BOSTON—Massachusetts Eye and Ear made medical history on Tuesday by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The treatment, commercially identified as Luxturna, was developed by Spark Therapeutics and approved in December by the Food and Drug Administration for patients aged 12 months and older. Luxturna has been shown to improve visual function in children and adults with inherited retinal disease caused by mutations in the gene RPE65.

It is the first and only FDA-approved gene therapy treatment for an inherited disease. The landmark procedure was performed by Jason Comander MD, PhD, associate director of the Inherited Retinal Disorders Service at Mass. Eye and Ear in Boston. The inaugural patient was a 13-year-old boy from New Jersey.

Dr. Commander said, "It is an honor to be involved in this exciting procedure following FDA approval, and to play a role in allowing a 13-year-old boy an opportunity to enjoy improved vision for years to come. This project has been underway for 20 years, and the early successes with this therapy demonstrated by Jean Bennett, Al Maguire and others, inspired me to dedicate my career toward helping patients with inherited retinal diseases. I am so excited the time has come when we can offer this groundbreaking therapy to our patients, who are truly in need of our help," he said.

The newly approved treatment involves injecting a modified virus into a patient's eyes to correct a deficiency caused by mutations in the RPE65 gene. These mutations prevent the production or function of a protein needed for proper functioning of the retina, the light-sensitive tissue in the back of the eye that initiates vision.

Mass. Eye and Ear is one of just eight medical centers in the U.S. certified to perform the gene therapy, and the only site in New England.