By Matthew Pillar, Editor, Bioprocess Online
Originally published in Leader in life sciences magazine.
At the time, I guest-hosted Ben Yerxa, Ph.D episode 94 of the Biotech company podcast, he served as CEO of Foundation Fighting Blindness, the Retinal Degeneration Fund and Opus Genetics – three distinct but intertwined organizations with one common goal to fight blindness. However, at the end of June, he handed over his leadership duties in managing the Foundation Funding Blindness and Retinal Degeneration Fund to focus on running Opus Genetics, a biopharmaceutical company with three preclinical programs targeting gene mutations that cause severe vision loss at an early age. (i.e. Leber congenital amaurosis).
Those programs, according to Dr. Yerxa just a start. There are more than 260 genes known to cause inherited retinal disorders, and Opus’ discovery and development work leaves no stone unturned. The company’s genesis story is unique, though reproducible, and representative of a larger trend among emerging biopharmaceutical companies.
BIOTECH BOOTSTRAPPED BY AN ESTABLISHED FUNDING MECHANISM
Founded in 2021, Yerxa says Opus Genetics “was born 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 their scientific view, were ready for translation to the clinic, but were not picked up by biotech companies or funded by Main Street venture capital. “It was about the small patient populations that these therapies targeted,” explains Yerxa. “As individual assets, there was no clear path to commercial viability,” he says.
Opus Genetics was founded on the somewhat counterintuitive perspective that if these assets were developed as “stacks” of three to six candidates at a time, the path to commercialization could be cleared. It was backed from within by $19 million in seed funding from the Retinal Degeneration Fund (RDF).
The RDF has historically operated in a “venture philanthropy” style, similar to that of another venture philanthropy fund – the JDRF T1D Fund – featured in episode 80 of the Biotech company. The fund’s coffers are based primarily on donations, and they are often distributed in co-financing agreements with biopharmaceutical companies to generate interest in developing candidates that fit their cause (fighting blindness in the case of the RDF and fighting type 2 diabetes). 1 in the case of the T1D fund). “If we offer to split development costs from pre-clinical to Phase 2 trials, it creates an incentive for the biopharma to focus on an interesting candidate in the next IND filing,” explained Yerxa. “It’s a very effective way for us to use our muscle power to influence pipeline priorities for larger companies.”
But the RDF also funds biopharmaceutical companies with seed money and exerts its influence during the recipient’s formation months. These deals offer the opportunity to exert even more influence, including in board positions and participation in personnel decisions for RDF and the foundation. Funded sponsoring companies, in turn, 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 disease therapy trials, and future pipeline development opportunities through exposure to nearly 100 academic labs funded by the RDF.
It was the last model that inspired the launch of Opus Genetics from the inside out. “Opus is the result of an adjustment to the RDF charter that allowed it to take the lead in the launch of a new biopharmaceutical entity for the first time,” explained Yerxa. “It is the largest investment RDF has made, and it marks a major step for the foundation’s practical conviction in its mission.”
A MISSION TO MANIPULATE THE PRODUCTION OF GENE THERAPY
To achieve its commercial viability goals, Dr. Yerxa that Opus Genetics should have legacy development and manufacturing cost and time paradigms twisted in their ears. Changing those paradigms is central to the mission of the homemade company, and Dr. Yerxa shares great insight into how the company is tackling the challenges in episode 94 of Biotech company. Tune in Apple, Spotify, BioprocessOnline.comor wherever you listen to podcasts.
Episode 94: Fighting blindness and funding the fight with Ben Yerxa, CEO of Opus Genetics