Vertex Forward: Illustrating Our Scientific Strategy

Follow along as we illustrate the science behind each disease in our pipeline, its underlying cause and how our work fits our strategy

At Vertex, we believe the most important medicines are those that have a transformative impact for patients. And while drug discovery and development are complicated processes, learning about our approach and the diseases in our pipeline doesn’t have to be. We’re excited to share this animated video series, “Vertex Forward: Illustrating our Scientific Strategy,” where you can see our philosophy and strategy explained. In the coming months, we’ll share additional videos to provide a glimpse into each disease within our pipeline, its underlying cause, and how we are working to invent potential treatments for each.

 

 

Illustrating Our Scientific Strategy

Learn about the Vertex research principles that are the connective thread behind everything we do — from discovering molecules to applying our work in the clinic — and our hopes for the future of our expanding pipeline. 

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Exploring APOL1-Mediated Kidney Diseases

See how inherited changes in the APOL1 gene can cause severe kidney diseases, including focal segmental glomerulosclerosis (FSGS), and how our scientists are working toward a potential treatment for APOL1-mediated FSGS.

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Taking on AATD

Take a few minutes to learn about the inherited lung and liver disease alpha-1 antitrypsin deficiency (AATD), and see how we're using our expertise in protein folding (from our research in cystic fibrosis) as the foundation for our research for a potential treatment for AATD. 

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Diving Into Type 1 Diabetes 

Join us in learning about the science behind this chronic, metabolic disease and our innovative research into cell therapy approaches for a potential treatment. 

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Our Focus on Hemoglobinopathies

Watch as we illustrate the underlying causes of both sickle cell disease and beta thalassemia and our work on an investigation al gene-editing approach to potentially treat those living with these diseases.  

Learn more about sickle cell disease

Learn more about beta thalassemia

 

Innovating in Pain Research

Learn about our investigational program to treat pain as we research new mechanisms to potentially block pain signals. 

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Navigating Duchenne Muscular Dystrophy

Take a few minutes to learn about Duchenne muscular dystrophy (DMD) and see how we're investigating CRISPR gene-editing technology in the hopes of changing precise parts of the DMD gene. 

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