GenSight Biologics announces that the journal Science Translational Medicine has just published the results of REVERSE, a pivotal phase III clinical trial of LUMEVOQ gene therapy in patients with hereditary optic neuropathy of Leber ND4 (LHON), as well as the main results of a study in primates investigating the contralateral effect of gene therapy. This publication, which appeared in the December issue under the title “ Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy ” is the first article on the subject of Leber’s hereditary optic neuropathy. published in a peer-reviewed scientific journal documenting, from phase III clinical data, a prolonged and clinically significant bilateral improvement in visual acuity following unilateral injection of gene therapy.
The results of the REVERSE trial and the primate study are essential elements of the body of data submitted by GenSight in September 2020 to the European Medicines Agency to support its application for marketing authorization for LUMEVOQ in the treatment of patients with vision loss secondary to LHON caused by a confirmed mutation in the mitochondrial ND4 gene. The Agency is expected to render its decision in the fourth quarter of 2021.
“This is a proven safe treatment that has the potential to change the lives of patients,” says Dr. Patrick Yu-Wai-Man, lead author of the article, principal investigator of the ‘essay REVERSE, Lecturer and Honorary Consultant Ophthalmologist at the University of Cambridge, Moorfields Eye Hospital and the UCL Institute of Ophthalmology, London, UK.
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