The long-term effects of rare pediatric cell therapies are unknown, preventing improvements of safety and efficacy for children with often life-threatening conditions. As compared to adults, the lack of clinical data from treated children is a major barrier to developing safer cures. This project will develop an accessible model that predicts which cell therapy product characteristics result in the most effective and durable cures in rare pediatric diseases. If successful, this innovation has a potential to positively impact ~600 devastating rare pediatric diseases. Including ultra rare diseases where correction of hematopoietic stem cells would be curative.