Gene therapy offers hope for rare blood disorder
Disease control
Completed
This phase 1 trial tested a gene therapy called RP-L301 in 4 people with pyruvate kinase deficiency, a rare inherited blood disorder that causes severe anemia and often requires frequent blood transfusions. The treatment uses the patient's own blood stem cells, which are modified…
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:53 UTC