LHON Gene Therapy GS010 Study Data to be Presented at NANOS Meeting

GenSight Biologics will present data from studies of its gene therapy GS010 for Leber hereditary optic neuropathy (LHON), a mitochondrial disease, at an upcoming Neuro-Ophthalmology Society conference.

According to a press release, the one oral and two poster presentations will be given at the 2017 North American Neuro-Ophthalmology Society‘s (NANOS) 43rd annual meeting in Washington, D.C., April 1-6.

LHON is rare, maternally inherited mitochondrial genetic disease that causes irreversible and severe loss of sight, leading to blindness and disability in teens and young adults. The disease is caused by defects in mitochondrial genes that encode proteins called NADH dehydrogenase.

GS010 is a proprietary gene-replacement therapy based on a mitochondrial targeting sequence (MTS). GS010 uses an adeno-associated virus (AAV) to deliver a functional gene to the mitochondria that can override the mutation. When the nonmutant gene reaches the inside of the cell, it produces a protein that restores mitochondrial function. Mitochondria are components of cells responsible for energy production.

Catherine Vignal, MD, principal investigator of the study and chief of the Department of Ophthalmology at the Rothschild Foundation Hospital in Paris, France, will present the results from a 78-week follow-up of an ongoing Phase 1/2 trial (NCT02064569), which is evaluating the safety and tolerability profile of increased doses of GS010 in LHON patients. In a December 2016 GenSight press release, the follow-up results confirmed the favorable safety and tolerability profile of GS010, while demonstrating a sustained improvement in patients’ visual acuity.

The oral presentation is titled “Phase I/IIa Visual Acuity Outcomes 1.5-Years Post-Treatment with rAAV2/2-ND4, Investigational Gene Therapy for ND4 LHON.”

GenSight also will present new data from its Phase 3 REVERSE (NCT02652780) and RESCUE (NCT02652767) trials at NANOS. Researchers of both studies are evaluating the effectiveness of a single intravitreal injection of GS010 in LOHN patients who have a G11778A mutation of their mitochondrial ND4 gene.

Patrick Yu-Wai-Man, MD, PhD, principal investigator of the REVERSE study and Honorary Consultant in Ophthalmology at the Moorfields Eye Hospital in London (and key researcher at the Newcastle Eye Centre) will present initial results of the trials. The poster presentation is titled “Preliminary Baseline Characteristics of Patients with LHON Enrolled in RESCUE and REVERSE Gene Therapy Trials.”

The second poster presentation, “Pediatric Leber Hereditary Optic Neuropathy (LHON): A Literature Review,” will provide an overview of published studies in pediatric LHON.  

GS010 has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) in the European Union.