JP2021512601A5 - - Google Patents
Info
- Publication number
- JP2021512601A5 JP2021512601A5 JP2020541862A JP2020541862A JP2021512601A5 JP 2021512601 A5 JP2021512601 A5 JP 2021512601A5 JP 2020541862 A JP2020541862 A JP 2020541862A JP 2020541862 A JP2020541862 A JP 2020541862A JP 2021512601 A5 JP2021512601 A5 JP 2021512601A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- seq
- sequence
- capsid protein
- pharmaceutical composition
- 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 |
|---|---|---|---|
| JP2022073374A JP7433360B2 (ja) | 2018-02-01 | 2022-04-27 | Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862625150P | 2018-02-01 | 2018-02-01 | |
| US62/625,150 | 2018-02-01 | ||
| PCT/US2019/016351 WO2019152841A1 (en) | 2018-02-01 | 2019-02-01 | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022073374A Division JP7433360B2 (ja) | 2018-02-01 | 2022-04-27 | Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021512601A JP2021512601A (ja) | 2021-05-20 |
| JP2021512601A5 true JP2021512601A5 (enExample) | 2022-02-09 |
| JP7066861B2 JP7066861B2 (ja) | 2022-05-13 |
Family
ID=67478560
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020541862A Expired - Fee Related JP7066861B2 (ja) | 2018-02-01 | 2019-02-01 | Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
| JP2022073374A Active JP7433360B2 (ja) | 2018-02-01 | 2022-04-27 | Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022073374A Active JP7433360B2 (ja) | 2018-02-01 | 2022-04-27 | Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
Country Status (14)
| Country | Link |
|---|---|
| EP (1) | EP3746113A4 (enExample) |
| JP (2) | JP7066861B2 (enExample) |
| KR (2) | KR20230065367A (enExample) |
| CN (1) | CN111886021A (enExample) |
| AU (1) | AU2019215150A1 (enExample) |
| BR (1) | BR112020015511A2 (enExample) |
| CA (1) | CA3089824A1 (enExample) |
| CL (1) | CL2020001992A1 (enExample) |
| CO (1) | CO2020010783A2 (enExample) |
| EA (1) | EA202091844A1 (enExample) |
| IL (2) | IL295830A (enExample) |
| MX (1) | MX2020008085A (enExample) |
| PE (1) | PE20210341A1 (enExample) |
| WO (1) | WO2019152841A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| 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 |
| WO2020251954A1 (en) * | 2019-06-10 | 2020-12-17 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
| TW202140791A (zh) * | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| JP2023510392A (ja) * | 2020-01-16 | 2023-03-13 | 武田薬品工業株式会社 | フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療 |
| 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 |
| CN116656740A (zh) * | 2023-05-30 | 2023-08-29 | 浙江大学 | 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用 |
| CN119876192B (zh) * | 2024-07-02 | 2025-11-07 | 浙江大学滨江研究院 | 用于治疗苯丙酮尿症的多核苷酸 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE69918090T2 (de) * | 1998-04-24 | 2005-06-16 | University Of Florida, Gainesville | Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie |
| US20030198620A1 (en) | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
| US20040142416A1 (en) * | 2002-04-30 | 2004-07-22 | Laipis Philip J. | Treatment for phenylketonuria |
| WO2010124180A1 (en) * | 2009-04-23 | 2010-10-28 | Phasebio Pharmaceuticals, Inc. | Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria |
| US9839696B2 (en) * | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| TR201908020T4 (tr) * | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| JP6506749B2 (ja) | 2013-10-22 | 2019-04-24 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | フェニルケトン尿症のためのｍＲＮＡ療法 |
| AU2015320694B2 (en) * | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
-
2019
- 2019-02-01 KR KR1020237014637A patent/KR20230065367A/ko not_active Ceased
- 2019-02-01 KR KR1020207025137A patent/KR102528641B1/ko active Active
- 2019-02-01 IL IL295830A patent/IL295830A/en unknown
- 2019-02-01 JP JP2020541862A patent/JP7066861B2/ja not_active Expired - Fee Related
- 2019-02-01 EP EP19748290.4A patent/EP3746113A4/en active Pending
- 2019-02-01 PE PE2020001141A patent/PE20210341A1/es unknown
- 2019-02-01 CN CN201980019659.2A patent/CN111886021A/zh active Pending
- 2019-02-01 WO PCT/US2019/016351 patent/WO2019152841A1/en not_active Ceased
- 2019-02-01 MX MX2020008085A patent/MX2020008085A/es unknown
- 2019-02-01 BR BR112020015511-0A patent/BR112020015511A2/pt not_active IP Right Cessation
- 2019-02-01 EA EA202091844A patent/EA202091844A1/ru unknown
- 2019-02-01 AU AU2019215150A patent/AU2019215150A1/en not_active Abandoned
- 2019-02-01 CA CA3089824A patent/CA3089824A1/en active Pending
-
2020
- 2020-07-22 IL IL276215A patent/IL276215B2/en unknown
- 2020-07-30 CL CL2020001992A patent/CL2020001992A1/es unknown
- 2020-08-28 CO CONC2020/0010783A patent/CO2020010783A2/es unknown
-
2022
- 2022-04-27 JP JP2022073374A patent/JP7433360B2/ja active Active
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