First systemic AAV gene therapy for a PD subtype; targets the most common genetic risk factor (GBA1 LoF).
Details
- nct_id
- NCT04127578
- phase
- phase_2
- status
- active
- domain
- parkinsons
- indication
- GBA1-mutant Parkinson's Disease
- intervention
- PR001 (AAV9-GBA1 intracisternal)
- sponsor
- Prevail Therapeutics / Lilly
- n_enrolled
- 78
- primary_endpoint
- Safety + GCase enzyme activity in CSF at 52 weeks
Raw fields (1)
- metadata
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