Creating an engine for addressing inherited retinal diseases
It’s estimated that over 180,000 patients are waiting for treatments for their individual genetic conditions. Addressing this problem will require a coordinated, sustainable development and manufacturing approach.
More than 280 genes are known to cause inherited retinal diseases, which affect hundreds of thousands of patients. Almost all of these IRDs lack effective therapies to halt disease progression or rescue visual function.
Opus Genetics’ mission is to pave a proven, derisked, efficient path to the clinic for these urgently needed new therapies. Our first three programs address mutations in the LCA5, RDH12 and NMNAT1 genes that cause Leber’s congenital amaurosis. We continue to develop novel gene therapies for many of the other 280+ unaddressed retinal disease indications.