First Gene Therapy for Inherited Retinal Disease restores sight to two young patients at the Royal Victoria Eye and Ear Hospital

The Royal Victoria Eye and Ear Hospital (RVEEH) successfully delivered Luxturna, the first gene therapy developed for an eye condition, to two young patients with inherited retinal disease marking a significant advancement for patients in Ireland living with this rare condition.

The procedure was carried out by husband and wife team, Mr Max Treacy, Consultant Vitreoretinal Surgeon and Ms Emma Duignan, Consultant Ophthalmic Surgeon. A 20 year old patient with biallelic RPE65-mediated inherited retinal dystrophy, received gene therapy to both eyes. Following the initial procedure, the patient's visual field doubled within a month, and he progressed from being unable to read any letters on an eye chart to reading the second line. His 15-year-old sister, with the same genetic condition, also received gene therapy to her first eye and is recovering well. She is scheduled for her second-eye treatment.

What is Luxturna?

Luxturna is a one-time treatment that works by replacing a faulty gene in the retina with a correct copy, enabling the body to produce a missing protein necessary for vision. The treatment is delivered during a vitrectomy, a surgical procedure that removes the jelly from the eye, followed by injection of the gene therapy beneath the retina. The corrected gene is carried by a viral vector. Once the protein is restored, visual function is expected to improve gradually over time. The treatment was funded through the HSE’s Managed Access Protocol.

RVEEH became an accredited gene therapy centre following a four-year onboarding process. The hospital is now participating in an additional gene therapy clinical trial, ensuring access to cutting-edge treatments for patients in Ireland.

Dr. Paul Kenna, who pioneered research into genetic eye diseases at RVEEH since the 1980s, was also present during the procedure. Dr. Kenna, part of the team that identified the first gene causing these disorders in 1989, described the moment as profoundly meaningful. Today, over 350 such genes have been identified.