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Trial intelligence / Profile preview
FANCOLEN-I Long-Term Follow-up
2024-511523-33-00Phase 2Authorised
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Aug 2024
- 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: Long-term safety of Fancalen. Planned start Aug 2024; status: authorised.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia type A (FA-A)
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