Leber’s Hereditary Optic Neuropathy is a rare genetic disorder of the retina caused by a defect in a mitochondrial gene which causes a sudden and asymmetric loss of central vision, usually between the age of 15 and 30. There is currently no effective treatment for this condition.
The therapeutic approach is to restore the activity of mitochondrial gene defective in retinal ganglion cells by administering to the patient’s eye of an AAV vector (Adeno Associated Virus) with a normal copy of the mutated gene.
This project has been initiated from the 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 will sponsor its clinical development in partnership with Genethon and Institut de la Vision.
The clinical trial is currently ongoing.
The gene therapy vector is produced at Genethon.