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Phase II Gene Therapy Trial of RP-L102 in Fanconi Anemia Subtype A
2018-002502-31Phase 2Active
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Aug 2018
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of PGK-FANCA-Wpre* transduced CD34+ cells in Fanconi anemia type A (FA-A), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Phenotypic correction of hematopoietic cells. Started Aug 2018; status: active.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia type A (FA-A)
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