GS010 (gene therapy)

The French biotechnology company Gensight are working on a gene-therapy based approach to treating mitochondrial diseases LHON (Leber's Hereditary Optic Neuropathy).

Gene therapy is the introduction of normal genes into cells, in place of missing or defective ones in order to correct genetic disorders.

Gensight published results from its Phase I/II study in June 2016 which confirmed good safety and tolerability of GS010 and improvement of visual acuity in patients with an onset of disease of less than 2 years.

Since then they have initiated two Phase III clinical trials in 6 European centres and in the USA to assess the efficacy of GS010 in ND4 LHON patients with earlier onset and results are expected in late 2017.