For those living with an inherited form of blindness that comes on suddenly, a one-time injection could be life-changing.
Leber’s hereditary optic neuropathy (LHON) is a rare form of blindness that comes on suddenly in young adults. It’s a genetic disorder that is diagnosed in about 1,500 people a year. Once it begins to erode someone’s vision, there is no existing treatment that can bring it back.
But GenSight Biologics, a French biotech company, thinks the solution to the condition could come from injecting the eye with a virus carrying new genes that could help restore part of a person’s sight.
The company’s gene therapy approach is currently in the middle of two phase 3 trials, each with 36 people in them in Europe and the US. One trial will see if the therapy can be used as soon as a patient is diagnosed with LHON to keep them from fully losing their eyesight. GenSight CEO Bernard Gilly told Business Insider that the trials should finish enrolling by the end of 2016, and then with a year of follow-up, wrap up by early 2018.
The treatment works like this: In people with LHON, the optic nerve cells in the eyes have trouble creating energy, so they begin to die. By injecting a virus that carries a certain gene that can keep these cells from dying, GensSight hopes they can keep the disease from progressing, and even reverse some of its effects.
The trials garnered additional attention after an American 17-year-old, who was at first too young to participate in the trial, got the OK to take part. He’ll get the therapy in October, Gilly said.
But these kind of procedures are not easy. Gene therapy has made a comeback after being stalled out in 1999 following the death of an 18-year-old who was taking part in a experimental trial died. Since then, more companies have started developing gene therapies, with a few getting approval in Europe, but the FDA has not approved any yet. That means the road to approval will likely be long and difficult, and in this second wave of potential gene therapies, there will likely be many that fail.
If it does get approved, GenSight is thinking it will cost more like what a organ transplant would cost: a one-time payment of $500,000 to $1 million.
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