Dr. Stephen Russell, along with other doctors, patients and parents, presented results from a Phase 3 clinical trial to a Food and Drug Administration (FDA) advisory panel. The panel sought to evaluate a treatment developed by Spark Therapeutics and known as voretigene neparvovec for individuals with RPE65-mutation associated retinal dystrophy. The genetic defect causes Leber congenital amaurosis (LCA), an inherited disorder that progressively leads to blindness.
The clinical trial involved using a genetically engineered virus to correct the genetic defect. The virus is modified to carry a healthy version of the gene and is surgically placed into the retina. Results thus far show significant improvements in vision for many of the patients enrolled in the trial.
Experts on the FDA panel voted unanimously to approve the gene therapy. The treatment will now progress to a final decision from the FDA and, if approved, would be the first of its kind approved for use in the U.S.
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