Editor’s note: This story is part of our 2022 PharmaVoice 100 feature.
Last June, Opus Genetics announced that it had appointed Ben Yerxa as its permanent CEO. It wasn’t a surprising announcement.
Yerxa was, in fact, a driving force behind the launch of Opus, an AAV-based gene therapy company specializing in retinal diseases, and already its acting CEO. But his roots in the company can be traced back further to when he served as CEO of the Foundation Fighting Blindness and then launched its spin-off venture arm, Retinal Degeneration Fund (RD Fund), which has backed several ocular therapy startups — including Opus.
The Foundation had already been hard at work funneling money into research for inherited retinal diseases, including more than 80 grants and the work that led to the FDA’s first-ever approval of a gene therapy: Spark Therapeutics’ Luxturna, a paradigm-changing treatment for patients with a rare and inherited form of blindness.
Yet, Yerxa saw more opportunities to develop leading-edge treatments in the space.
“There are about 40-50 clinical trials underway for sight-saving therapies, but more research and ideas emerge daily that offer strong clinical potential,” Yerxa’s nominator writes.
With the goal of creating a novel venture philanthropy-backed biotech company, Yerxa launched Opus, which is supported by the Foundation and RD Fund.
“The first-of-its-kind patient funded/backed biotech leverages the expertise of pioneers in ocular gene therapy to advance a wholly owned portfolio of therapies to treat underserved forms of inherited blindness with an urgent patient-first priority,” a nominator says.
“I've always had a general fascination with the blurry line between biology and chemistry mixed together with the desire to help people.”
Ben Yerxa
CEO, co-founder, Opus Genetics
To date, Opus has built a pipeline of three AAV-based gene therapy preclinical assets targeting inherited forms of blindness that have otherwise been neglected by the industry. The company plans to launch clinical trials for its lead asset this year, with studies anticipated for its other two candidates in 2023 and 2024.
“By bringing together scientific leaders, industry resources, a patient advisory board and the backing of the Foundation and RD Fund, Yerxa sees Opus as a new kind of company built for the long term, creating novel manufacturing scale and efficiencies to address as many of the spectrum of 260+ related orphan inherited retinal diseases possible,” a nominator says.
This novel approach is the culmination of Yerxa’s long history in pharma. Yerxa previously served as the president and co-founder of Envisia Therapeutics, and held other executive roles at Liquidia Technologies, Clearside, Inspire Pharmaceuticals and more. According to his nominator, Yerxa has had more than 50 patents issued in the U.S. and was instrumental in launching two ophthalmic products.
“It’s rare that a single person’s impact on the therapeutic landscape in biopharma is quantifiable or tangible, but [Yerxa] is that rare case,” his nominator says.
Here, Yerxa describes the initial spark that led him to the life sciences and how he tries not to take himself too seriously.
PharmaVoice: What is the most meaningful item in your office?
Ben Yerxa: It's a small piece of art that has a still from the movie ‘Blood Diamond’ showing Leonardo DiCaprio running away from clear danger, but the danger is actually C-3PO chasing and fussing at him. I might be the only one who thinks it's funny. It reminds me not to take myself so seriously.
What initially drew you into the life sciences?
I've always had a general fascination with the blurry line between biology and chemistry mixed together with the desire to help people, but the initial hook was studying marine biology in high school and making ‘potions’ in the backyard as a kid.
What keeps you up at night?
The need for more funding for the gene therapies we are developing. The science is there, it's ready, but it needs the human and financial resources to make the therapies a reality.
How would you describe your leadership style?
I've had it described to me recently as ‘quiet confidence’ and that seems to fit as my brand.
What is your key piece of leadership advice?
It's hard for me to give general leadership advice, because to me it is always personal and situation dependent. However, at this moment when the world seems turned upside down, I think it is key for leaders to really pay attention to how the external environment affects their business and strategy. By this, I mean that one has to master all the different kinds of forces that are at play, whether that's finance, government, pandemic, supply chain, work from home, etc.