JP2021523198A5 - - Google Patents
Info
- Publication number
- JP2021523198A5 JP2021523198A5 JP2020564142A JP2020564142A JP2021523198A5 JP 2021523198 A5 JP2021523198 A5 JP 2021523198A5 JP 2020564142 A JP2020564142 A JP 2020564142A JP 2020564142 A JP2020564142 A JP 2020564142A JP 2021523198 A5 JP2021523198 A5 JP 2021523198A5
- Authority
- JP
- Japan
- Prior art keywords
- composition according
- therapeutic
- aav virus
- young
- concentration
- Prior art date
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023191118A JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671271P | 2018-05-14 | 2018-05-14 | |
| US62/671,271 | 2018-05-14 | ||
| PCT/US2019/032092 WO2019222132A1 (en) | 2018-05-14 | 2019-05-14 | Stable expression of aav vectors in juvenile subjects |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191118A Division JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021523198A JP2021523198A (ja) | 2021-09-02 |
| JPWO2019222132A5 JPWO2019222132A5 (https=) | 2022-05-24 |
| JP2021523198A5 true JP2021523198A5 (https=) | 2022-05-24 |
Family
ID=66770562
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020564142A Pending JP2021523198A (ja) | 2018-05-14 | 2019-05-14 | 幼若対象におけるａａｖベクターの安定的発現 |
| JP2023191118A Pending JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191118A Pending JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20200069819A1 (https=) |
| EP (1) | EP3794112A1 (https=) |
| JP (2) | JP2021523198A (https=) |
| KR (1) | KR20210008491A (https=) |
| CN (1) | CN112424345A (https=) |
| AR (1) | AR117427A1 (https=) |
| AU (1) | AU2019270972A1 (https=) |
| BR (1) | BR112020023159A2 (https=) |
| CA (1) | CA3100000A1 (https=) |
| MX (1) | MX2020012167A (https=) |
| SG (1) | SG11202010832YA (https=) |
| TW (1) | TW202016298A (https=) |
| WO (1) | WO2019222132A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PE20190401A1 (es) | 2016-07-26 | 2019-03-13 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado |
| BR112020022722A8 (pt) | 2018-05-09 | 2022-01-18 | Biomarin Pharm Inc | Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| US20240376496A1 (en) | 2021-09-03 | 2024-11-14 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| US20240384242A1 (en) | 2021-09-03 | 2024-11-21 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4396203A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| KR20250108100A (ko) | 2022-09-22 | 2025-07-15 | 디나코 아게 | Aav 유전자 치료법 벡터를 이용한 심근병증의 치료 |
| UY40442A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav |
| JP2025534900A (ja) * | 2022-10-08 | 2025-10-21 | リンイ バイオテック カンパニー リミテッド | 遺伝子発現を増強するためのコンストラクト |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2025214477A1 (en) | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US298A (en) | 1837-07-29 | Process for purifying salt-water preparatory to manufacturing salt | ||
| US6531A (en) | 1849-06-19 | Beed musical instrument | ||
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| EP4219547A3 (en) | 2008-09-15 | 2023-10-18 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| RS61039B1 (sr) | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| RU2742352C2 (ru) * | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| CA3002524A1 (en) * | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| CN110770346B (zh) * | 2017-03-15 | 2024-01-12 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
-
2019
- 2019-05-14 US US16/411,841 patent/US20200069819A1/en not_active Abandoned
- 2019-05-14 TW TW108116635A patent/TW202016298A/zh unknown
- 2019-05-14 BR BR112020023159-2A patent/BR112020023159A2/pt unknown
- 2019-05-14 CN CN201980047182.9A patent/CN112424345A/zh active Pending
- 2019-05-14 EP EP19728796.4A patent/EP3794112A1/en not_active Withdrawn
- 2019-05-14 KR KR1020207034760A patent/KR20210008491A/ko not_active Withdrawn
- 2019-05-14 AU AU2019270972A patent/AU2019270972A1/en not_active Abandoned
- 2019-05-14 AR ARP190101290A patent/AR117427A1/es not_active Application Discontinuation
- 2019-05-14 WO PCT/US2019/032092 patent/WO2019222132A1/en not_active Ceased
- 2019-05-14 CA CA3100000A patent/CA3100000A1/en active Pending
- 2019-05-14 MX MX2020012167A patent/MX2020012167A/es unknown
- 2019-05-14 JP JP2020564142A patent/JP2021523198A/ja active Pending
- 2019-05-14 SG SG11202010832YA patent/SG11202010832YA/en unknown
-
2023
- 2023-11-08 JP JP2023191118A patent/JP2024028696A/ja active Pending
-
2024
- 2024-05-13 US US18/662,491 patent/US20240285806A1/en not_active Abandoned
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