Leber hereditary optic neuropathy, abbreviated as LHON is an inherited form of vision loss. It is a chromosomal disorder that majorly affects central vision, which is vital for tasks like reading, driving, and recognizing faces. Vision loss is practically the only symptom of this disorder, although there might be a few exceptions. LHON has no curative treatment. Approximately 95% of cases are caused by gene mutations. These genes that undergo mutations are involved in ATP metabolism and encode for the NADH dehydrogenase complex.
A French biotech company, GenSight is the only biotech on the path to develop a gene therapy for this disorder. Now, its candidate drug, GS010, has proven to be extremely useful and has showcased highly promising results after two years of trial; it is now being tested on patients in a Phase II trial. A patient’s ETDRS test or the early treatment diabetic retinopathy study which is used to evaluate changes in vision was carried out. Then these subjects were treated with GS010. It was found that they exhibited undeniable improvement in the number of letters they were able to read over time. The study follow-up details that stretched over a period of 96 weeks are on
route to be published.GenSight is presently carrying out two studies in Europe and in the US in Phase III evaluating the further effect GS010 has in patients with a ND4 mutation which could possibly be affected by LHON for a year or less.
“We are now less than a year away from Phase III efficacy data, and more than ever committed to find a cure for patients and their families affected by this devastating condition,” said Bernard Gilly, CEO and co-founder of Gensight, in a press release.
