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RP-L301 Gene Therapy for Pyruvate Kinase Deficiency
NCT04105166Phase 1Completed
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Jul 2020
- Primary completion
- Jun 2025
- Modality
- Gene Editing, Gene Silencing, Cell Therapies
- Countries
- 2
01
Overview
Phase 1, open-label, single-arm trial of RP-L301 in pyruvate kinase deficiency (PKD), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Number of participants with treatment-related adverse events at 2 years. Started Jul 2020; status: completed.
02
Drugs
RP-L301
03
Conditions
pyruvate kinase deficiency (PKD)
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