I remember first learning about CRISPR years ago and thinking, “this technology could really work.” At the time, I didn’t realize that I would get to be part of figuring out how to actually make it work. Fast forward several years and it’s very exciting to be leading the hemoglobinopathies program at Vertex and collaborating with our partners at CRISPR Therapeutics. Every day, I’m grateful to our teams for their commitment and dedication to patients and to the CRISPR/Cas9 technology. We’re working hard and carefully, and we truly believe we can make a difference. 

Everyone at Vertex is passionate about the science and the potential our work might have for patients. This singular mission allows us to stay connected and focused on our end goals. And yet, the field of genetic therapies is still relatively new. With each step we’re navigating uncharted territory, facing unique challenges and opportunities head on and collaborating to solve problems in ways we’ve never done. 

This is one of the reasons I’m proud to share an illustrated video from our “Vertex Forward” series, which describes our research in hemoglobinopathies. We want everyone to understand the problems we’re trying to solve and how we’re building a robust toolkit of technologies and talent to further our research. 
 

Sickle cell disease and beta thalassemia are both blood disorders that are caused by a mutation in the beta-globin (HBB) gene. The mutation leads to issues with the production of hemoglobin, the protein crucial for allowing red blood cells to carry oxygen throughout the body. Our approach, which uses CRISPR/Cas9 gene-editing technology, aims to turn on the body’s natural machinery to make a type of hemoglobin that can carry oxygen. This is in line with the Vertex approach of getting to the underlying cause of a disease. While there is still much more work to be done, we’re hopeful that the precision of our technology may contribute to scientific and therapeutic advances for sickle cell disease, beta thalassemia and potentially other serious genetic diseases. 

It’s incredible to me that we’ve known about the cause of sickle cell disease and beta thalassemia for more than 50 years, but many patients are still waiting for a treatment. At Vertex, we hope to change that.