US retinal diseases focussed start-up Opus Genetics has acquired the rights to two pre-clinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio (Nasdaq: ISEE).
Opus, a spin-out company internally conceived and launched by the RD Fund to further the Foundation’s mission in August 2021, 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 USA. 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 USA alone.
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