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Working Wonders: Gene therapy for patients with a form of inherited blindness

Posted March 26, 2018

Image credit: Jacek Halicki via Wikimedia(CC BY-SA 4.0)

Harvard Medical School researchers at Massachusetts Eye and Ear made medical history this week by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine as it is the first time any FDA-approved gene therapy has been given to a patient for an inherited disease.

The treatment, commercially identified as Luxturna, was developed by Spark Therapeutics and approved in December by the U.S. Food and Drug Administration (FDA) for patients ages 12 months and older. It 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, HMS assistant professor of ophthalmology and associate director of the Inherited Retinal Disorders Service at Mass. Eye and Ear. The inaugural patient was a 13-year-old boy from New Jersey.

“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,” Comander said. “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.”

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.

“I am proud of our team for earning the distinction of being selected as a Center of Excellence for the administration of Luxturna,” said Eric A. Pierce, the HMS William F. Chatlos Professor of Ophthalmology and director of the Inherited Retinal Disorders Service at Mass. Eye and Ear. “It was a natural fit because of our department’s ongoing experience with developing gene and genetic therapies here in the Ocular Genomics Institute and with their testing in ongoing clinical trials.”

“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. We are thrilled to be one of the first centers to offer this life-changing therapy to patients, and proud of our long, successful history of developing translational retinal therapies,” said Joan Miller, the HMS David Glendenning Cogan Professor of Ophthalmology, chair of the Department of Ophthalmology and chief of ophthalmology at Mass. Eye and Ear and Mass General Hospital.  “Our hope is that our ophthalmology community can leverage Luxturna’s success to accelerate the development of similar gene therapies for the many blinding retinal diseases that still afflict our patients.”

Source: HMS

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