FDA may approve gene therapy that could cure hereditary blindness


FDA may approve gene therapy that could cure hereditary blindness

There may soon be a cure for some forms of blindness, because the U.S. Food and Drug Administration is considering a gene therapy that will help those who are visually impaired. 

The organization made the announcement earlier this week, revealing that a treatment called Luxturna, made by Philadelphia-based Spark Therapeutics, has already worked for three youth and other individuals with hereditary blindness. 

It would be the first gene therapy in America for an inherited disease and the first method that would give a corrective gene directly to a patient, according to FDA briefing documents

So how does it work?

The procedure tackles Leber congenital amaurosis, or LCA, a condition caused by flaws in the RPR65 gene, the FDA briefing documents state. Those with LCA do not have the ability to produce protein that the retina and tissues at the back of the eye need to convert light into signals that are translated by the brain. Therefore, those with it typically only see bright lights and blurry shapes, which eventually lead in blindness.  

To correct it with Luxturna, doctors puncture the white part of the eye and inject a modified virus, which contains the corrective gene, into the retina, Spark Therapeutics explained. Patients see results in a month. 

While it doesn’t work for everyone and doesn’t provide 20/20 vision, the company noted it has been mostly successful thus far and proven safe. Scientists are still determining whether the procedure needs to be conducted more than once. 

Now children, parents, doctors and scientists are preparing to speak before a FDA panel on Thursday, and the administration must make a decision by Jan. 18. 

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