JP2022518931A5 - - Google Patents
Info
- Publication number
- JP2022518931A5 JP2022518931A5 JP2021544194A JP2021544194A JP2022518931A5 JP 2022518931 A5 JP2022518931 A5 JP 2022518931A5 JP 2021544194 A JP2021544194 A JP 2021544194A JP 2021544194 A JP2021544194 A JP 2021544194A JP 2022518931 A5 JP2022518931 A5 JP 2022518931A5
- Authority
- JP
- Japan
- Prior art keywords
- composition according
- aav
- vector
- approximately
- sequence
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025030051A JP2025098018A (ja) | 2019-02-01 | 2025-02-27 | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962800131P | 2019-02-01 | 2019-02-01 | |
| US62/800,131 | 2019-02-01 | ||
| PCT/US2020/016235 WO2020160486A1 (en) | 2019-02-01 | 2020-01-31 | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025030051A Division JP2025098018A (ja) | 2019-02-01 | 2025-02-27 | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022518931A JP2022518931A (ja) | 2022-03-17 |
| JP2022518931A5 true JP2022518931A5 (enExample) | 2023-05-23 |
Family
ID=71841678
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021544194A Pending JP2022518931A (ja) | 2019-02-01 | 2020-01-31 | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
| JP2025030051A Withdrawn JP2025098018A (ja) | 2019-02-01 | 2025-02-27 | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025030051A Withdrawn JP2025098018A (ja) | 2019-02-01 | 2025-02-27 | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220184188A1 (enExample) |
| EP (1) | EP3917522A4 (enExample) |
| JP (2) | JP2022518931A (enExample) |
| CN (1) | CN113891712A (enExample) |
| AU (1) | AU2020215592A1 (enExample) |
| BR (1) | BR112021015050A2 (enExample) |
| CA (1) | CA3127889A1 (enExample) |
| MX (1) | MX2021009250A (enExample) |
| WO (1) | WO2020160486A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA202192328A1 (ru) * | 2019-03-22 | 2022-02-21 | Раш Юниверсити Медикал Сентер | Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления |
| JP2024533163A (ja) | 2021-09-03 | 2024-09-12 | タシト・セラピューティクス・インコーポレーテッド | スプライソソーム構成要素のリクルートを介したｒｎａ編集 |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008103993A2 (en) * | 2007-02-23 | 2008-08-28 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating glycogen storage diseases |
| ES2745470T3 (es) * | 2011-03-31 | 2020-03-02 | Univ Iowa Res Found | Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| EP3221456B1 (en) * | 2014-11-21 | 2021-09-22 | University of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| AU2016341428B2 (en) * | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| US20190269797A1 (en) * | 2016-11-04 | 2019-09-05 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
-
2020
- 2020-01-31 MX MX2021009250A patent/MX2021009250A/es unknown
- 2020-01-31 JP JP2021544194A patent/JP2022518931A/ja active Pending
- 2020-01-31 CA CA3127889A patent/CA3127889A1/en active Pending
- 2020-01-31 BR BR112021015050-1A patent/BR112021015050A2/pt not_active Application Discontinuation
- 2020-01-31 EP EP20749051.7A patent/EP3917522A4/en not_active Withdrawn
- 2020-01-31 CN CN202080024872.5A patent/CN113891712A/zh active Pending
- 2020-01-31 AU AU2020215592A patent/AU2020215592A1/en not_active Abandoned
- 2020-01-31 WO PCT/US2020/016235 patent/WO2020160486A1/en not_active Ceased
- 2020-01-31 US US17/310,378 patent/US20220184188A1/en not_active Abandoned
-
2025
- 2025-02-27 JP JP2025030051A patent/JP2025098018A/ja not_active Withdrawn
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