Gene therapy offers hope for 'Bubble Boy' disease without a donor
Disease control
Terminated
This trial tests a gene therapy for infants under 2 years old with RAG1-deficient severe combined immunodeficiency (SCID), a life-threatening condition where the immune system barely works. The therapy uses the child's own blood stem cells, modified with a corrected gene, to try …
Phase: PHASE1, PHASE2 • Sponsor: Videja B.V. • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC