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UF teams with company to advance gene therapy treatments

A biotechnology company has raised $30 million to advance gene therapy treatments developed at the University of Florida Powell Gene Therapy Center. The funding also will help UF Health researchers bring a novel form of gene therapy to patients with Pompe disease, a rare form of muscular dystrophy.

The involvement of the company, Audentes Therapeutics Inc., should help scientists speed potential treatments to the patients who need them most, said Barry Byrne, M.D., Ph.D., director of the Powell Gene Therapy Center and a professor of pediatrics in the UF College of Medicine, who is collaborating with the company on Pompe disease studies. Eventually, the research generated from this collaboration could lead to additional gene therapy treatments for other congenital muscular diseases, Byrne said.

“The ability to have a commercial sponsor for a scientific program allows our discoveries to be developed into real products that will help people,” Byrne said. “And that is really the goal.”

In gene therapy, doctors use harmless viruses as a vehicle to shepherd corrective genes into the body. Once there, these genes compensate for the faulty ones causing disease.

Byrne’s team has already conducted safe and effective gene therapy studies in human patients with Pompe disease. Now, with the help of Audentes, scientists are testing what they hope will be a more effective vector for delivering corrective genes.

UF researchers pioneered the use of adeno-associated viruses to transport therapeutic genes safely into the body. Now, Byrne and colleagues are using a new form of the virus, AAV-9, to conduct gene therapy on Pompe disease patients.

Earlier trials showed that another vector, AAV-1, was safe and produced positive results when used to transport corrective genes into patients. But because AAV-1 could not penetrate the central nervous system, the therapy only affected the areas of the body where the genes were injected, improving only some aspects of a patient’s disease. AAV-9, on the other hand, can deliver genes not only to affected muscles and organs, but also into the more difficult-to-reach central nervous system. If successful, researchers potentially could provide patients with a single-dose treatment that corrects the disease throughout the body, Byrne said.

In addition to UF, the company also is teaming with Harvard Medical School, the University of Washington and Boston Children’s Hospital on a gene therapy program for X-linked myotubular myopathy, another inherited muscular disease.

“This collaboration exemplifies our goal of putting the patient first by taking basic research and seeing it all the way into improving care for patients — an essential part of our strategic plan,” said David S. Guzick, M.D. Ph.D., UF senior vice president of health affairs and president, UF Health.

“This financing is a tremendous step forward for the development of new potential treatments for patients affected by very serious orphan muscle diseases,” said Matthew R. Patterson, president and CEO of Audentes, in a written statement. “I’m delighted to have the opportunity to work with such a fantastic group of people who share our vision for success in bringing these therapies to patients.”

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