BioMarin Pharmaceutical, Inc., recently announced enrollment of the first patient in a phase 1/2 clinical trial for BMN 270, the company’s investigational gene therapy for the treatment of individuals with hemophilia A. The gene therapy program associated with BMN 270 was originally licensed in February 2013 from the University College London and St. Jude Children’s Research Hospital in Memphis, TN, and has since been developed by San Rafael, CA-based BioMarin.
BMN 270 uses adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that prompt the production of the factor VIII (FIX) protein that is deficient in people with hemophilia A. Ideally, AAVs deliver the genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses. The purpose of the clinical trial will be to evaluate the safety and efficacy of the therapy in up to 12 individuals with severe hemophilia A.
“Hemophilia A results from mutations at the genetic level, making gene therapy a potentially powerful technique to treat patients with a single dose,” said Hank Fuchs, MD, Executive Vice President, Chief Medical Officer of BioMarin. “For the first clinical trial of BMN 270, we are looking to demonstrate that treatment with BMN 270 increases the expression of the factor VIII protein, necessary for blood clotting.”
Source: BioMarin press release dated September 28, 2015
