NEC Member Massachusetts Eye and Ear recently announced its first injection of a gene therapy drug, Luxterna, which treats a form of blindness, following approval of the therapy by the FDA in December 2017.
Gene therapies modify a patient’s DNA by inserting healthy genes into cells to replace faulty or missing ones. 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 which prevent the production or function of a protein needed for proper functioning of the retina. Jason Comander, M.D., Ph.D., associate director of the hospital’s inherited retina disorders service, performed the procedure on a thirteen -year-old boy from New Jersey using the gene therapy drug which is manufactured by Spark Therapeutics.
“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,” Dr. Comander said.
While costly, gene therapies have the potential to cure a wide range of diseases and have spawned scores of new biotechs in recent years, including several in Massachusetts. Massachusetts Eye and Ear is one of eight medical centers in the country that is certified to inject Luxterna in patients and remains the only site to do so in New England