Rocket Pharmaceuticals, Inc. announces that the U.S. FDA has granted Fast Track designation to RP-L401, the Company’s lentiviral vector -based gene therapy for the treatment of Infantile Malignant Osteopetrosis, a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure.
August 27, 2020
· 7 min read