On Friday, REGENXBIO Inc. (NASDAQ:RGNX) shared new data from the Phase 1/2/3 CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025.
REGENXBIO submitted these longer-term pivotal results to the U.S. Food and Drug Administration (FDA) in response to an information request in the ongoing Biologics License Application (BLA) review of RGX-121.
