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, I’m proud to lead the team at Vertex that’s working to develop a gene editing investigational treatment for DMD.
Our team is committed to raising awareness about this rare and life-shortening disease, and we join the rare disease community this week and on February 28 to recognize both Rare Disease Week and Rare Disease 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 the next video in our Vertex Forward series, which 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. There are and 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.