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RP-L102 Gene Therapy for Pediatric Fanconi Anemia Subtype A
NCT04069533Phase 2Active, not recruiting
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Nov 2019
- Primary completion
- Feb 2025
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 2
01
Overview
A Phase 2 trial of PGK-FANCA-Wpre* transduced CD34+ cells in Fanconi anemia (FA), sponsored by Rocket Pharmaceuticals, Inc.. Started Nov 2019; status: active, not recruiting.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia (FA)
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