Gene therapy breakthrough: could a single infusion free thalassemia patients from lifelong transfusions?
Disease control
ENROLLING_BY_INVITATION
This study tests a one-time gene therapy called BRL-101 for people with transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. The therapy uses the patient's own blood stem cells, modified with CRISPR-Cas9, to help the body produce healthy re…
Phase: PHASE1, PHASE2 • Sponsor: Bioray Laboratories • Aim: Disease control
Last updated May 11, 2026 20:39 UTC