One-Shot gene editing could end debilitating pain crises
Disease control
Ongoing
This study is testing a new, one-time treatment for severe sickle cell disease. Doctors take a patient's own blood stem cells, edit their genes in a lab, and then return them to the patient. The goal is to see if this single treatment can safely and significantly reduce the numbe…
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC