Overview
- In the largest cohort to date, 62 children were treated between 2012 and 2019; 59 achieved durable immune reconstitution with 100% survival over a median 7.5 years of follow-up (~474 patient-years).
- Responders discontinued enzyme replacement, nearly all stopped immunoglobulin infusions, and they developed normal responses to routine vaccinations.
- Most adverse events were mild or related to conditioning chemotherapy, with no treatment-limiting safety signals reported among responders.
- More than half received cryopreserved gene-corrected stem cells with outcomes comparable to fresh products, supporting centralized processing and wider geographic access.
- The program has been licensed to Rarity PBC, which is preparing pharmaceutical-grade manufacturing as investigators aim to seek FDA approval within two to three years.