June 29, 2020
Rocket Pharmaceuticals gets FDA clearance for its gene therapy for Infantile Malignant Osteopetrosis
Rocket Pharmaceuticals has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-L401, its, lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder.