One-Shot gene fix for blood disorder enters human testing
Disease control
ENROLLING_BY_INVITATION
This early-stage trial tests a one-time gene therapy called BRL-101 for people with transfusion-dependent beta-thalassemia, a severe blood disorder. The treatment uses the patient's own stem cells, edited with CRISPR to boost healthy hemoglobin production. The goal is to see if a…
Phase: PHASE1, PHASE2 • Sponsor: Bioray Laboratories • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC