A team from the Massachusetts Eye and Ear performed the first FDA-approved gene therapy procedure for patients with inherited RPE65 mutation-associated retinal dystrophy, a condition that leads to vision loss and complete blindness. The treatment, Luxturna (voretigene neparvovec-rzyl), is the first and only FDA-approved gene therapy treatment for an inherited disease. It was approved last December with the inaugural procedure taking place on March 20, 2018, on a 13-year-old boy from New Jersey.
Jason Comander M.D., Ph.D., Associate Director of the Inherited Retinal Disorders Service at Mass. Eye and Ear in Boston performed the landmark procedure. “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,” said Dr. Comander. “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.”
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 United States certified to perform the gene therapy, and the only site in New England.
“Today we celebrate the decades of work by many individuals to bring gene therapy from science fiction to clinical reality for patients with inherited retinal disease. Gene therapy will now join the list of innovative therapies used routinely at Mass. Eye and Ear to treat patients, said Joan W. Miller, M.D., Chief of Ophthalmology at Mass. Eye and Ear and Mass General Hospital.
Luxturna was developed by Spark Therapeutics. See the full press release from Massachusetts Eye and Ear.
Source: Massachusetts Eye and Ear