Putting science first

Investing in science
to improve lives
We believe in the promise and potential of science to have a transformative impact for people living with serious diseases. It’s how we create value for patients, families and society as a whole. It’s why we dedicate the vast majority of resources toward innovation, from building teams of top talent to creating a robust toolkit of technologies — all of our efforts are aimed at treating serious diseases. We invest in science so people can live better lives.
Creating a virtuous cycle
We invest most of our resources back into our research and development engine.
*2023 GAAP figures. Operating expenses defined as R&D and Sales, General and Administrative.
Our strategy in action

Pioneers in CF research and development
We discovered, developed and produced the first medicines to target the underlying cause of cystic fibrosis (CF) — the result of more than 20 years of research and development. And we haven’t stopped. We continue to investigate additional potential therapies for all people with CF.

Building our toolkit in cell and genetic therapies
We’re investing in cell and genetic therapies to build an industry-leading portfolio of technologies, teams and manufacturing capabilities. We're committed to pushing the scientific envelope by leveraging emerging therapeutic modalities that have the potential to have a transformative impact for patients who are waiting.

A portfolio approach to development
When we decide to work on a disease, we investigate it from multiple angles. For example, our sickle cell disease and beta thalassemia programs include multiple approaches. We've developed a gene-editing treatment that is currently approved in some countries for certain eligible people living with sickle cell disease. At the same time, we continue our research to discover and develop conditioning regimens aimed at enhancing the stem cell transplant process, and exploring in vivo gene-editing approaches and small molecule treatment options.
At Vertex, we believe “impossible” is just a starting point.
We’re not limited by what we've done before. We're leveraging our robust toolkit of technologies and expertise to tackle serious diseases like cystic fibrosis, sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes and more.

A team sport
Drug discovery is a team sport. We partner with organizations who are driven to innovate together to solve some of science’s most difficult problems. Our partners share our passion and our goals to better the lives of patients.