Belief BioMed has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (RPDD) for its gene therapy drug, BBM-D101 injection, indicated for the treatment of Duchenne muscular dystrophy (DMD).
BBM-D101: A Pioneering rAAV Gene Therapy for Duchenne Muscular Dystrophy
BBM-D101 injection, developed and manufactured by Belief BioMed, is a novel recombinant adeno-associated virus (rAAV) gene therapy. The treatment utilizes an engineered AAV as a vector to deliver an optimized gene expression cassette systemically to muscles, with the goal of effectively treating DMD. The drug has officially commenced Investigator-Initiated Trials (IIT) as of July 2024.- Flcube.com
