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CLINICAL_TRIALS research · 2025-12-22

Clinical Trial Update: Gene Therapy for Fanconi Anemia, Complementation Group A [Phase 2]

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Summary

This entry reports a Phase 2 gene therapy trial for Fanconi anemia, complementation group A, which is currently active but not recruiting.

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Diseases / indications

Drugs mentioned

  • Rocket Pharma FANCA gene therapy (investigational) (FANCA gene therapy)

Snippet

Sponsor: Rocket Pharmaceuticals Inc. | Conditions: Fanconi Anemia Complementation Group A | Phase 2 | Status: ACTIVE_NOT_RECRUITING