Opus Genetics Acquires Rights to Two Gene Therapy Product Candidates from Iveric Bio

Opus Genetics Acquires Rights to Two Gene Therapy Product Candidates from Iveric Bio

December 29, 2022
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Opus Genetics has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio. Opus will explore novel gene therapy options in order to treat rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP) and bestrophin-1 (BEST1)-related inherited retinal diseases, respectively.

Nearly 5,000 Americans, or one in 69,000 people, are believed to be affected by inherited retinal diseases linked to BEST1. In order to manufacture bestrophin-1 protein and restore the balance between photoreceptors and retinal pigment epithelial cells, the BEST1 gene therapy is designed to deliver a functioning copy of the BEST1 gene to 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.

“The addition of these innovative BEST1 and RHO-adRP programs significantly increases the patient population that could benefit from Opus therapies and complements our existing pipeline of gene therapies for inherited retinal diseases,” Ben Yerxa, PhD, Chief Executive Officer of Opus, said in a company news release. “Opus is building an engine for addressing multiple IRDs, and deals like this one help us achieve our desire for sustainable growth of our infrastructure, operations, scientific expertise and ultimately our patient impact, with the goal of at least one IND per year.”

Opus plans to finish more IND-enabling studies and submit an IND for BEST1 in the second half of 2023.

As part of the agreement, Opus will be in charge of the BEST1 and RHO-adRP programs' global research, development, and commercialization. Iveric received a $500,000 upfront payment and a high single-digit ownership stake in Opus in return. Additionally, Iveric is qualified for sales milestone payments, development and regulatory payments, and a low single-digit earnout on the products' net sales. Under certain circumstances, Iveric reserves certain rights with regard to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP.