Rocket Pharmaceuticals gets FDA fast track status for RP-L401 in infantile malignant osteopetrosis
Rocket Pharmaceuticals has secured fast track designation for RP-L401 gene therapy candidate from the US Food and Drug Administration (FDA) for the treatment of infantile malignant osteopetrosis (IMO). RP-L401 is a lentiviral vector (LVV)-based gene therapy which is being studied in a non-randomized, open-label phase 1 clinical trial for the treatment of the rare childhood […]