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In the fall of 2019, Vertex Pharmaceuticals Incorporated acquired Semma Therapeutics for $950 million. Founded by Douglas Melton, Ph.D. and his team, Semma’s work was solely focused on developing life-changing therapies for people with type 1 diabetes.
Thanks to tremendous financial support from JDRF’s T1D Fund, Melton and his team at Semma were able to generate billions of functional, insulin-producing beta cells grown from stem cells.
Semma then pursued further developments in cell-based therapy for the treatment of diabetes.
“Semma was founded to dramatically improve the lives of patients with type 1 diabetes,” said Douglas Melton, Ph.D., Scientific Founder of Semma, in a 2019 press release. “Vertex is ideally suited to accelerate the achievement of this goal.”
Today, Vertex continues this groundbreaking research with two potential strategies aimed at protecting implanted beta-cells from being attacked and destroyed by the patient’s immune system.
In this interview, we take a deep dive into Vertex’s commitment to type 1 diabetes and their goal of transforming the lives of people with T1D.
T1D Exchange Q&A with Felicia Pagliuca, Disease Area Executive, Vertex
T1D Exchange: For those unfamiliar with the company you work for, can you tell us “who is Vertex?”
Felicia Pagliuca, Disease Area Executive, Vertex: We are a global biotechnology company based in Boston, MA that invests in scientific innovation to create transformative medicines for people with serious diseases. We have multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and have several ongoing clinical and research programs.
We have a robust pipeline of investigational small molecule medicines in other serious diseases where we have deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, we have a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and of course type 1 diabetes (T1D).
What is the overall Vertex approach to drug development?
Our corporate strategy is to invest in scientific innovation to create transformative medicines for serious diseases with high unmet need, that are served by specialty markets. Our research strategy is to combine advances in the understanding of human disease and in the science of therapeutics.
Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them. We put our resources behind this strategy and invest more than 70% of operating expenses in research and development and have 3/5 employees devoted to R&D.
As the Disease Area Executive for the Vertex type 1 diabetes program, can you talk about your background in T1D and how you came to join the team at Vertex?
My work in diabetes started when I was a postdoctoral fellow in Professor Doug Melton’s laboratory at the Harvard Stem Cell Institute many years ago. Our team discovered how to generate stem cell derived beta cells in the laboratory for the first time, and published a seminal paper describing that discovery. But publishing a paper wasn’t enough for us. We wanted to figure out how to translate this discovery into a potential new therapy that could treat people with T1D.
This is why in 2015 we founded a small biotech company, Semma Therapeutics, that was totally focused on the mission of developing a cell therapy for T1D based on those earlier academic discoveries. At Semma, I led our research team, up until Semma was acquired by Vertex in September 2019. Since then, I’ve served as the head of Vertex’s overall T1D program and I am thrilled to be part of company with such a deep commitment – and track record – of creating transformative medicines for people with serious diseases.
How does T1D fit within the Vertex strategy?
Type 1 diabetes is a disease in which the underlying causal biology is well understood. Pancreatic islet beta cells are destroyed by an autoimmune attack. Glucose levels, insulin utilization and hemoglobin A1c are all routinely measured markers of disease state and can clearly demonstrate whether a therapeutic approach is effective. Our approach has the potential to replace the cells destroyed by the immune system in T1D by transplanting cells with the ability to respond to changing glucose levels and produce insulin that is needed.
What is a cell therapy?
Cell therapies are living medicines; the cells replace or repair damaged tissue within a person’s body to help treat a disease or disorder. Some cell therapies can be manufactured from stem cells, which are a type of cell that can be coaxed to differentiate into many cell types through different techniques. In cell therapies, patients may be treated by human cell-based medicines from two sources: autologous or allogeneic cell therapy.
In autologous cell therapy, cells come from the patient’s own body and will not trigger a response by the immune system. In allogeneic cell therapy, cells come from a source other than the patient’s own body and require immunosuppression or other means of immunoprotection to protect the cells from the body’s immune response.
What is the Vertex research approach to T1D?
Our investigational approach, aimed at the underlying cause of type 1 diabetes, is to use transplant technology to replace the insulin-producing cells that are destroyed in people with T1D. We are evaluating approaches to deliver the insulin-producing cells, including a transplant approach that would require immunosuppression similar to an organ transplant, and a device approach with the goal of protecting the transplanted cells from the immune system without the need for the immunosuppression.
Proof-of-concept data in animals has indicated that there may be an opportunity to combine our ability to make large quantities of stem-cell derived human insulin-producing islet cells and transplant technology to initiate clinical research with a new investigative treatment for people with type 1 diabetes.
How does Vertex make the specialized cells?
Our manufactured cells are allogeneic and produced in batches from master cell banks, meaning we do not face the same logistical and technical challenges of autologous (vein-to-vein) cell therapies. Using specialized cell culture, we can direct the fate of stem cells to turn them into insulin producing stem cell-derived islets.
In addition, we have a strong manufacturing team and have been growing this team to ensure long-term success. We are committed to continuing to build world-class cell and genetic therapy capabilities through future investments in people, facilities and technology.
What’s next for the Vertex T1D programs?
Our first approach, cells alone, will go into the clinic in 2021 and the device approach will follow. Our first trial with this approach will begin in people with T1D, not healthy volunteers, so we have an opportunity to validate our approach more quickly than in traditional clinical trials.
Thank you, Vertex, for your dedication to this work!
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