Opus Genetics Acquires Rights to Two Experimental Gene Therapies for Inherited Retinal Diseases

Opus Genetics said it has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases from Iveric Bio for $500,000 and a “high, single digit” ownership stake in Opus.

Photo: Ben Yerxa, CEO of Opus Genetics

Opus will develop the gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP).

Under the agreement, Opus will assume responsibility for the global research, development, and commercialization of BEST1 and RHO-adRP programs. Iveric is also eligible to receive development and regulatory milestone payments, sales milestone payments, and a low single-digit earnout on net sales of the products. Iveric retains certain rights with respect to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP under certain circumstances.

BEST1-related IRDs are estimated to affect 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 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,” said Ben Yerxa, CEO of Opus. “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 expects to complete preclinical studies and file an application to begin human clinical testing for BEST1 in the second half of 2023.

Author: Rare Daily Staff