By Matthew Pillar, Editor of Bioprocess Online
Originally posted in Head of life sciences magazine.
At the time I hosted Ben Yerxa, Ph.D., as a guest on episode 94 of the Business of Biotech podcast, he was CEO of Foundation Fighting Blindness, Retinal Degeneration Fund, and Opus Genetics, three distinct but interconnected organizations with a common goal fight blindness. In late June, however, he left his leadership positions at the management of the Foundation Funding Blindness and Retinal Degeneration Fund to focus on leading Opus Genetics, a biopharmaceutical with three preclinical programs addressing the genetic mutations that cause severe loss of vision. seen at an early age (eg, Leber congenital amaurosis).
Those programs, according to Dr. Yerxa, are just the beginning. There are more than 260 genes known to cause inherited retinal diseases, and Opus’s discovery and development work leaves no stone unturned. The story of the company’s genesis is unique, yet replicable and representative of a major trend among emerging biopharmaceutical companies.
BIOTECH BOOTSTRAPPED BY AN ESTABLISHED FINANCING MECHANISM
Founded in 2021, Yerxa says Opus Genetics “evolved after three years of observation and a little bit of frustration.” The Foundation Fighting Blindness had recognized a number of academically backed gene therapies that were scientifically ready to be translated into the clinic but were not being picked up by biotech or funded by Main Street venture capital. “The problem was the small patient populations these therapies address,” Yerxa explains. “As individual assets, there was no clear path to commercial viability,” she says.
Opus Genetics was formed based on a somewhat countercurrent perspective that if these assets were developed as “stacks” of three to six candidates at a time, the path to commercialization could be opened. It was supported from within by $19 million in seed funding from the Retinal Degeneration Fund (RDF).
The RDF had historically operated in a style of “venture philanthropy” not unlike that of another venture philanthropy fund – the JDRF T1D Fund – featured in episode 80 of the The business of biotechnology. The coffers of these funds are primarily based on donations and are often distributed in co-financing agreements with biopharmaceutical companies to stimulate interest in developing candidates suitable for their cause (fighting blindness in the case of 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 studies, that creates an incentive for the biopharmaceutical to focus on a candidate of interest as their next IND filing,” Yerxa explains. “It’s a very effective way for us to use our muscle to influence priorities in the pipelines of the largest companies.”
But RDF also funds biopharma with initial startup equity, exerting its influence during the beneficiary’s formative months. Such arrangements provide the opportunity for even greater influence, including positions on the board of directors and a hand in personnel decisions for RDF and the foundation. In turn, funded corporate sponsors gain access to resources including the foundation’s patient registry for clinical trial enrollment, its clinical consortium, an infrastructure of dozens of global clinical sites equipped for retinal diseases and future pipeline development opportunities through exposure to nearly 100 RDF-funded academic laboratories.
It was this latter pattern that inspired the launch of Opus Genetics from within. “Opus is the result of a change to the RDF charter, which allowed it, for the first time, to take the lead in the launch of a new biopharmaceutical entity,” explains Yerxa. “It’s the largest investment RDF has made and marks a big step for the foundation’s practical belief in its mission.”
A MISSION TO MANIPULATE THE PRODUCTION OF GENE THERAPY
To meet its commercial viability goals, Dr. Yerxa says Opus Genetics must upend legacy development, cost and manufacturing time paradigms. Shifting these paradigms is central to the company’s self-made mission, and Dr. Yerxa shares a lot of insight into how the company is addressing the challenges in episode 94 of The business of biotechnology. Tune in to Apple, Spotify, BioprocessOnline.com or wherever you listen to podcasts.
The business of biotechnology
Episode 94: Fighting blindness and funding the fight with Ben Yerxa, CEO of Opus Genetics