The FDA has approved Luxturna, a breakthrough new gene therapy eye medicine for a very rare inherited retinal disease called “biallelic RPE65”. Approximately 1 to 2 thousand people in the United States have this disease, but perhaps an even larger significance to this medicine is that it sets the stage to treat other, more common genetic eye disorders and other genetic diseases throughout the body. This is a landmark drug in that it is the first FDA approved gene therapy for an inherited disease. Gene therapies also hold great promise for cancer treatment. Luxturna  directly delivers a normal gene into cells in the retina to work along side the abnormal inherited genes. This newly inserted gene allows the cells to produce normal proteins that can actually restore sight to the patients who otherwise would be blind. Before this medicine was developed, there was a very poor prognosis for patients with this disease as there were no other viable treatment options. Interestingly, unlike most medicines, Luxturna is a one-time treatment. It is extremely expensive, but third party payers are working on ways to cover payment for this drug. While this eye drug is not in any way used for LASIK, I wanted to blog about Luxturna given its extreme medical significance.