Largest study to date to characterize natural history of rare retinal diseases
Research Triangle Park, N.C. – Nov. 14, 2022 – Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the company will co-sponsor the Uni-Rare Study, a new natural history study for approximately 1,500 people with mutations in one of more than 300 rare genes associated with inherited retinal diseases (IRDs).
The two-part Uni-Rare Study is being launched to characterize the course of retinal degeneration and vision loss for people with these rare genetic mutations, as well as inform the design of future clinical trials and assist in trial recruitment. The study will be conducted by the Foundation Fighting Blindness Clinical Consortium, a global, 40-site network of clinical research centers prepared to launch IRD clinical trials and natural history studies, with the Jaeb Center for Health Research to serve as the coordinating center.
“Opus is building a strong portfolio of programs focused on treating genetic mutations that cause inherited retinal diseases. The natural history data collected as part of the Foundation’s Uni-Rare Study, a first of its kind in retinal diseases, will be critical to the future research and development of new therapies for inherited retinal diseases. Opus’ second program, OPGx-002, is focused on restoring protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene, which is one of the genetic mutations that currently qualifies for the advanced portion of the Uni-Rare study,” said Jennifer Hunt, Chief Development Officer. “We’re proud to join with other leading ophthalmology organizations in supporting this important step forward to accelerate our understanding and development of new treatments for inherited retinal diseases.”
The Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases. For more information on the Uni-Rare Study, please read the Foundation’s press release or visit www.clinicaltrials.gov.
About Opus Genetics
Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’s venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com.