Grant furthers treatment for rare eye disease

ALACHUA – A gene therapy company in Alachua and the University of Florida's ophthalmology department are closer to developing a treatment for a rare eye disease.

AGTC in Alachua and the university's ophthalmology department secured an $8.4 million grant from the National Eye Institute to help them study a treatment for a genetic form of vision impairment called achromatopsia.

"It's really quite a debilitating form of blindness," said William Hauswirth, a professor from UF who will be the principal investigator in charge of the research. There will be multiple sites where the studies are conducted, including New Jersey, Miami, Chicago, Wisconsin, Alachua-based AGTC, the University of Florida and the University of Oregon

"We already have very encouraging results in several animal models that this treatment has the potential to restore vision function,” Hauswirth said.

Achromatopsia is well understood, but funding has been the issue in treating it, he said. It's estimated there are 10,000 patients of the disease in North America and Europe.

The disease is caused by a missing gene which the company hopes to correct by injecting a virus behind the patient's retina carrying the missing pieces of the patient's genetics.

"It's not a complicated surgery," Hauswirth said.

The animal-testing phase of the research is already finished.

The grant will last for five years. FDA approval is still several years off, though. Human trials could begin within three to five years, treating about 50 patients, Hauswirth said. The human trials should go on for about four years.

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