First patients receive One-Time gene therapy for rare blood disorder
Disease control
Completed
This early-stage study tested the safety of a one-time gene therapy for adults and children with pyruvate kinase deficiency, a rare genetic blood disorder. Doctors collected patients' own blood stem cells, corrected the faulty gene in a lab, and then infused the cells back. The m…
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Mar 16, 2026 15:26 UTC