Long-term KRESLADI follow-up data demonstrate survival of 100% in the absence of allogeneic hematopoietic stem cell transplantation (HSCT) from 18 to 45 months with a well-tolerated safety profile in all nine patients with severe LAD-I.
Previously disclosed results from the global RP-L102 Fanconi Anemia Phase 1/2 trial demonstrate genetic and phenotypic correction combined with hematologic stabilization extending to 42 months with polyclonal integration patterns