NEW YORK – Researchers at the Indiana University School of Medicine were awarded a $3.8 million grant from the National Institutes of Health's National Institute of Allergy and Infectious Diseases to investigate response to gene therapies for degenerative muscle disorders, the university said Thursday.
The five-year grant will support research into the body's initial immune response to muscle-directed adeno-associated virus (AAV) vector-based gene therapies, such as how to prevent a patient's immune system from attacking treated muscles and promote longer or more targeted responses. Researchers will also test alternative treatment options for disorders like Duchenne muscular dystrophy (DMD).
"It's an especially noteworthy time for DMD gene therapies as companies trying to get their treatments on the market face extra scrutiny from scientists and the US Food and Drug Administration," Roland Herzog, an immunology professor in IU School of Medicine's pediatrics department who is leading the research, said in a statement. "Our goal is to make gene therapies safer and more effective."
The FDA, for example, is expected to issue a decision on SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics' investigational AAV gene therapy for DMD, by June 22.
Herzog previously coauthored an article published last month in Human Gene Therapy reviewing evidence of immune responses against vectors or transgenes in muscle-directed AAV gene therapies, which can lead to immunotoxicities. One of the coauthors on the article, Dongsheng Duan, a professor at the University of Missouri School of Medicine, is also a co-investigator on the immune response study.