JP2020519629A5 - - Google Patents
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- Publication number
- JP2020519629A5 JP2020519629A5 JP2019562293A JP2019562293A JP2020519629A5 JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5 JP 2019562293 A JP2019562293 A JP 2019562293A JP 2019562293 A JP2019562293 A JP 2019562293A JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- sgsh
- particle according
- variant
- raav particle
- 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
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505423P | 2017-05-12 | 2017-05-12 | |
| US62/505,423 | 2017-05-12 | ||
| PCT/US2018/032454 WO2018209317A1 (en) | 2017-05-12 | 2018-05-11 | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519629A JP2020519629A (ja) | 2020-07-02 |
| JP2020519629A5 true JP2020519629A5 (enExample) | 2021-07-26 |
| JP7196099B2 JP7196099B2 (ja) | 2022-12-26 |
Family
ID=64104969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562293A Active JP7196099B2 (ja) | 2017-05-12 | 2018-05-11 | ムコ多糖症ｉｉｉａ型（ｍｐｓ ｉｉｉａ）を処置するための、スルファミダーゼ（ｓｇｓｈ）バリアント、ベクター、組成物並びに方法及び使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12221635B2 (enExample) |
| EP (1) | EP3622060A4 (enExample) |
| JP (1) | JP7196099B2 (enExample) |
| CN (1) | CN110809626A (enExample) |
| AU (1) | AU2018265869C1 (enExample) |
| BR (1) | BR112019023869A2 (enExample) |
| CA (1) | CA3061925A1 (enExample) |
| MX (1) | MX2019013528A (enExample) |
| RU (1) | RU2019140862A (enExample) |
| WO (1) | WO2018209317A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
| ES3035673T3 (en) * | 2018-12-20 | 2025-09-08 | Esteve Pharmaceuticals Sa | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| WO2021011841A1 (en) * | 2019-07-18 | 2021-01-21 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| CN115023242B (zh) | 2019-11-22 | 2025-03-14 | 费城儿童医院 | 腺相关病毒载体变体 |
| KR20220153021A (ko) * | 2020-03-11 | 2022-11-17 | 리모터 테라퓨틱스, 인코포레이티드 | 중추신경계를 통한 단백질을 확산시키기 위한 방법 및 재료 |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| AU2022388800A1 (en) * | 2021-11-12 | 2024-06-27 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of mucopolysaccharidosis iiia |
| US20250034533A1 (en) * | 2021-11-12 | 2025-01-30 | Amicus Therapeutics, Inc. | Compositions And Methods For Treating Mucopolysaccharidosis IIIA |
| WO2023204202A1 (ja) * | 2022-04-19 | 2023-10-26 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
| CN120379648A (zh) * | 2022-12-23 | 2025-07-25 | 株式会社绿十字 | 具有改善的稳定性的包含乙酰肝素n-硫酸酯酶的药物组合物 |
| WO2026162810A1 (en) * | 2025-02-03 | 2026-08-06 | Institut National de la Santé et de la Recherche Médicale | Methods for the intravenous treatment of sanfilippo syndrome type iiia |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5863782A (en) * | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| EP2550359B1 (en) * | 2010-03-23 | 2017-08-09 | Intrexon Corporation | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of 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 |
| EP3166626B1 (en) * | 2014-07-11 | 2021-04-28 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 US US16/611,458 patent/US12221635B2/en active Active
- 2018-05-11 AU AU2018265869A patent/AU2018265869C1/en active Active
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en not_active Ceased
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 BR BR112019023869-7A patent/BR112019023869A2/pt active Search and Examination
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
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