autologous CD34+-enriched HSPCs transduced with lentiviral vector carrying FANCA gene RP-L102

A preparation of autologous CD34+-enriched hematopoietic stem and progenitor cells (HSPCs) from mobilized peripheral blood of patients with Fanconi anemia subtype A (FA-A) that are transduced ex vivo with a lentiviral vector carrying the FANCA gene, with potential to restore FANCA expression and function. Upon re-infusion of autologous CD34+-enriched HSPCs transduced with lentiviral vector carrying FANCA gene RP-L102 back into the patient, these cells express functional FANCA gene. Mutations in the FANCA gene, common in patients with FA, prevent normal DNA repair which may lead to chromosomal breakage, increased sensitivity to oxidative and environmental stress, and bone marrow failure.