Opus Genetics Secures $1.7 Million Funding for Two Preclinical Programs

Opus Genetics, a pioneering gene therapy company focused on inherited retinal diseases, has secured $1.7 million in project-based funding from the Foundation Fighting Blindness. This financial support aims to accelerate the development of two preclinical candidate programs critical for advancing potential treatments.

"Opus is immensely grateful for the generous support from the Foundation Fighting Blindness, which will catalyze our efforts in pioneering treatments for inherited retinal diseases. This $1.7 million funding infusion makes a significant impact to help accelerate the development of these two preclinical candidates," stated Ben Yerxa, PhD, Chief Executive Officer of Opus.

TRAP Award for RHO-adRP Gene Therapy

The funding includes a $1 million Translational Research Acceleration Program (TRAP) award dedicated to conducting a pivotal preclinical safety study. This study will evaluate a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP) using an established canine model at the University of Pennsylvania School of Veterinary Medicine (Penn Vet). Opus anticipates this study will be the final preclinical step before advancing to clinical trials. RHO-adRP affects approximately one in 51,000 individuals in the United States alone.

Novel Viral Vector for MERTK-Associated Retinitis Pigmentosa

In addition, Opus received approximately $700,000 in project-based and operational funding to support the development of a novel viral vector addressing retinitis pigmentosa caused by mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene. Collaborating with the Foundation, Opus plans to initiate IND-enabling studies for a newly designed adeno-associated virus (AAV) viral vector aimed at replacing mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina.

This initial funding will facilitate testing in an established animal model and early safety assessments in larger animals. MERTK mutations are associated with a rod-cone dystrophy leading to early macular atrophy, with retinitis pigmentosa being the most common retinal phenotype.

Foundation Fighting Blindness' Commitment to Advancing Therapies

Jason Menzo, CEO of the Foundation Fighting Blindness, emphasized the organization's commitment to advancing therapies for inherited retinal diseases: "We're excited to announce our funding commitment to Opus, a trailblazer in inherited retinal disease therapeutics and a company created based on our mission of ultimately curing blindness caused by retinal degenerative diseases. This investment highlights our steadfast dedication to hastening innovative solutions for those combating inherited retinal diseases. In collaboration with Opus, we're propelling forward promising clinical candidate programs with the potential to revolutionize the lives of those affected by these challenging conditions."

Opus Genetics continues to pursue these programs with the aim of developing transformative treatments for patients affected by inherited retinal diseases, leveraging the support and collaboration of the Foundation Fighting Blindness to achieve significant milestones in gene therapy research.