The FDA Just Greenlit a Gene Therapy That Cures Blindness

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This week, an FDA review panel made a breakthrough decision to approve of a new drug that treats an inheritable form of blindness through gene therapy. If the drug is fully approved by the agency, it will become the first gene therapy treatment for an inheritable disease approved in the US. That’s a huge deal.

The drug, made by Spark Therapeutics, is called Luxturna. It treats a condition known as Leber congenital aumaurosis, which affects vision by causing mutations to the RPE65 gene. Luxturna fixes this by inserting correct copies of the gene into retinal cells, allowing them to produce the vital enzyme which is blocked by the condition.

This FDA greenlight comes after a study demonstrated that the therapy significantly improved peoples vision. Of the 21 patients, 11 showed much improved vision, while 93 percent had some improvement in vision.

The drug was unanimously approved of by the committee this week. They also released a paper on the topic, stressing that though they believed the treatment worked to alleviate blindness caused by the disease, there is so far no evidence of how the medication works in the long term.

The FDA now has until January to decide whether to approve the therapy.

Source: FDA via Gizmodo

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