Across research, manufacturing, process development, clinical, regulatory and more, Vertex Pharmaceuticals’ work in discovering and developing potential cell therapies for type 1 diabetes (T1D) requires incredible talent and cross-functional effort to help bring its approach to life — and hopefully, to patients.
In T1D, pancreatic islet beta cells, which normally respond to changes in blood glucose levels and secretes insulin, are destroyed. These cells are essential because insulin helps the body use glucose (i.e., from carbohydrates in as a source of energy. When patients lose these cells, they require insulin replacement therapy for the rest of their lives.
But the T1D team has a plan to potentially change that. They’re working to make pancreatic islet cells in the lab and are investigating multiple approaches to deliver these cells to individuals with T1D. The first approach, currently under investigation in the clinic, involves infusing cells into a vein that connects to the liver and requires immunosuppression medications to protect the cells from being rejected. The second is an encapsulated islet cell therapy approach, currently in preclinical development, that’s intended to protect the cells from the immune system without the need for ongoing immunosuppression.
Meet just a few of the people and teams that are working tirelessly to advance Vertex’s program in T1D.
Mourad Chettibi is a Manufacturing Operations Senior Scientific Associate on the T1D Chemistry Manufacturing Controls (CMC) team. The team’s main responsibilities are to provide support for the manufacture, testing and release of Vertex’s investigational product for use in clinical trials and to ensure good manufacturing practice (GMP) according to industry quality standards. GMP is an essential step in the drug discovery process to ensure that products are consistently produced and controlled according to quality standards. In his role, he supports the program from the very first step (preparing the cells) and all the way through to shipping the cells for use in our clinical trial.
“Diabetes is a chronic disease that can affect various organs in our bodies, and as a former nurse, I’ve seen people suffering from diabetes complications. I feel grateful for opportunity to be part of the T1D team.”
Beza Gashaw, also on the T1D CMC team, is a Quality Control Specialist. In her role, she tests different assays, in-process samples and final release samples for clinical trials while always adhering to high standards of accountability and stewardship.
“I know a lot of people living with T1D, so I know how they suffer from the disease. When I heard Vertex was developing a potential treatment for T1D, I was so excited for the opportunity to get involved.”
Once the investigational product passes the manufacturing and testing steps, it’s up to the Global Medicines Development and Medical Affairs (GMDA) group, including the T1D Global Clinical Operations (GCO) team, to lead the strategy, operational planning and execution of Vertex’s clinical trials. Marielle Gonzalez, initially in the role of Clinical Operations Study Lead during the initial execution of Vertex’s T1D clinical trial, now provides program management oversight of the cross-functional team as a Project Management Associate Director. She, and many others within GMDA, worked tirelessly to design, plan and initiate Vertex’s first T1D clinical trial in 2021. Marielle started at Vertex in 2012 and has grown her career and responsibilities.
"It’s a privilege to be a part of this journey and the team that has developed a potential therapy ready to be tested clinically.”
Benjamin Flagg is a Clinical Scientist on the T1D GCO team. He supports all clinical study start-up activities and works with the Clinical Development team to provide scientific, clinical and operational input. He attributes the team’s success to never losing sight of the larger purpose behind each task. For him, and many others, this work is personal — his brother has T1D.
“This T1D study team has balanced and executed with exceptionally high efficiency and quality. There is a palpable passion that motivates this team to strive for higher and higher heights.”
Along every step of the way is the Process Engineering and Manufacturing team. Rishi Dua, Director of Process Engineering, helps bring concepts to plan for potential commercialization through designing and qualifying equipment, developing and validating processes, and ensuring compliance to produce high-quality devices. As Rishi puts it, “engineering is all about making things that go on to help make people’s lives better.”
“Engineering is all about making things that go on to help make people’s lives better. It is an opportunity to adopt and perfect the development of cutting-edge technologies, a great reason to get up for work in the morning.”
Thank you to Mourad, Beza, Marielle, Ben, Rishi and the hundreds of other Vertexians who wake up every day energized and motivated to make a difference in the lives of people living with T1D.