Gene fix for thalassemia: first human test shows promise
Disease control
Ongoing
This early-stage study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the …
Phase: PHASE1 • Sponsor: San Rocco Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC