FDA Panel Considers Gene Therapy for Hereditary Eye Disease

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A gene therapy to treat a type of hereditary eye disease will be considered Thursday by a U.S. Food and Drug Administration advisory panel.

The treatment, called Luxturna, targets a condition called Leber congenital amaurosis. The vision of people with the condition is often limited to bright light and blurry shapes and most eventually lose all sight, the Associated Press reported.

A study funded by maker Spark Theraeuptics found that that the gene therapy was safe and improved vision for nearly all patients who received it.

The advisory panel will consider whether to recommend FDA approval of Luxturna. The FDA typically follows the advice of it advisory panels.

If approved, Luxturna would be the first gene therapy in the U.S. for a hereditary condition, and the first in which a corrective gene is given directly to a patient, according to the AP.

Currently, only one gene therapy is approved in the U.S. It's a cancer treatment that engineers patients' blood cells in the lab.

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