New gene therapy aims to fix sickle cell disease at its root
Disease control
Recruiting now
This early-stage study tests a new gene therapy called nula-cel in 15 people with severe sickle cell disease. The treatment uses the patient's own blood stem cells to change the sickle hemoglobin into healthy adult hemoglobin. The main goals are to check safety and see if it can …
Phase: PHASE1, PHASE2 • Sponsor: Kamau Therapeutics • Aim: Disease control
Last updated May 26, 2026 10:36 UTC