Opus Genetics, a gene therapy company developing treatments for orphan inherited retinal diseases, announced an agreement to license its third preclinical program to address mutations in the NMNAT1 gene, which cause a specific form of Leber congenital amaurosis (LCA), from Massachusetts Eye and Ear.
The new program, OPGx-003, is based on the work of Eric Pierce, MD, PhD, Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, as well as a scientific co-founder of Opus.
OPGx-003 is a gene treatment that aims to prevent functional decline in children with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.
Preclinical studies have shown that this gene augmentation strategy has the ability to restore retinal structure and function in a stable manner. In the first half of 2023, Opus plans to file an IND for OPGx-003.
“We’re thrilled to collaborate with Dr. Pierce, who will bring his expertise in retinal gene therapy to researching a novel treatment of NMNAT1-associated retinal degeneration,” Ash Jayagopal, PhD, Chief Scientific Officer of Opus Genetics, said in a company news release.
“Adding OPGx-003 to the Opus pipeline further underscores our commitment to bringing the required resources and expertise together to take promising science from the lab through the clinic and ultimately to patients who need it, and reinforces the importance of our innovative patient-focused model.”
“I’ve seen firsthand the need for new treatments for rare inherited retinal diseases,” said Dr. Pierce. “As someone who has devoted my life to research in this space, I believe strongly that Opus is an ideal company to advance this work to make a difference for patients in need.”
The primary Opus program, OPGx-001, is aimed at mutations in the LCA5 gene, which codes for the lebercilin protein.
OPGx-002 will focus on restoring protein expression and slowing functional degeneration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13).