FDA panel mulls gene therapy for kids with rare eye disease

U. S. Food and Drug Administration advisory panel was poised on Thursday to recommend approval for a gene therapy that could grant the gift of sight to young people with a rare type of inherited vision loss. 

Replacing a nonworking gene with a new one is opening a new world for children and teens with the inherited retinal disease called Leber congenital amaurosis.

"This is a gene therapy that can restore some vision to people who have very limited vision or no vision due to the mutation in the RPE65 gene, and as such, it's a great breakthrough," said Stephen Rose, chief research officer at the Foundation Fighting Blindness. For those who have already received the therapy, the treatment has been life-changing.

If approved by the FDA advisory panel, the treatment would be on its way to becoming the first gene therapy approved for an inherited disease, said Rose. His foundation helped fund the research that led to the treatment. The FDA typically follows the advice of its expert panels.

Only one other gene therapy has so far met FDA approval - a cancer treatment that the agency sanctioned in August.

This type of vision loss is rare and affects about 1,000 people in the United States, Rose said. "But for these individuals who are essentially blind, it's a huge impact," he said.

In total, about 200,000 Americans have some type of inherited disease that causes blindness involving some 250 different genes, Rose said.