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Long-Term Follow-up of RP-L102 Gene Therapy in Fanconi Anemia Subtype A
2022-501082-52-00Phase 2Authorised
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Oct 2022
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of PGK-FANCA-Wpre* transduced CD34+ cells in Fanconi anemia (FA), sponsored by Rocket Pharmaceuticals, Inc. Planned start Oct 2022; status: authorised.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia (FA)
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