GenSight Biologics, a french biopharma company, just submitted to EMAMarketing Autorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON), a rare, mitochondrial genetic disease, caused by mutation in the ND4 mitochondrial gene.
LHON mainly affects young males, between 15 and 30 years old. The ND4 mutation results in the worst possible visual outcome, with most patients becoming legally blind. The therapeutic approach involves restoring the activity of the failed mitochondrial gene in the retinal cells by administering to the patient’s eye an Adeno Associated Viral (AAV) vector carrying a normal copy of the mutated gene.
It is the first gene therapy for mitochondrial disease. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to the eye by intravitreal injection to offer patients a sustainable functional visual recovery.
This project is the result of Dr. Marisol Corral-Debrinski and Pr. José-Alain Sahel’s (Institut de la Vision, Paris) original works, in collaboration with Genethon until completion of preclinical stage. Gensight Biologics ensured clinical development in partnership with Genethon, during the early phases, and with the Institut de la Vision.
Clinical trials in France and USA showed significant improvement in vision, both in terms of visual acuity and sensitivity to contrasts.
Genethon welcomes this new step for this gene therapy, specifically developed by GenSight Biologics for the treatment of LHON associated with mutation in the ND4 gene, a major step for the 800-1200 new LHON patients in Europe and the U.S. each year.