AAV-GBA1 Gene Therapy in Parkinson's (PR001)

Phase 2 Active

GBA1-mutant Parkinson's Disease

NCT ID NCT04127578 ↗

Phase Phase 2

Status Active

Sponsor Prevail Therapeutics / Lilly

Enrolment n = 78

Last updated 2026-04-30

Registered 2026-04-30

Target intervention

PR001 (AAV9-GBA1 intracisternal)

Primary endpoint

Safety + GCase enzyme activity in CSF at 52 weeks

Summary

First systemic AAV gene therapy for a PD subtype; targets the most common genetic risk factor (GBA1 LoF).

Protocol timeline

Phase I

Phase II

Phase III

Phase IV

Current status Active

Enrolled n = 78

View on ClinicalTrials.gov ↗

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