Opus Genetics announces $1.7 million in project funding from Foundation Fighting Blindness to support two preclinical programs

$1 Million TRAP Award to Support Preclinical Safety Study of Gene Therapy Vector Targeting Rhodopsin-RHO-adRP

Additional project and operating funding to support MERTK gene therapy studies enabling IND

RESEARCH TRIANGLE PARK, NC, June 13, 2024 (GLOBE NEWSWIRE) — Opus Genetics, a patient-focused, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that it has received 1 $.7 million in project funding from Foundation Fighting Blindness to help advance two preclinical program candidates.

“Opus is extremely grateful for the generous support of the Foundation Fighting Blindness, which will catalyze our efforts in developing innovative treatments for inherited retinal diseases. This $1.7 million capital infusion will have a significant impact in helping accelerate the development of these two preclinical candidates,” said Ben Yerxa, Ph.D., Chairman and CEO of Opus. “Together, we are striving toward a future where therapies to treat patients with inherited retinal diseases are readily available. »

Opus received a $1 million Translational Research Accelerator Program (TRAP) award to conduct a preclinical safety study for a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO- adRP). The safety study will be conducted on the canine animal model established at the University of Pennsylvania School of Veterinary Medicine (Penn Vet). The Company anticipates that this is the last preclinical study required before gene therapy enters clinical trials. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone.

Additionally, Opus received approximately $700,000 in project and operational funding to support the preclinical development of a novel viral vector for the treatment of retinitis pigmentosa due to mutations in the MER tyrosine-protein kinase proto-oncogene (MERTK) embarrassed. The Company is collaborating with the Foundation to initiate IND-enabling studies for a new adeno-associated virus (AAV) viral vector intended to replace mutated viruses. MERTK genes in retinal pigmented epithelial (RPE) cells of the retina. The initial funding will provide the resources needed to test the vector in an established animal model of the disease and to conduct early safety evaluations in larger animals. MERTK mutations cause rod-cone dystrophy with early macular atrophy, and retinitis pigmentosa is the most common retinal phenotype.

“We are delighted to announce our financial commitment to Opus, a pioneer in the therapeutic area of ​​inherited retinal diseases and a company founded on our mission to ultimately cure blindness caused by degenerative retinal diseases . This investment underscores our unwavering commitment to accelerating innovative solutions for those fighting inherited retinal diseases,” said Jason Menzo, CEO of the Foundation Fighting Blindness. “Together with Opus, we are advancing promising clinical candidate programs with the potential to revolutionize the lives of people affected by these challenging conditions.

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About the Fight Blindness Foundation
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for research into degenerative retinal diseases. The Foundation has raised more than $816 million for its mission to accelerate research to prevent, treat and cure blindness caused by all blinding retinal diseases, including: retinitis pigmentosa, macular degeneration and retinal syndrome. of Usher. Visit FightingBlindness.org for more information.

About Opus Genetics
Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and focus. Backed by the venture capital arm of the Foundation Fighting Blindness, the RD Fund, Opus combines unprecedented vision and commitment to patient needs with wholly owned programs in many orphan retinal diseases. Its portfolio of AAV-based gene therapies, including de-risked gene therapy ACV5 The lead program, currently in Phase 1/2 clinical trial, addresses some of the most neglected forms of hereditary blindness while creating new scale and efficiency of orphan manufacturing. Headquartered in Research Triangle Park, North Carolina, the company leverages the most advanced science and expertise of pioneers in ocular gene therapy to seamlessly deliver transformative treatments to patients. For more information, visit www.opusgenetics.com.

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