patent/EP3883954A4/en active Pending
- 2019-11-21 PE PE2021000747A patent/PE20211419A1/es unknown
- 2019-11-21 MX MX2021005997A patent/MX2021005997A/es unknown
- 2019-11-21 KR KR1020217017068A patent/KR20210103469A/ko not_active Ceased
- 2019-11-21 CA CA3120289A patent/CA3120289A1/en active Pending
- 2019-11-21 JP JP2021529002A patent/JP7704998B2/ja active Active
- 2019-11-21 AR ARP190103423A patent/AR117145A1/es unknown
- 2019-11-21 AU AU2019385506A patent/AU2019385506A1/en active Pending
- 2019-11-21 BR BR112021009733A patent/BR112021009733A2/pt unknown
- 2019-11-21 CN CN201980088932.7A patent/CN113302201A/zh active Pending
- 2019-11-21 SG SG11202105326WA patent/SG11202105326WA/en unknown
- 2019-11-21 EA EA202191418A patent/EA202191418A1/ru unknown
- 2019-11-21 TW TW108142416A patent/TW202039533A/zh unknown
-
2021
- 2021-05-20 PH PH12021551155A patent/PH12021551155A1/en unknown
- 2021-05-20 CL CL2021001327A patent/CL2021001327A1/es unknown
- 2021-05-20 IL IL283344A patent/IL283344A/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
-
2025
- 2025-03-05 JP JP2025034621A patent/JP2025102783A/ja active Pending
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