Gene fix for blood disorder shows early promise in small trial
Disease control
Ongoing
This early-stage study tests a gene therapy for people with beta-thalassemia major, a severe inherited blood disorder. Doctors take the patient's own stem cells, fix the faulty gene in the lab, and return the corrected cells through an IV. A low dose of busulfan is given beforeha…
Phase: PHASE1 • Sponsor: San Rocco Therapeutics • Aim: Disease control
Last updated May 17, 2026 05:05 UTC