First publication based on Phase III data to document sustained and clinically meaningful bilateral improvement in visual acuity from unilateral injection of a gene therapy
Non-human primate study clarifies mechanism behind contralateral effect
Paris, France, December 10, 2020, 7:30 am CET –
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the journal Science Translational Medicine has published results from the REVERSE pivotal Phase III clinical trial of LUMEVOQ® gene therapy in ND4 Leber Hereditary Optic Neuropathy (LHON) subjects along with key results from a non-human primate study investigating the contralateral effect of the gene therapy. The paper*, published in the December issue under the title “Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy”, is the first peer-reviewed article based on Phase III clinical trial data to document sustained and clinically meaningful bilateral improvement in visual outcomes from a unilateral injection of a gene therapy.
The findings from the REVERSE trial and the non-human primate study were key components of the data package submitted by GenSight Biologics in September 2020 to the European Medicines Agency when it applied for marketing authorization for LUMEVOQ® as treatment for patients with visual loss due to LHON caused by a confirmed mutation in the ND4 mitochondrial gene. The agency’s decision is expected in Q4 2021.
“The treatment has been shown to be safe and the outcomes can be life changing,” said Dr. Patrick Yu-Wai-Man, MD, PhD, lead author, REVERSE principal investigator and Senior Lecturer and Honorary Consultant Ophthalmologist at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, London, United Kingdom.
“Our study provides great hope for treating this blinding disease in young adults,” said Dr. José-Alain Sahel, MD, co-corresponding author, co-founder of GenSight and Director of the Institut de la Vision (Sorbonne-Université/Inserm/CNRS), Paris, France, where LUMEVOQ®’s underlying mitochondrial targeting technology was developed. “Our approach isn’t just limited to vision restoration: other mitochondrial diseases could be treated using the same technology,” added Dr. Sahel, who is also Chairman of the Department of Ophthalmology at Centre Hospitalier National d’Ophtalmologie des XV-XX, Paris, France, and Professor and Chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA.
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