The drugs of tomorrow
The event showcased the breadth and depth of work underway on the therapeutic discovery front both at HMS and across Harvard’s innovation ecosystem.
Presenters included recipients of Q-FASTR grants and Blavatnik Therapeutic Challenge Award, as well as founders of companies who have taken place in the lab. The program also featured discussions with regulatory science fellows who discussed the challenges of meeting regulatory requirements to market a drug globally.
Among the first tenants of the new space is the company intrECate Biotherapeutics, founded by Ulrich von AndrianEdward Mallinckrodt Jr. Professor of Immunopathology in the Department of Immunology at HMS Blavatnik Institute. The company is developing technology that uses cellular markers to precisely guide drugs to specific tissues in the body, a process von Andrian likens to a “GPS for drugs.”
When drugs pass through the body’s blood vessels, it’s like a car going through a tunnel.
“All you can see is the tunnel. Without additional information, you don’t know if you’re traveling under a body of water, under a city, or through a mountain,” von Andrian explained.
As a graduate student in the 1980s, von Andrian wondered how blood cells knew where to go inside these seemingly unmarked tunnels of the body’s circulatory system. He discovered that the endothelial cells that form the inner lining of blood vessels in every tissue have unique markers that serve as orientation posts telling blood cells where they are in the body.
If drugs can be configured to seek out and bind to specific tissue markers on endothelial cells, a drug might have a much better chance of reaching its intended destination and doing its job.
Another advantage of this approach is that by targeting specific tissues and spending time there, the drug delivery system could have time to deploy its payload locally, instead of washing itself out through the liver or kidneys. , where barriers to drug diffusion are far more permeable.
The company is just coming out of doors, von Andrian said, and has yet to receive funding from investors.
“When you’re first starting out with a new business in Boston, one of the biggest challenges is just finding lab space,” von Andrian said.
While he’s been working on the science behind the company since he was a graduate student, von Andrian said the technology needed to translate these findings into real therapy has only become available in the past five years.
This makes Blavatnik Life Lab an excellent choice for a project such as this, as it is integrated with the rich scientific culture and infrastructure, including a wide variety of base facilities, from the medical faculty, a said von Andrian.
“When you’re working on biology that requires a lot of advanced technology for experiments, buying all that equipment and establishing the necessary in-house expertise up front could be very expensive and time-consuming,” he said. .
Other presenters noted that the Blavatnik Life Lab facilities were an exciting addition to the resources available at HMS to help those interested in therapeutic development meet the unique challenges of moving from a biological idea to the development of a useful drug. , something that hasn’t traditionally been a strong suit of academia.
Andrew Dates, a graduate student in Therapeutics who works in Stephen Blacklow’s lab in the Department of Biological Chemistry and Molecular Pharmacology at HMS, presented a poster describing his work to understand how a class of cellular receptors bind to their targets on different organs and tissues, a classic problem of basic biology. However, Dates said, he saw this issue as a potential area for drug development early on.
“It’s a whole class of receptors responsible for a very diverse set of biological functions, from hearing to enamel growth on teeth,” Dates said. “At the moment, we don’t really know how cells are activated to do their job, but if we can unlock this knowledge, it could give us the ability to improve health for a whole variety of diseases.”
Other presenters emphasized the importance of diverse and multidisciplinary ways of thinking.
Ricky Cordoba, Leerink Innovation Fellow at the Harvard HealthTech Scholarship, and Nancy AnoruoHMS Instructor in Medicine at Brigham and Women’s Hospital, Associate Director of Innovation at UMass Chan Medical School, and Leerink Innovation Fellow at Harvard HealthTech Fellowship, presented their work on Madernity, a digital therapeutic tool designed to address the large unmet need for physical therapy to prevent and treat pelvic floor dysfunction. The tool, which uses AI to guide exercises, is currently in the process of obtaining FDA approval.
Cordova and Anoruo underlined the importance of one of the key principles of design thinking, the emphasis on the importance of first identifying a need in the community, rather than trying to find a use for a technology or tool.
They did not venture into the fellowship seeking to create digital therapeutics or treat pelvic floor dysfunction, but continued to hear about the shortage of therapists to treat people with the condition from those to whom they spoke as part of their needs assessment research.
“We were looking at needs first,” Anoruo said. “The most important question we can ask is: how can we help? »
Cordova said he was excited about the potential of Blavatnik Life Lab.
“It’s a space buzzing with energy,” Cordova said. “It’s perfect for anyone looking to get something off the ground.”
This combination of a search for solutions to pressing problems and a sense of optimism that there are exciting new tools and discoveries to help improve health and well-being in ways previously unimaginable were common themes at the event.
Many presenters and visitors expressed their hope in the transformation power of new or emerging scientific approaches such as machine learning, genetic engineering, and synthetic biology to transform healthcare and alleviate suffering, and were excited by the passion community members have shown to make this vision a reality.
Pamela Silverthe Elliott T. and Onie H. Adams Professor of Biochemistry and Systems Biology at the Blavatnik Institute at HMSwork with Jeffrey RoadHMS Lecturer on Systems Biology at the HMS Blavatnik Institute and Researcher at the Wyss Institute, on the start-up and new tenant of the Blavatnik Life Lab General Biologics, a synthetic biology company exploring the use of rational design to create new drugs for therapeutic areas with high unmet need.
Silver is also the founder of the Synthetic Biology Hive at HMS, which promotes the use of biology to solve some of the most pressing problems 21st century.
“The beauty is that after 50 years of molecular biology,” Silver said, “we’re at a point where we can now start applying it to solve real-world problems.”