JPWO2020142752A5 - - Google Patents
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- Publication number
- JPWO2020142752A5 JPWO2020142752A5 JP2021538993A JP2021538993A JPWO2020142752A5 JP WO2020142752 A5 JPWO2020142752 A5 JP WO2020142752A5 JP 2021538993 A JP2021538993 A JP 2021538993A JP 2021538993 A JP2021538993 A JP 2021538993A JP WO2020142752 A5 JPWO2020142752 A5 JP WO2020142752A5
- Authority
- JP
- Japan
- Prior art keywords
- expression construct
- gal
- cell
- item
- protein
- 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 |
|---|---|---|---|
| JP2025121650A JP2025142225A (ja) | 2019-01-04 | 2025-07-18 | ファブリー病の処置のための方法及び組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962788439P | 2019-01-04 | 2019-01-04 | |
| US62/788,439 | 2019-01-04 | ||
| PCT/US2020/012274 WO2020142752A1 (en) | 2019-01-04 | 2020-01-03 | Methods and compositions for the treatment of fabry disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025121650A Division JP2025142225A (ja) | 2019-01-04 | 2025-07-18 | ファブリー病の処置のための方法及び組成物 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022516317A JP2022516317A (ja) | 2022-02-25 |
| JP2022516317A5 JP2022516317A5 (https=) | 2023-01-19 |
| JPWO2020142752A5 true JPWO2020142752A5 (https=) | 2023-01-19 |
| JP7716338B2 JP7716338B2 (ja) | 2025-07-31 |
Family
ID=71407140
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538993A Active JP7716338B2 (ja) | 2019-01-04 | 2020-01-03 | ファブリー病の処置のための方法及び組成物 |
| JP2025121650A Pending JP2025142225A (ja) | 2019-01-04 | 2025-07-18 | ファブリー病の処置のための方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025121650A Pending JP2025142225A (ja) | 2019-01-04 | 2025-07-18 | ファブリー病の処置のための方法及び組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20200239911A1 (https=) |
| EP (1) | EP3906043A4 (https=) |
| JP (2) | JP7716338B2 (https=) |
| KR (1) | KR20210112337A (https=) |
| CN (1) | CN113423419A (https=) |
| AU (1) | AU2020204718B2 (https=) |
| BR (1) | BR112021012249A2 (https=) |
| CA (1) | CA3124564A1 (https=) |
| IL (1) | IL284414A (https=) |
| MX (1) | MX2021008131A (https=) |
| SG (1) | SG11202107325TA (https=) |
| WO (1) | WO2020142752A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3039673A1 (en) | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| US20220298514A1 (en) * | 2019-09-11 | 2022-09-22 | Aav Gene Therapeutics, Inc. | Aav-zyme and use for infusion replacement therapy |
| US20230355733A1 (en) * | 2020-07-14 | 2023-11-09 | Evaxion Biotech A/S | APC targeting units for immunotherapy |
| AU2021356684A1 (en) * | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| KR20230150836A (ko) * | 2021-02-26 | 2023-10-31 | 다케다 야쿠힌 고교 가부시키가이샤 | 파브리병 치료를 위한 조성물 및 방법 |
| KR20240073025A (ko) * | 2021-08-25 | 2024-05-24 | 캔브리지 파마슈티컬스, 인크. | 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도 |
| EP4426846A4 (en) * | 2021-11-03 | 2025-11-05 | Sangamo Therapeutics Inc | METHODS OF USING VIRAL VECTOR CONSTRUCTIONS FOR THE TREATMENT OF FABRY DISEASE |
| WO2023102517A1 (en) * | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| CN118475373A (zh) * | 2021-12-31 | 2024-08-09 | 九天生物医药(上海)有限公司 | 用于法布雷病的基因治疗的重组aav |
| WO2023164060A2 (en) * | 2022-02-25 | 2023-08-31 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy |
| CN117551636B (zh) * | 2023-04-30 | 2024-11-08 | 四川至善唯新生物科技有限公司 | 工程化α-GAL A肽及其功能变体和治疗法布里病的相关方法 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR100545945B1 (ko) * | 2000-07-03 | 2006-01-25 | 갈라 디자인, 인크. | 발현 벡터 |
| US6852510B2 (en) * | 2000-07-03 | 2005-02-08 | Gala Design Inc | Host cells containing multiple integrating vectors |
| GB0024550D0 (https=) * | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| AU2003221733A1 (en) * | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| EP1995309A1 (en) * | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| EP2708600A4 (en) * | 2011-04-28 | 2015-01-07 | Univ Yamaguchi | REVERSE PRIMER WITH A TERMINATION SEQUENCE FOR OVEREXPRESSION AND LINEAR DNA |
| CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| US10179918B2 (en) * | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| WO2016210170A1 (en) * | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| CN105950664B (zh) * | 2016-05-17 | 2019-03-29 | 上海优卡迪生物医药科技有限公司 | 一种靶向cd123的复制缺陷性重组慢病毒car-t转基因载体及其构建方法和应用 |
| AU2017266932B2 (en) * | 2016-05-18 | 2023-04-20 | Modernatx, Inc. | Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease |
| CA3039673A1 (en) * | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CA3089587A1 (en) * | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
| DE102020111571A1 (de) * | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
-
2020
- 2020-01-03 JP JP2021538993A patent/JP7716338B2/ja active Active
- 2020-01-03 AU AU2020204718A patent/AU2020204718B2/en active Active
- 2020-01-03 CN CN202080010090.6A patent/CN113423419A/zh active Pending
- 2020-01-03 CA CA3124564A patent/CA3124564A1/en active Pending
- 2020-01-03 SG SG11202107325TA patent/SG11202107325TA/en unknown
- 2020-01-03 EP EP20735863.1A patent/EP3906043A4/en active Pending
- 2020-01-03 MX MX2021008131A patent/MX2021008131A/es unknown
- 2020-01-03 US US16/734,271 patent/US20200239911A1/en active Pending
- 2020-01-03 BR BR112021012249-4A patent/BR112021012249A2/pt unknown
- 2020-01-03 KR KR1020217023276A patent/KR20210112337A/ko active Pending
- 2020-01-03 WO PCT/US2020/012274 patent/WO2020142752A1/en not_active Ceased
-
2021
- 2021-06-27 IL IL284414A patent/IL284414A/en unknown
-
2025
- 2025-07-18 JP JP2025121650A patent/JP2025142225A/ja active Pending
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