Putting science first
Creating a virtuous cycle
We invest most of our resources back into our research and development engine.
*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
No matter the disease, our work is defined 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 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 every angle. 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 or transfusion-dependent beta thalassemia. 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 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.