Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0XZ4Y
|
|||
| Drug Name |
PR001
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Gaucher disease [ICD-11: 5C56.0Y; ICD-9: 272.7] | Phase 1/2 | [1] | |
| Parkinson disease [ICD-11: 8A00.0; ICD-10: F02.3, G20; ICD-9: 332] | Phase 1/2 | [2] | ||
| Company |
Prevail Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal acid glucosylceramidase (GBA1) | Target Info | . | [3] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04411654) An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease. U.S.National Institutes of Health. | |||
| REF 2 | ClinicalTrials.gov (NCT04127578) A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of LY3884961 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL). U.S.National Institutes of Health. | |||
| REF 3 | Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations. J Parkinsons Dis. 2021;11(s2):S183-S188. | |||
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