The FDA approved a gene therapy that can reverse a form of childhood blindness

The US Food and Drug Administration just approved a new gene therapy that can treat an inherited disease.

The FDA gave doctors the clearance to use Luxturna, a gene therapy, to treat the effects of biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic eye disease. Patients have two copies of a mutated gene (one from each parent) called RPE65, which gradually leads to severely impaired sight in childhood and, in some cases, blindness by adolescence. Luxturna can stop and even in come cases reverse the effects of the condition.

Luxturna, made by Spark Therapeutics, is essentially a benign virus with a functional version of the gene. It’s injected directly into the retina. Once in the eye, the gene can do some of the work the patient’s own mutated copies cannot, which stops the progress of the disease, and in some cases, improves vision. The FDA approved Luxturna after a stage three clinical trial in which patients who received the drug could navigate an obstacle course at night significantly better than those who received a placebo injection. Some patients in the trial who got the injection recovered their ability to do things like read, play sports, and see the stars, according to NPR.

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