FDA advisers urge approval for first gene therapy for inherited disease

The Washington Post – A Food and Drug Administration advisory committee unanimously endorsed an experimental gene therapy Thursday for patients with a rare kind of hereditary blindness, setting the stage for a historic approval.

If the agency agrees with the recommendation, the one-time treatment would be the first gene therapy cleared in the United States for an inherited disorder.

Made by Philadelphia-based Spark Therapeutics, the therapy involves injecting a healthy version of the RPE65 gene — responsible for making a protein needed for sight — into the eyes of patients who have defective copies of the gene. The treatment does not give patients perfect vision but does produce substantial improvements, researchers said.

Read more at The Washington Post.

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