Rocket -Pharmaceuticals a leading U.S.-based multi-platform gene therapy company, today announces a research agreement to support the clinical development of Rocket’s lentiviral vector (LVV)-based gene therapy programs in Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO) towards registrational trials. Rocket’s LVV-based gene therapy program for LAD-I, RP-L201, is in clinical development with Rocket’s European partners at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT). The LVV-based gene therapy for IMO, RP-L401, is in preclinical development in Sweden with European partner Lund University.
UCLA and its Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research will serve as the lead U.S. clinical research center for the planned registrational clinical trial for LAD-I and also a lead U.S. clinical site for IMO. Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), and Molecular and Medical Pharmacology at UCLA, will serve as the principal investigator for the planned trials and will oversee the management of the clinical trial site. Additional terms of the agreement were not disclosed.