One-Shot CRISPR treatment aims to free patients from lifelong blood transfusions
Disease control
ENROLLING_BY_INVITATION
This early-stage study is testing a single infusion of a patient's own genetically modified stem cells to treat severe beta-thalassemia. The therapy uses CRISPR gene editing technology with the goal of helping the body produce healthy red blood cells. If successful, it could redu…
Phase: PHASE1, PHASE2 • Sponsor: Bioray Laboratories • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC