At the time I hosted Ben Yerxa, Ph.D., as a guest on episode 94 of the Business of Biotech podcast, he was serving as CEO of Foundation Fighting Blindness, the Retinal Degeneration Fund, and Opus Genetics — three distinct but interwoven organizations with one common goal fighting blindness. In late June, though, he handed off his leadership duties at Foundation Funding Blindness and Retinal Degeneration Fund management to focus on leading Opus Genetics, a biopharma with three preclinical programs addressing gene mutations that cause severe vision loss at an early age (i.e., Leber congenital amaurosis).
Those programs, according to Dr. Yerxa, are just a start. There are more than 260 genes known to cause inherited retinal diseases, and Opus’ discovery and development work is leaving none of those stones unturned. The company’s genesis story is unique, though replicable and representative of a greater trend among emerging biopharmaceutical companies.
BIOTECH BOOTSTRAPPED BY AN ESTABLISHED FUNDING MECHANISM
Founded in 2021, Yerxa says Opus Genetics “evolved out of three years of observation and a little bit of frustration.” The Foundation Fighting Blindness had recognized a number of academia-backed gene therapies that, in its scientific purview, were ready for translation into the clinic but which weren’t being picked up by biotechs or financed by Main Street venture capital. “At issue was the small patient populations these therapies addressed,” explains Yerxa. “As individual assets, there wasn’t a clear path to commercial viability,” he says.
Opus Genetics was formed on the somewhat contrarian perspective that if these assets were developed as “stacks” of three to six candidates at a time, the path to commercialization might be cleared. It was backed from within by $19 million in seed funding from the Retinal Degeneration Fund (RDF).
The RDF had historically operated in a “venture philanthropy” style not unlike that of another venture philanthropy fund — the JDRF T1D Fund — featured on episode 80 of the Business of Biotech. Those fund’s coffers are primarily donation-based, and they’re often distributed in co-funding deals with biopharma companies to drive interest in the development of candidates that fi t their cause (fighting blindness in the case of the RDF and fighting type 1 diabetes in the case of the T1D Fund). “If we offer to split development costs from preclinical to Phase 2 trials, it creates an incentive for the biopharma to focus on a candidate of interest as its next IND filing,” explains Yerxa. “It’s a very effective way for us to use our muscle to influence priorities in bigger companies’ pipelines.”
But the RDF also funds biopharmas with initial startup equity, exerting its influence during the recipient’s formative months. Those deals offer an opportunity to exert even more influence, including board positions and a hand in personnel decisions for RDF and the foundation. In turn, funded sponsor companies gain access to resources including the foundation’s patient registry for clinical trials enrollment, its Clinical Consortium, an infrastructure of dozens of global clinical sites outfitted for retinal disease therapy trials, and future pipeline development opportunities via exposure to nearly 100 academic labs funded by the RDF.
It was the latter model that inspired the launch of Opus Genetics from within. “Opus is the result of a tweak to the RDF charter, which allowed it to, for the first time, take the lead on the launch of a new biopharma entity,” explains Yerxa. “It’s the largest investment RDF has made, and it marks a big step for the foundation’s hands-on conviction to its mission.”
A MISSION TO MANIPULATE GENE THERAPY MANUFACTURING
To achieve its commercial viability goals, Dr. Yerxa says Opus Genetics must turn legacy development and manufacturing cost and time paradigms on their ears. Changing those paradigms is central to the mission of the self-made company, and Dr. Yerxa shares plenty of insight into just how the company is tackling the challenges on episode 94 of Business of Biotech. Tune in on Apple, Spotify, BioprocessOnline.com, or anywhere you listen to podcasts.