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CureDuchenne, MDA, and Parent Project Muscular Dystrophy Fund Study to Suppress Anti-AAV Antibodies

NEW YORK – Three muscular dystrophy nonprofits have joined forces to fund research into an approach that could allow Duchenne muscular dystrophy (DMD) patients who currently aren't eligible for gene therapy access to such treatments, the organizations said Thursday.

CureDuchenne, the Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy each provided $100,000 to sponsor a one-year clinical trial in which investigators will assess whether Vyvgart (efgartigimod alfa), a drug approved by the US Food and Drug Administration to treat autoimmune diseases, can suppress anti-adeno-associated virus (anti-AAV) antibodies.

Multiple drugmakers including Regenxbio, Solid Biosciences, and Pfizer are developing gene therapies for DMD, and the FDA is expected to issue a decision on Sarepta Therapeutics' AAV-based gene therapy, SRP-9001 (delandistrogene moxeparvovec), next week.

However, a subset of DMD patients won't be eligible for such therapies on account of having anti-AAV antibodies from natural infections or previous participation in AAV-based gene therapy trials. Investigators are testing whether administering Vyvgart to those patients can lower their anti-AAV antibodies to levels that would allow safe and effective gene therapy delivery.

The clinical trial is an open-label Phase II study taking place at the Powell Center for Rare Disease Research and Therapy at the University of Florida. Investigators plan to enroll 12 DMD patients, half of whom have elevated anti-AAV titers before receiving gene therapy and the other half of whom have elevated titers after gene therapy.

The clinical trial is being led by Barry Byrne, chief medical adviser at MDA and director of the Powell Center.

"Disease-modifying therapies are no longer a thing of the future," Byrne said in a statement. "Our goal now is to figure out how to best maximize and achieve the biggest impact with the technology in our hands."