$1 million TRAP award to support preclinical safety study of gene therapy vector targeting rhodopsin-RHO-adRP
Project and additional operational funding to support MERTK Gene therapy IND-enabling studies
RESEARCH TRIANGLE PARK, NC, June 13, 2024 (GLOBE NEWSWIRE) — Opus Genetics, a first-in-patient clinical phase gene therapy company developing treatments for inherited retinal diseases, announced today that it has received $1.7 million in funding based project from the Foundation to Fight Blindness to help advance two preclinical candidate programs.
“Opus is extremely grateful for the generous support from the Foundation to Fight Blindness, which will catalyze our efforts in pioneering treatments for inherited retinal diseases. This $1.7 million funding infusion has a significant impact in helping to accelerate the development of these two preclinical candidates,” said Ben Yerxa, Ph.D., Chief Executive Officer 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 (TRAP) award to conduct a preclinical safety study of a gene therapy vector designed to target rhodopsin-mediated autosomal dominant autosomal dominant retinitis pigmentosa (RHO- adRP). The safety study will be conducted in 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 the 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.
In addition, Opus received approximately $700,000 in project-based and operational funding to support the preclinical development of a novel viral vector for the treatment of retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene. The company is collaborating with the Foundation to initiate IND-enabling studies for a newly developed adeno-associated virus (AAV) viral vector to replace mutations MERTK genes in retinal pigmented epithelium (RPE) cells of the retina. The initial funding will provide the resources to test 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.
“We are excited to announce our funding commitment to Opus, a pioneer in the therapy of inherited retinal diseases and a company built 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, executive director of the Foundation to Fight Blindness. “In collaboration with Opus, we are advancing promising clinical candidate programs with the potential to revolutionize the lives of those affected by these challenging conditions.”
About the Foundation to fight blindness
Founded in 1971, the Foundation to Fight Blindness is the world’s leading private source of funding for research into degenerative retinal diseases. The foundation has raised more than $816 million toward its mission of accelerating research to prevent, treat and cure blindness caused by the full spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration and Usher syndrome. Visit FightingBlindness.org for more information.
About Opus Genetics
Opus Genetics is a clinical-stage genetic therapy company for inherited retinal diseases with a unique model and purpose. Backed by the Fighting Blindness Foundation’s venture arm, the RD Fund, Opus combines unparalleled knowledge and dedication to patient needs with fully owned programs in multiple orphan retinal diseases. Its AAV-based gene therapy portfolio, including a mock LCA5 the flagship program currently in a Phase 1/2 clinical trial, addresses some of the most neglected forms of inherited blindness while creating new scale and efficiency of orphan production. Based in Research Triangle Park, NC, the company leverages the insights of the best science and the expertise of pioneers in ocular gene therapy to transparently direct transformative treatments for patients. For more information, visit www.opusgenetics.com.
Media contact:
Gina Mangiaracina
6 degrees
gmangiaracina@6degreesPR.com
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