Opus, Resilience Team Up to Develop Gene Therapies for Inherited Retinal Diseases

Opus, Resilience Team Up to Develop Gene Therapies for Inherited Retinal Diseases

April 14, 2022

Opus Genetics has signed a strategic manufacturing services partnership with National Resilience, a technology-focused biomanufacturing firm dedicated to expanding access to complex medicines, to help with 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 take place at Resilience's locations in Waltham, Massachusetts, and Research Triangle Park, North Carolina.

“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,” Ben Yerxa, PhD, CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus, said in a company news release.

“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.