Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio. Opus will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), respectively.
BEST1-related IRDs are estimated to affect approximately one in 69,000 people, or nearly 5,000 people, in the United States. The BEST1 gene therapy is designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells to produce bestrophin-1 protein and normalize homeostasis between the photoreceptors and retinal pigment epithelial cells. 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.
