What we do
We’re focused on discovering, developing and bringing innovative medicines to people with serious diseases so they can lead better lives. In pursuit of this mission, we are advancing a robust research pipeline that includes potentially transformative treatments for cystic fibrosis, pain, sickle cell disease, beta thalassemia, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, Duchenne muscular dystrophy, type 1 diabetes and more.

We’re focused on discovering, developing and bringing innovative medicines to people with serious diseases so they can lead better lives. In pursuit of this mission, we are advancing a robust research pipeline that includes potentially transformative treatments for cystic fibrosis, pain, sickle cell disease, beta thalassemia, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, Duchenne muscular dystrophy, type 1 diabetes and more.
Science first
Scientific innovation is at the core of everything we do. Science is where we create value for patients, families, caregivers and society.
*One of only a handful of companies, among companies founded since 1976 (as of December 2021).
**Based on U.S. registry data

Vertex Forward: Illustrating Our Scientific Strategy
Our R&D strategy, illustrated
Today, after more than 20 years of research and development in cystic fibrosis (CF), the results of our efforts include multiple approved medicines that treat the underlying cause of the disease.
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, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, pain, type 1 diabetes and more. Learn more about our pipeline.
No matter the disease, our work is defined by a common strategy that drives a culture of innovation and scientific discovery.