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Interim Phase I/II trial results show Rocket’s ex vivo lentiviral gene therapy candidate, RP-L201, is safe and of benefit to paediatric patients with severe Leukocyte Adhesion Deficiency-I.
Rocket Pharmaceuticals, Inc., a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L201.