New gene therapy aims to rebuild immune system in 'Bubble Boy' disease
Disease control
Recruiting now
This study is testing a one-time gene therapy for young children with SCID-X1, a life-threatening genetic disorder where the immune system doesn't work. Doctors will collect the child's own blood stem cells, fix the faulty gene in a lab, and then infuse the corrected cells back a…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC