Gene therapy has enormous potential to treat and cure some of humankind’s most devastating diseases. However, current methods of delivering these therapies face deep challenges. Some methods cause an immune reaction that renders future therapy deliveries ineffective, while other methods are so complicated and expensive that they put transformative treatment out of reach for most patients. STRM.BIO, a venture founded in 2019, aims to change this by developing extracellular vesicles to deliver gene therapies in vivo – directly to the patient.
Extracellular vesicles are tiny packages of biomolecules secreted by cells that help cells communicate with each other. By utilizing these extracellular vesicles to deliver gene therapies for rare blood diseases, STRM.BIO avoids the immune system response other methods of delivery can trigger. And by administering the therapy in vivo – meaning that the patient’s cells are not removed from the body to administer the therapy and then reinjected – STRM.BIO’s solution is far more targeted and can be used for repeat dosing. It’s also much simpler and more accessible, making it possible for more patients to take advantage of life changing – and often lifesaving – therapies.
To develop this technology, STRM.BIO received grants from the Bill and Melinda Gates Foundation and the Mass Life Sciences Center (MLSC).
Read on for our interview with STRM.BIO founder Jonathan Thon!
Harvard Innovation Labs: What inspired you to start this venture? How did you come to do this work?
Jonathan Thon: STRM was founded on the possibility and promise of using extracellular vesicles to deliver gene therapies in vivo.
Over the last decade there has been tremendous progress not only in our ability to develop gene therapies but also in our understanding of the tools and technologies necessary to deliver them in vivo. Pivotal proof-of-concept studies by many academic labs, including our scientific advisors and collaborators, have demonstrated that extracellular vesicles could be used to deliver gene therapies in vivo. The field still had to progress significantly before we could realistically start a company to realize this opportunity, but we arrived there a few years ago and there was suddenly the opportunity to solve a major bottleneck in gene therapy.
HIL: What keeps you motivated as you develop your venture?
Thon: The vision of what the world could look like if we’re successful! The idea that we could enable gene therapies to become viable therapeutic options for people is transformative. For many people, it’s going to change the landscape of medicine by curing diseases as opposed to treating their symptoms. For others, it offers the possibility of effective, life-changing treatment where none is currently available.
HIL: Have there been any unexpected challenges while developing STRM.BIO?
Thon: Not unexpected! One of the things we pride ourselves on at STRM is the culture we are building. We believe strongly that it is people who build companies – not capital – and that a biotech company is only as good as its science. We put a lot of value on bringing in not just the brightest and most talented people, but also the most collaborative and innovative personalities to the company. It matters a lot to us that we continue to build a strong, cohesive team where we trust each other, that we give each other ownership of programs, and that we can work together alongside our partners to build the future we collectively see.
HIL: Has STRM.BIO been affected by COVID-19 over the past year and a half?
Thon: Luckily, not as much as others. We founded STRM on a hybrid-virtual operating model. The idea was to avoid reinventing infrastructure and recreating pre-existing expertise when we could leverage them instead to accelerate programs and keep our focus where it matters – our research.
When I first began pitching the operational idea behind STRM, in the fall of 2019 (pre-COVID), it was met with little enthusiasm. Frankly, it was very hard to get investors even to return my calls. A couple months later, in February 2020, COVID became our new reality. The premise that we could leverage existing infrastructure and work remotely suddenly became a lot more exciting and attractive. It also turned out to be true! Its execution made it possible for STRM to move a lot faster than any of us expected in our first year of operation and accelerate while others were slowing down during a global pandemic.
STRM turned out to be the right company built at the right time under the right operating model. Of course, there are challenges related to COVID. We’re a growing team that’s not entirely virtual but hybrid, and there are instances when we benefit from bringing people together. For example, STRM occupies physical laboratory space at the Life Lab to advance programs that we are best positioned to deliver on faster than our collaborators or partners. In these spaces we make sure our employees are vaccinated and that we are adhering to safety standards so nobody is put at risk.
HIL: What’s next for your venture? How do you see STRM.BIO growing?
Thon: We’ve significantly outperformed this year against some very ambitious targets and are moving into 2022 ahead of schedule. In addition to a growing team, we have shown that we can reproducibly manufacture our extracellular vesicles at scale, have successfully developed gene therapy assets and validated them. We have also shown that we can load these assets into our vesicles for delivery to stem cells, that they impart therapeutic effect, and, when injected into animal models, that they biodistribute to targeted tissues in vivo. This year, we are advancing dose finding and safety studies, completing a QA/QC testing, and tech transferring our process to cGMP in advance of formal (pre)clinical development.
HIL: Can you tell me about how you got involved at the Life Lab? What has it been like to be part of this community?
Thon: I first became aware of Harvard’s i-lab while I was an Assistant Professor at Harvard Medical School and Brigham and Women’s Hospital, and that’s where I incubated my first biotech company (PlateletBio). At the time, the Life Lab didn’t exist, otherwise we would have moved our R&D here. Instead, we moved into LabCentral, and, as the company grew, progressively larger incubators and laboratory spaces.
Our chief operating officer at STRM.BIO, David Raiser, had a very similar experience with his previous company. Aldatu also spent time at the i-lab before moving to the Life Lab during its inaugural year. When we began looking at space for STRM, it made all the sense to revisit the Life Lab. David had very positive experiences there, as PlateletBio did at LabCentral, and the Life Lab ended up being the best choice for STRM through its first year.
The community at the Life Lab is great. We believe strongly in a culture of collaboration and trust at STRM and you see those same cultural values reflected not just in the staff at the Life Lab but in the companies that are invited to join the Life Lab as well. It is incredibly energizing and exciting to work with companies that share the same passion for transformative science as we do. The space is very comfortable to be in, the laboratory is appropriately sized, and we have access to all the equipment we need. When it comes to fluid working spaces and availability of resources, all our needs are met. Location is essential, and the Life Lab is central not just to our employees, but also our investors, advisors, and collaborators. The price per bench is also reasonable, which for an early-stage company matters quite a bit.
HIL: Has there been anything else significant about your experience building STRM.BIO that you’d like to share?
Thon: People are the driving force of any organization. STRM is building a culture and team that realizes our values of simplicity, purpose, collaboration, and people, and the opportunity that creating a strong and vibrant working environment brings. At our core, this is the most transformative element of STRM.BIO.