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Opus Enters Strategic Collaboration with Resilience for AAV-based Gene Therapy for Inherited Retinal Diseases

April 12, 2022

Gene therapy developer Opus Genetics said it entered a strategic manufacturing services agreement with biomanufacturer National Resilience to support the development and manufacturing of the Opus pipeline.

Photo: Ben Yerxa, acting CEO of 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’ adeno-associated viral vector-based gene therapies for inherited retinal diseases for use in both preclinical studies and future clinical trials in the United States.

The work will be conducted at Resilience’s facilities in Waltham, Mass. and Research Triangle Park, North Carolina.

“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,” said Ben Yerxa, CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, and acting CEO of Opus.

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 haltfunctional 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.

Author: Rare Daily Staff

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