The startup, which is headquartered at Research Triangle Park, North Carolina, will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related IRDs and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), assuming responsibility for the global research, development, and commercialization of both candidates.
In exchange, Iveric received an upfront payment of $500,000 and an ownership stake in Opus.
That New Jersey based company 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 and it retains certain rights with respect to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP under certain circumstances.
Opus said the BEST1 and RHO-adRP programs complement its existing pipeline of gene therapies for IRDs and that it intends to file an Investigational New Drug (IND) for the BEST1 program later in 2023. That 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.
“Affecting over 28,000 people across the US, EU, and UK, BEST1 and RHO-adRP represent a significant portion of all inherited retinal diseases and an urgent unmet need for effective treatment. IRDs are ideal targets for genetic therapies to stop the retinal degeneration and improve the lives of patients living with severe vision loss or blindness,” said Bart P Leroy, head of the Department of Ophthalmology, professor of Ophthalmology and member of the Center for Medical Genetics at Ghent University and Ghent University Hospital in Belgium.
LCA5 program
Opus was formed in September 2021, with a US$19m seed financing round led by the venture capital arm of the Foundation Fighting Blindness, the RD Fund, with participation also from the Manning Family Foundation and Bios Partners.
At the start of December last year, the US Food and Drug Administration (FDA) cleared its IND application for a Phase 1/2, first-in-human clinical trial of its therapy, OPGx-001, in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).
OPGx-001 is an AAV8 vector aimed at delivering a functional LCA5 gene to retinal photoreceptors. Currently, there are no approved treatments for individuals with LCA5-related vision loss.
