18 Jul Opus Genetics
George Magrath, M.D., CEO
Oct. 6 | 9:15am | FLW Ballroom G
Durham, NC
(NASDAQ: IRD)
Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing important new therapies that target mutations in genes causing loss of vision and Inherited Retinal Diseases (IRDs) and other retinal and refractive disorders. Our portfolio spans approved drugs, Ph III programs, as well as a deep gene therapy pipeline in bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) to aim to solve inherited blinding conditions. Our current clinical focus is on Leber Congenital Amaurosis (LCA) type 5, a type of LCA that deals with the LCA5 gene which encodes lebercilin, a protein involved in centrosomal or ciliary functions. This program recently received a RMAT designation from FDA. Our bestrophinopathy program is kicking off in 2025 in the US, with other assets in RDH12 building a path towards IND later this year. It’s estimated that over 300,000 patients are waiting for treatments for their individual genetic conditions, and more than 280 genes are known to cause inherited retinal diseases. Opus’ mission is to pave a proven, derisked, efficient path to the clinic for these urgently needed new therapies. Our IRD programs address mutations in genes that cause ophthalmic disease based on world-class science from gene therapy pioneers at the University of Pennsylvania (including the lab of Jean Bennett, MD, PhD), Harvard Medical School, and the University of Florida.
