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The idea is to use tiny modified viruses known as adeno-associated viruses (AAVs) to deliver genes to fix underlying genetic problems for patients with X-linked myotubular myopathy and Pompe disease. AAVs have long been attractive to gene therapy researchers because they have a virus’s natural ability to efficiently get inside cells. They’re also thought to be safer than other viruses, like retroviruses or adenoviruses. The newest generations of AAVs have shown good safety profiles in humans, an ability to be efficiently distributed throughout tissues in the body, and can even be modified to be delivered to specific tissue types if desired, Patterson says.
The first disease, X-linked myotubular myopathy, is a good candidate for gene therapy because it’s a single gene defect in newborns that is deadly after a couple years, and currently has no effective therapy, Patterson says. About one in 50,000 newborn males worldwide are thought to have this condition, according to the National Institutes of Health.
The second program at Audentes, for Pompe disease, is different in that there are already a couple of enzyme-replacement therapies available—Sanofi/Genzyme’s alglucosidase alfa (sold as Myozyme and Lumizyme)—although those treatments have limited effectiveness, Patterson says. Audentes is hopeful that by making a gene therapy for Pompe, it might be able to penetrate skeletal muscle tissues, and motor neurons, which could address some of the muscle weakness and respiratory problems that patients often struggle with while on the existing therapies, Patterson says. An estimated one out of every 40,000 children is born with Pompe.
Audentes doesn’t have any data yet from clinical trials, but it has gotten its venture investment based on promising animal studies, Patterson says. In Florida, Byrne is running a clinical trial with an older version of an AAV delivery vector in clinical trials for Pompe, which could help pave the way for Audentes to move ahead with a newer version, Patterson says.
With the financing in hand, Audentes is moving ahead to flesh out its management team by making 10-20 hires, Patterson says. He’s looking for people with experience in the science of AAV vectors, manufacturing them, and for clinical development types with experience in rare diseases. Although Patterson has personally lived and worked in both Boston and San Francisco in the past—both great biotech hubs—he picked the Bay Area as the place for Audentes largely to tap into its talent pool. He says he’s gotten more than a few inquiries from potential hires since the Series A financing announcement, and he’s eager to get a few of those folks on staff to help him and the company’s scientific advisors. “[Job] offers are going out the door,” Patterson says.