RARE Daily

Foundation for Fighting Blindness Provides Opus Genetics $1.7 Million in Funding

June 13, 2024

Rare Daily Staff

The Foundation for Fighting Blindness provided $1.7 million in project-based funding to Opus Genetics to help advance two preclinical programs to treat inherited retinal diseases.

“This $1.7 million funding infusion makes a significant impact to help accelerate the development of these two preclinical candidates,” said Ben Yerxa, CEO of Opus. “Together, we strive toward a future where therapies to help treat patients with inherited retinal diseases are readily available.”

Opus received a $1 million Translational Research Acceleration Program award to conduct a preclinical safety study for a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), one of the most common inherited retinal diseases affecting more than 6,000 people in the United States. The safety study will be conducted in the established canine animal model at the University of Pennsylvania School of Veterinary Medicine. The company anticipates this is the last preclinical study required before the gene therapy will enter clinical trials.

Additionally, Opus received approximately $700,000 in project-based and operational funding to support the preclinical development of a novel viral vector for treating retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene.

The company is collaborating with the Foundation for Fighting Blindness to begin IND-enabling studies for a newly designed adeno-associated virus (AAV) viral vector to replace mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina. The initial funding will provide the resources for testing the vector in an established animal model of the disease and to conduct early safety assessments in larger animals. MERTK mutations cause a rod-cone dystrophy with early macular atrophy, and retinitis pigmentosa is the most common retinal phenotype.

Photo: Ben Yerxa, CEO of Opus

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