Groundbreaking Gene Therapy for Retinal Disease

In March, a 13-year-old boy, Jack Hogan, was the first patient to receive an FDA-approved gene therapy for inherited blindness called Luxturna. The treatment, administered by RPB-supported researchers and physicians at Massachusetts Eye and Ear, is designed to improve visual function in children and adults with inherited retinal disease caused by mutations in the gene RPE65. The lead surgeon is an RPB Career Development Awardee, Jason Comander, MD, PhD, Associate Director of the Inherited Retinal Disorders Service at Mass. Eye and Ear. 

According to Mass. Eye and Ear, Luxturna 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. 

Since receiving his treatment, Hogan has experienced a significant impact on his quality of life. He’s enjoying better vision in low light, such as when playing basketball outside in the evening with friends or going to the movies, and is reading 40 percent smaller print.

"It is truly amazing to see large improvements in Jack’s vision, which would have been impossible without this treatment," said Dr. Comander.  "It is going to make a big difference in his life. These results are representative of just how big a moment this is for gene therapy; it is helping our patients and it is here to stay."

August 14, 2018