Gene therapy aims to fix sickle cell at its source
Disease control
Ongoing
This study tests a gene therapy for people with severe sickle cell disease. The approach uses a patient's own blood stem cells, modified to boost fetal hemoglobin, which can reduce sickling. Ten participants will receive the treatment to see if it is safe and can increase fetal h…
Phase: PHASE1 • Sponsor: David Williams • Aim: Disease control
Last updated May 01, 2026 16:00 UTC