CA3134841A1 - Therapies geniques pour troubles lysosomaux - Google Patents
Therapies geniques pour troubles lysosomaux Download PDFInfo
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- CA3134841A1 CA3134841A1 CA3134841A CA3134841A CA3134841A1 CA 3134841 A1 CA3134841 A1 CA 3134841A1 CA 3134841 A CA3134841 A CA 3134841A CA 3134841 A CA3134841 A CA 3134841A CA 3134841 A1 CA3134841 A1 CA 3134841A1
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- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
L'invention concerne, selon certains aspects, des compositions et des méthodes pour le traitement de maladies du système nerveux central (SNC) par exemple la maladie de Parkinson (MP) et la maladie de Gaucher. Dans certains modes de réalisation, l'invention concerne des constructions d'expression comprenant un transgène codant pour un ou plusieurs produits géniques associés à une maladie du SNC et/ou un ou plusieurs acides nucléiques inhibiteurs ciblant un gène ou un produit génique associé à une maladie du SNC. Dans certains modes de réalisation, l'invention concerne des méthodes de traitement de maladies du SNC par administration de telles constructions d'expression à un sujet en ayant besoin.
Applications Claiming Priority (19)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831840P | 2019-04-10 | 2019-04-10 | |
| US201962831856P | 2019-04-10 | 2019-04-10 | |
| US201962831846P | 2019-04-10 | 2019-04-10 | |
| US201962832223P | 2019-04-10 | 2019-04-10 | |
| US62/831,856 | 2019-04-10 | ||
| US62/831,846 | 2019-04-10 | ||
| US62/831,840 | 2019-04-10 | ||
| US62/832,223 | 2019-04-10 | ||
| US201962934450P | 2019-11-12 | 2019-11-12 | |
| US62/934,450 | 2019-11-12 | ||
| US201962954089P | 2019-12-27 | 2019-12-27 | |
| US62/954,089 | 2019-12-27 | ||
| US202062960471P | 2020-01-13 | 2020-01-13 | |
| US62/960,471 | 2020-01-13 | ||
| US202062998665P | 2020-03-12 | 2020-03-12 | |
| US62/988,665 | 2020-03-12 | ||
| US202062990246P | 2020-03-16 | 2020-03-16 | |
| US62/990,246 | 2020-03-16 | ||
| PCT/US2020/027658 WO2020210615A1 (fr) | 2019-04-10 | 2020-04-10 | Thérapies géniques pour troubles lysosomaux |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3134841A1 true CA3134841A1 (fr) | 2020-10-15 |
Family
ID=72750865
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3134841A Pending CA3134841A1 (fr) | 2019-04-10 | 2020-04-10 | Therapies geniques pour troubles lysosomaux |
Country Status (4)
| Country | Link |
|---|---|
| AU (1) | AU2020272974A1 (fr) |
| BR (1) | BR112021020157A2 (fr) |
| CA (1) | CA3134841A1 (fr) |
| WO (1) | WO2020210615A1 (fr) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11993790B2 (en) | 2017-10-03 | 2024-05-28 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US11802294B2 (en) | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN111465691A (zh) | 2017-10-03 | 2020-07-28 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CA3136117A1 (fr) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Therapies geniques pour troubles lysosomaux |
| WO2023278305A1 (fr) * | 2021-06-28 | 2023-01-05 | The Board Of Regents Of The University Of Texas System | Méthodes et compositions pour une thérapie génique de réduction de la protéine tau |
| WO2023114816A1 (fr) * | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Constructions du galc optimisées recombinantes et méthodes de traitement de troubles associés au galc |
| WO2023193004A2 (fr) * | 2022-04-01 | 2023-10-05 | Triplet Therapeutics, Inc. | Procédés de traitement de troubles neurodégénératifs |
| WO2023215947A1 (fr) * | 2022-05-13 | 2023-11-16 | Children's Medical Research Institute | Capsides de virus adéno-associés |
| WO2024076940A1 (fr) * | 2022-10-04 | 2024-04-11 | Eli Lilly And Company | Thérapie génique pour des maladies et des troubles associés à trem2 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2006254796B2 (en) * | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| US10648001B2 (en) * | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| SG11201509419QA (en) * | 2013-05-15 | 2015-12-30 | Univ Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| CA2985235A1 (fr) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Therapie genique par glucocerebrosidase pour la maladie de parkinson |
-
2020
- 2020-04-10 AU AU2020272974A patent/AU2020272974A1/en not_active Abandoned
- 2020-04-10 CA CA3134841A patent/CA3134841A1/fr active Pending
- 2020-04-10 WO PCT/US2020/027658 patent/WO2020210615A1/fr unknown
- 2020-04-10 BR BR112021020157A patent/BR112021020157A2/pt not_active Application Discontinuation
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020210615A1 (fr) | 2020-10-15 |
| AU2020272974A1 (en) | 2021-11-11 |
| BR112021020157A2 (pt) | 2021-12-14 |
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