Clinical Trials:

Leukocyte Adhesion Deficiency-I (LAD)

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RP-L201 is being developed for severe LAD-I, a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant (HSCT).

The Biologics License Application (BLA) has been accepted with Priority Review by the U.S. Food and Drug Administration (FDA), based on the positive efficacy and safety data from the Phase 1/2 study of RP-L201 for severe LAD-I. The Prescription Drug User Fee Act (PDUFA) target action date is June 30, 2024.

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