Making Gene Therapy History
There’s now an FDA-approved gene therapy drug for inherited blindness. Meet the first patient in the United States to receive it, Jack Hogan, and his care team from Mass. Eye and Ear.
Last week, medical history was made in an operating room at Mass. Eye and Ear.
A team led by Jason Comander, M.D., Ph.D., performed the first ever post-FDA approval gene therapy administration for patients with a form of inherited blindness. The inaugural patient was Jack Hogan, a 13-year-old from New Jersey. Jack’s surgery marked the first time that any FDA-approved gene therapy had been given to a patient for any inherited disease.
Jack was born with retinitis pigmentosa associated with RPE65 gene mutations. He’s night-blind, meaning that he can’t stay outside after dark to play basketball with his friends, and he’s losing peripheral vision at a growing rate. Without treatment, Jack would become legally blind by his 20s or 30s.
Gene Therapy for Inherited Blindness
The treatment, known as Luxturna, was developed by Spark Therapeutics and approved in December by the Food and Drug Administration (FDA) 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.
Much of the work leading to Luxturna’s development began at the University of Pennsylvania, in a lab run by Jean Bennett, Ph.D.
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.
We’ve entered a new era of medicine. One where patients with a form of inherited blindness can receive a gene therapy drug – not as part of a clinical trial, but as part of a treatment plan.
Mass. Eye and Ear is so proud to be a part of this groundbreaking moment.
For more on Jack, Dr. Comander and gene therapy for blindness, watch the video above (Download Transcript). We’ll be sure to keep you posted on Jack – and the great hope we have that this treatment will improve his vision.