Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, announced a strategic manufacturing services agreement with National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, to support the development and manufacturing of Opus’ pipeline.
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’ adeno-associated viral (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.
“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,” said Ben Yerxa, Ph.D., CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus. “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.”
“Helping Opus advance their gene therapies for inherited retinal diseases to the clinic will hopefully lead to new treatments for underserved patients,” said Rahul Singhvi, ScD, Chief Executive Officer of Resilience. “We’re excited to partner with Opus on process development and manufacturing at this pivotal time for the company.”
Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. Opus anticipates filing an IND and entering first-in-human trials with OPGx-001 this year. Opus’ earlier stage programs include OPGx-002, to restore protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene, and OPGx-003, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.