OPGx-NMNAT1

OPGx-NMNAT1 (formerly designated OPGx-003) is an adeno-associated virus (AAV)-based gene augmentation therapy being developed by Opus Genetics for the treatment of retinal degeneration caused by mutations in the NMNAT1 gene, which results in a specific form of Leber congenital amaurosis (LCA). The therapy is designed to deliver a functional copy of the NMNAT1 gene to retinal cells to halt disease progression. NMNAT1 encodes an enzyme essential for the regeneration of nicotinamide adenine dinucleotide (NAD+), a critical metabolic cofactor required for the survival and function of retinal cells. The program was licensed from Massachusetts Eye and Ear and Harvard Medical School, based on the research of Dr. Eric Pierce, and is currently in preclinical development.