It’s only been a handful of years since the FDA issued its landmark first approval of an adeno-associated virus gene therapy. But up-and-comers in biotech have already been looking to improve upon the first wave of gene therapies and overcome its limitations with next-gen tech.
Among those generating buzz is Switzerland-based Anjarium Biosciences, who crowned Stephen Yoo as its new CEO in June. Drawing on his medical training and extensive background in helping to build growth stage biotech companies, including others in the gene therapy space, Yoo is now laser-focused on unlocking the full potential of gene therapies with the company’s non-viral delivery platform that offers several key advantages over existing tech.
Traditionally, the use of viral vectors in gene therapies has been beset by challenges associated with payload capacity, immunogenicity, targeted delivery and scalable manufacturing. Yet, Anjarium’s platform was developed to overcome these hurdles by using proprietary DNA-based gene vectors, and natural and synthetic nanoparticle delivery modalities. By combining genetic medicine, synthetic biology and nanoparticle engineering, the company says it can more effectively and durably address the underlying causes of many genetic diseases.
The prospect of Anjarium’s success excited investors enough that it raised $61 million in series A financing last year — a thick purse that Yoo is now looking to leverage wisely as the company works to expand its platform and advance its preclinical pipeline.
Before joining Anjarium, Yoo was the chief medical officer and head of R&D for Third Harmonic Bio, a clinical-stage company focused on severe allergy and inflammation. He also spent four years at Regenxbio working on recombinant adeno‐associated virus (AAV) gene therapy, as well as many years at larger biopharma companies including AstraZeneca and MedImmune.
PharmaVoice spoke to Yoo as he began establishing early objectives and getting to know the team, which he says is one of his top priorities.
“We're only going to go as far as our talent takes us, so it’s important to build a positive and empowering culture,” he says. “It all starts with communication and transparency and setting the compass for the kind of culture we want as the company grows.”
Here, Yoo shares his goals and vision for the next stage of Anjarium’s growth, what he brings to the role of CEO and the challenges that lie ahead.
PharmaVoice: What do you believe makes you a good fit to lead Anjarium?
Stephen Yoo: There are a lot of synergies from my last two roles. I’m leaning on a lot of my previous experiences of successfully building those two companies, and I believe that fits what Anjarium needs. Most recently at Third Harmonic, I was the second employee and helped to build the company from the ground up. And at Regenxbio, I was one of the first 10 employees and today that company is a leader in the AAV gene therapy space. When I started at Regenxbio, prior experience in gene therapy wasn't a prerequisite because no one had it, but now there’s so much going on in the space, so I think having the experience of working with this unique modality of therapeutics helps in terms of strategic focus and understanding where to take the company.
"It’s a yin and yang of fostering innovation while maintaining the required processes to help build a strong foundation."
CEO, Anjarium Biosciences
At both those companies, the focus was on developing preclinical or research-stage assets and transitioning them into the clinic to show whether or not they could become effective medicines. Again, at both we received approval to start clinical trials, and were able to generate positive clinical data. I also have a lot of experience in taking products from non-clinical through all stages of clinical development, and that helps us to set the compass of how to effectively use our platform to create therapeutics. It’s about building the plane as it flies.
In what direction are you looking to take Anjarium, and what steps are you taking to achieve those goals?
With Anjarium’s gene therapy platform we’re aiming to leverage gene therapy in a more predictable, effective and lasting way, for a range of genetic diseases. We have the luxury of having multiple platforms under one roof: our proprietary DNA-based gene vectors, natural and synthetic nanoparticle delivery modalities — including our novel Hybridosome delivery technology —and our manufacturing solutions.
We want to address the limitations of current gene therapy platforms and tackle more diseases more precisely. Ultimately, we’re in the drug-making business and we need to make therapeutics for patients, so we need to figure out how we’re going to take advantage of this incredible biology that we have in all our different platforms and translate that into effective and compelling medicines for patients.
Innovative, agile thinking is obviously important at a company like Anjarium. But how does your experience help you take that innovation and ensure it achieves desired outcomes?
Innovative people tend to join companies like Anjarium because they enjoy the reduced bureaucracy, the quick decision making and the ability to be in the middle of the action. At the same time, you need the proper amount of intention to help the company build the necessary infrastructure to support effective decision-making without stifling that innovative spirit. It’s a yin and yang of fostering innovation while maintaining the required processes to help build a strong foundation. It can be a tricky balance and the center changes as the company grows. Having lived through this type of growth twice helps me manage that at Anjarium.
What do you regard as the most important skill set for a leader to keep teams focused, motivated and engaged?
I’ve always focused on empowering people to do their jobs, and that starts with establishing a positive culture. Really, the two most important assets that a company has are its time and its people. How do we hire the best people and enable them to do their jobs? And then, we really have to have a sense of urgency, because patients are waiting for us to unlock this potential.
When you're a young company, maintaining alignment in the middle of the chaos that goes with growth is really important. So, we have regular conversations with everyone in the company on what's important to us. How do we want to operate? How do we put this into practice? How do we hold people accountable to our values? Transparency is essential to building a positive culture. Everyone has to know the why, not just the what or the how so everyone believes in the mission, and that tends to be very empowering.
What hurdles does Anjarium face in its quest to achieve breakthroughs in tackling genetic disease?
With the platform we have, there is a feeling that we can do anything. But the key to managing growth is developing focus. We have to balance developing a robust understanding of the capabilities of our platforms, while also defining our strategy, and how we're going to develop new medicines that will make it to patients. There will be initial drugs we develop based on the current status of our platform, but as the science continues to evolve, and we continue to expand the capabilities of our platforms, we're going to have the privilege of taking on more disease biology as we continue to expand those capabilities. It’s a balance of developing the platform to open up future opportunities and focusing on the here and now.
We’re exploring new frontiers with the technology at Anjarium. It's exciting to be at the forefront of science, to help grow the organization and to help develop and advance its pipeline strategy. Ultimately, I believe this technology will translate into really meaningful medicines for patients in the future.