Opus Genetics Acquires Rights to Gene Therapies for BEST1 and RP (RHO)
Eye On the Cure Research News
The company plans to seek clinical trial authorization for the BEST1 gene therapy during the second half of 2023
Opus Genetics, a company developing gene therapies for inherited retinal diseases (IRDs), has acquired the rights from Iveric Bio for emerging gene therapies targeting autosomal dominant retinitis pigmentosa (RP) caused by RHO mutations and inherited retinal conditions caused by mutations in BEST1, including Best disease, a form of macular degeneration.
Opus plans to seek authorization from the U.S. Food and Drug Administration to launch a clinical trial for the BEST1 gene therapy during the second half of 2023, after preparatory preclinical studies are completed.
Best disease and other IRDs caused by BEST1 mutations affect approximately 5,000 people in the US. The BEST1 gene therapy will use a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the BEST1 gene to retinal pigment epithelial (RPE) cells, which provide support for photoreceptors, the retinal cells that make vision possible.
Autosomal dominant RP caused by RHO mutations affects about 6,000 people in the US. The RHO gene therapy, delivered to photoreceptors by an AAV, will shut down the patient’s RHO gene copies and replace them with healthy copies.
Both the BEST1 and RHO gene therapies will be delivered by a one-time subretinal injection. Researchers believe that a single gene therapy injection will last for many years, perhaps the lifetime of the patient.
Under terms of the rights acquisition, Iveric Bio received an upfront payment of $500,000 and high, single-digit percentage ownership of Opus. Iveric Bio is also eligible to receive development and regulatory milestone payments, sales milestone payments, and a portion of 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 under certain circumstances.
The RD Fund, the venture philanthropy fund of the Foundation Fighting Blindness, launched Opus Genetics with $19 million in seed funding in 2021. The Foundation also previously funded preclinical studies for the BEST1 and RHO gene therapies.
Opus is also developing gene therapies for three forms of Leber congenital amaurosis (LCA): LCA5 (lebercillin), LCA13 (RDH12), and LCA9 (NMNAT1). The company has received authorization from the U.S. Food and Drug Administration to launch a clinical trial for its LCA5 gene therapy.