Opus Genetics announces agreement with Massachusetts Eye


RALEIGH, NC, Nov. 11, 2021 (GLOBE NEWSWIRE) – Opus Genetics, a patient-centered gene therapy company that develops treatments for inherited orphan retinal diseases, today announced an agreement to license its third preclinical program to treat mutations in the NMNAT1 gene, which cause a specific form of Leber’s congenital amaurosis (ACL), from the Massachusetts Eye and Ear, the main teaching hospital of Harvard Ophthalmology.

The new program, OPGx-003, is based on the work of Eric Pierce, MD, Ph.D., director of the Ocular Genomics Institute and William F. Chatlos professor of ophthalmology at the Massachusetts Eye and Ear and the Harvard Medical School, and co-founder of Opus. OPGx-003 is a gene augmentation therapy designed to arrest functional deterioration in pediatric patients with degenerative retinal disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene. Recent preclinical data have demonstrated the potential of this gene augmentation approach to achieve stable rescue of retinal structure and function. Opus plans to file an IND for OPGx-003 in the first half of 2023.

“We are delighted to collaborate with Dr. Pierce, who will bring his expertise in retinal gene therapy to the search for a novel treatment for retinal degeneration associated with NMNAT1,” said Ash Jayagopal, Ph.D., Scientific Director of NMNAT1 Opus Genetics. “The addition of OPGx-003 to the Opus pipeline further underscores our commitment to bringing together the resources and expertise necessary to take promising science from the laboratory to the clinic and ultimately to the patients who need it, and strengthens the importance of our innovative patient-centric model. . “

“I saw with my own eyes the need for new treatments for rare inherited retinal diseases,” said Dr. Pierce. “As someone who has dedicated my life to researching this space, I firmly believe that Opus is an ideal company to advance this work and make a difference for patients in need.”

Opus’ main program, OPGx-001, is designed to process mutations in the LCA5 gene, which encodes the lebercilin protein. The company’s second program, OPGx-002, will focus on restoring protein expression and stopping functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene. (LCA13).

About Opus Genetics
Opus Genetics is a revolutionary gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by the venture capital arm of the Foundation Fighting Blindness, the RD Fund, Opus combines unparalleled insight and commitment to the needs of patients with wholly owned programs in many orphan retinal diseases. Its portfolio of AAV-based gene therapy tackles some of the most neglected forms of inherited blindness while creating a new scale and efficiency of orphan manufacturing. Based in Raleigh, North Carolina, the company leverages the insights of top scientists and the expertise of pioneering ocular gene therapy to seamlessly drive transformative treatments for patients. For more information, visit www.opusgenetics.com.

Dr Pierce owns shares and is a consultant for Opus Genetics.

Media contact:
Heather anderson
[email protected]


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