Opus Genetics Inc., a private ocular gene therapy company based in Raleigh, North Carolina, has announced a strategic manufacturing services agreement with National Resilience, Inc. (Resilience), aimed to support the development and manufacturing of gene therapy opportunities. Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, encoding the lebercilin protein, which causes one of the most severe forms of LCA, and affects approximately one in 1.7 million people. A second program, OPGx-002, will focus on restoring protein expression and halting functional deterioration in patients with retinal dystrophy, caused by mutations in the retinal dehydrogenase (RDH12) gene (LCA13), affecting one in 288,000 people.
Opus Genetics is supported by the Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases. The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). According to the company, “AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. The company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients.”
According to the announcement from Opus, Resilience will provide process and analytical development, quality control testing, and GMP manufacturing services for IND-enabling toxicology and first-in-human material for Opus’ AAV vector-based gene therapies for inherited retinal diseases (IRD) for use in both preclinical studies, and future clinical trials in the U.S. The work will be conducted at Resilience’s facilities in Waltham, Mass., and Research Triangle Park, N.C. Commenting on the announcement, Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus stated that, “Resilience embraces our innovative model to create a clinical manufacturing infrastructure that’s scaled to address rare inherited retinal diseases, and we are pleased to enter into this strategic collaboration. Leveraging Resilience’s expertise puts Opus in the best position to efficiently advance our AAV-based gene therapies into the clinic and toward the patients who need them.”