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 the majority of our resources back into our R&D efforts.
*2022 GAAP figures. Operating expenses defined as R&D and Sales, General and Administrative.
Vertex Forward: Illustrating Our Scientific Strategy
A blueprint for serial innovation
Regardless of the disease, our work is guided by a common strategy that drives a culture of innovation and scientific discovery. And while drug discovery and development are complicated processes, learning about our approach and the diseases in our pipeline doesn’t have to be.
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 potential new treatment options for people with CF. Our ultimate goal is to find suitable therapies for all CF patients.
Building our capability in cell and gene therapies
We are investing in cell and gene therapies to build an industry-leading portfolio of technologies, teams and manufacturing capabilities. We strive to push the boundaries of science by leveraging emerging therapeutic modalities that have the potential to improve the lives of people impacted by rare genetic diseases.
A portfolio approach to development
When we decide to work on a disease, we investigate it from every angle. We've developed a gene-editing treatment that is currently approved in some countries for certain eligible people living with sickle cell disease or transfusion-dependent beta thalassaemia. At the same time, we continue to advance our research to discover and develop new conditioning regimens with the aim of improving the stem cell transplant process, and explore oral 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 other serious diseases like cystic fibrosis, sickle cell disease, beta thalassaemia, APOL1-mediated kidney disease, pain, type 1 diabetes and more.