Perspectives on Cell and Genetic Therapies at ASGCT 2021

May 17, 2021

Perspectives on Cell and Genetic Therapies at ASGCT 2021

I just had the opportunity to attend this year’s American Society of Gene & Cell Therapy (ASGCT) annual meeting — one of my favorite conferences of the year. Even though we were virtual (and despite the technical difficulties), I loved connecting with colleagues in the cell and genetic therapies space, listening to Nobel Laureate Dr. Jennifer Doudna’s fireside chat and learning more about new advances in the field.

ASGCT’s mission is to “advance knowledge, awareness and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.” It is one of the places for the cell and genetic therapies community to get together. Not too long ago, however, it was a small conference that was mostly focused on academic research. The community was small, but one where lasting connections were made — I even met my scientific partner from a previous company through ASGCT. I’m excited to see this conference, and the connections we made, grow and parallel the growth and potential we all see in cell and genetic therapies.

This year, Vertex presented data across three different therapeutic areas, Duchenne muscular dystrophy, hemoglobinopathies (sickle cell disease and beta thalassemia) and type 1 diabetes, demonstrating the breadth of our pipeline and our commitment to these technologies. If you haven’t made the connection between Vertex and cell and genetic therapies yet, now’s the time. We have a broad and exciting portfolio in cell and genetic therapies, and we’re continuing to build our expertise, our tools and our collaborations. And we’re proud to be a member of this community.

Our research strategy guides us, and when we decide to work on a disease, we investigate it from every angle. That could mean gene editing using CRISPR/Cas9, working to optimize a viral delivery mechanism, researching conditioning regimens, or any and all of the above. It could also mean working on more than one type of potential treatment to try to identify the one that will ultimately meet our goal of bringing forward a treatment option for patients. That’s why it was great to share updates from our Duchenne muscular dystrophy, type 1 diabetes and hemoglobinopathies programs via two oral presentations and one poster presentation.

It was also wonderful to facilitate a Vertex-sponsored symposium with Dr. Eric Olson and Dr. Douglas Melton, two leaders in their respective fields. Our discussion focused on the biology underpinning CRISPR/Cas9-mediated gene editing and pluripotent stem cell-derived technologies. I’m always inspired when I hear them speak, both by the amazing science they have accomplished and by their personal connections to their work. Hearing Doug Melton’s personal story is what moved me to join Semma (which was acquired by Vertex). Without that research, we would not be in the clinic right now with an investigational cell therapy for the treatment of type 1 diabetes.

For me, the ASGCT conference has always been a place that motivates and re-energizes me, and this year was no different. The progress that’s being made in the field is incredible, and our commitment at Vertex to leverage the best technologies, manufacturing capabilities and expertise with a patients-first philosophy is how we will continue to push forward.