Vertex Forward: Navigating Duchenne Muscular Dystrophy

Sep 28, 2021

Vertex Forward: Navigating Duchenne Muscular Dystrophy

At Vertex, we’re dedicated to creating transformative medicines that treat the underlying cause of serious diseases — like Duchenne muscular dystrophy (DMD), a genetic disease that affects the skeletal muscles and the heart. The progressive nature of DMD leads to severe complications that drastically impact quality of life and life expectancy. As a geneticist and the lead executive for Vertex's DMD program, I’m proud to lead the team that’s working to develop a potential gene-editing treatment for DMD.

Our team is committed to raising awareness about this rare and life-shortening disease, and we join the DMD community this month and on September 7 to recognize both Muscular Dystrophy Awareness Month and World Duchenne Awareness Day. But our involvement and our commitment to listen to the patient voice doesn’t happen just once a year — this is work we live and breathe every day. One of our team objectives is to educate more people about what we are doing and who we’re working for. That’s why I’m excited to share a video from our Vertex Forward series that illustrates our investigational approach to DMD.

As you can see from the video, our team is investigating the use of CRISPR/Cas9-based gene-editing technology to potentially target a devastating disease like DMD at the level of the DNA. The DMD gene is the largest known human gene among the thousands of genes in the human genome. People living with DMD have a change in their DMD gene that causes the disease. Through gene editing, we aim to alter the gene and correct the problem. Given the novelty of this approach, we know our science must be of the highest caliber from start to finish. Everyone on the team, from research to development, is excited and committed to getting it right. We know there will be challenges, however people with DMD and their families inspire us to keep pushing forward.

While it’s incredible to have the opportunity to contribute to a treatment modality that’s on the leading edge of medicine, it’s the potential impact for patients that motivates us and keeps us focused each and every day.