Clinical Trials:

RP-L102 is being developed for FA, complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. FA impacts an estimated 5,500 to 7,000 individuals in the U.S. and EU.

An open-label, global registrational clinical trial of RP-L102 for the treatment of FA has completed enrollment. The trial enrolled 12 patients total from the U.S. and EU. The primary endpoint of the trial is mitomycin-C (MMC) resistance, a measure of bone marrow cell correction. The Marketing Authorization Application (MAA) has been accepted for review by the European Medicines Agency (EMA) based on the positive efficacy and safety data from the Phase 1/2 study of RP-L102. The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.