Gene therapy offers hope for children with SCID-X1
Disease control
Recruiting now
This trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC