JP2024506860A5 - - Google Patents
Info
- Publication number
- JP2024506860A5 JP2024506860A5 JP2023546548A JP2023546548A JP2024506860A5 JP 2024506860 A5 JP2024506860 A5 JP 2024506860A5 JP 2023546548 A JP2023546548 A JP 2023546548A JP 2023546548 A JP2023546548 A JP 2023546548A JP 2024506860 A5 JP2024506860 A5 JP 2024506860A5
- Authority
- JP
- Japan
- Prior art keywords
- hsmpd1
- seq
- sequence
- coding sequence
- raav
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163144103P | 2021-02-01 | 2021-02-01 | |
| US63/144,103 | 2021-02-01 | ||
| US202163286939P | 2021-12-07 | 2021-12-07 | |
| US63/286,939 | 2021-12-07 | ||
| PCT/US2022/014742 WO2022165421A1 (en) | 2021-02-01 | 2022-02-01 | Compositions and methods for treatment of niemann pick type a disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024506860A JP2024506860A (ja) | 2024-02-15 |
| JP2024506860A5 true JP2024506860A5 (https=) | 2025-02-07 |
Family
ID=82654923
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023546548A Pending JP2024506860A (ja) | 2021-02-01 | 2022-02-01 | ニーマンピック病ａ型を治療するための組成物及び方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240115733A1 (https=) |
| EP (1) | EP4284442A4 (https=) |
| JP (1) | JP2024506860A (https=) |
| AU (1) | AU2022214529A1 (https=) |
| CA (1) | CA3205351A1 (https=) |
| MX (1) | MX2023009030A (https=) |
| WO (1) | WO2022165421A1 (https=) |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) * | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| US7527956B2 (en) * | 2003-06-12 | 2009-05-05 | Genzyme Corporation | Modified human acid sphingomyelinase having increased activity, and methods for making the same |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| AR059371A1 (es) * | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| MX2018012537A (es) * | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| PY1939780A (es) * | 2018-05-25 | 2019-12-04 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
-
2022
- 2022-02-01 EP EP22746872.5A patent/EP4284442A4/en active Pending
- 2022-02-01 MX MX2023009030A patent/MX2023009030A/es unknown
- 2022-02-01 WO PCT/US2022/014742 patent/WO2022165421A1/en not_active Ceased
- 2022-02-01 AU AU2022214529A patent/AU2022214529A1/en active Pending
- 2022-02-01 JP JP2023546548A patent/JP2024506860A/ja active Pending
- 2022-02-01 US US18/263,085 patent/US20240115733A1/en active Pending
- 2022-02-01 CA CA3205351A patent/CA3205351A1/en active Pending
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