Pharma Focus Asia

First-of-its Kind Gene Therapy Could Treat Inherited Blindness

A new gene therapy might be able to recover vision lost in inherited retinal disease patients.

This treatment could pave the way for treating inherited retinal diseases and more common loss of vision due to age degeneration (Unsplash/Vanessa Bumbeers).

Leber Congenital Amaurosis (LCA) patients were capable to see well enough to navigate a maze created for trials, after receiving an investigatory gene therapy.

LCA begins in childhood and eventually causes complete blindness. It is a rare condition only affecting about 1 in every 80,000 individuals that can be caused by 1 or more of 19 different genes.

Ophthalmologist Stephen R. Russell, M.D., of the University of Iowa, is one of the lead researchers for this pioneering treatment. Data from the first randomized, controlled, phase 3 study showed that, 27 of 29 treated patients (93 percent) experienced meaningful improvements in their vision. This result showcases that the patients can be able to see in low to moderate high levels.

The treatment, called voretigene neparvovec (Luxturna, Spark Therapeutics), involves a genetically modified version of a harmless virus. The virus is modified to carry a healthy version of the gene into the retina.

There has been a progress in peripheral vision and light sensitivity, which are two visual deficits these patients experience.

The therapy is under review by the US Food and Drug Administration (FDA) for potential approval this year.

On it’s approval, doors would open to various other gene therapies which would eventually be able treat more than 225 genetic mutations known to cause blindness can be done with this research.

The gene therapy can also help to restore vision in more common diseases, such as age-related macular degeneration, a leading cause of vision loss for people over the age of 50.

Note: The research is presented at AAO 2017, the 121st Annual Meeting of the American Academy of Ophthalmology on November 10, 2017.

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