Leber’s Hereditary Optic Neuropathy (LHON)

Research Pre-clinical phase Phase I or I/II  

Leber’s Hereditary Optic Neuropathy is a rare genetic disease of the retina caused by a defect in a mitochondrial gene which causes sudden and asymmetric loss of central vision, usually between the ages of 15 and 30.

The decrease in visual acuity usually affects only one eye at the beginning and can be either sudden, leading to an acuity of less than 20/400 in less than a week, or progressive, taking 2 to 3 months. The other eye is affected later.

There is currently no effective treatment for this condition.

The therapeutic approach consists in restoring the activity of the defective mitochondrial gene in retinal ganglion cells by administration into the patient’s eye of an AAV (Adeno Associated Virus) vector with a normal copy of the mutated gene.

The project was initiated based on seminal studies by Dr Marisol Corral-Debrinski and Prof. José-Alain Sahel (Institut de la Vision, Paris).

It was brought to preclinical stage in the framework of a collaboration with Genethon, and the company Gensight Biologics will sponsor its clinical development in partnership with Genethon and Institut de la Vision.

The clinical trial is currently ongoing: GenSight Biologics annonce les résultats de l’essai clinique de phase III REVERSE sur GS010 chez des patients atteints de Neuropathie Optique Héréditaire de Leber (NOHL) (3 avril 2018)