Our Future Journey in Cystic Fibrosis

May 04, 2021

Our Future Journey in Cystic Fibrosis

When we started our journey in cystic fibrosis (CF) more than 20 years ago, the idea of fixing the broken protein that causes the disease seemed more like science fiction than what was scientifically possible to most people. But the researchers at Vertex Pharmaceuticals Incorporated dove deep into the science and were unwilling to give up, even when faced with setbacks and apparent dead ends. And today, our scientists are still just as committed to our CF research and development program as they were back then, including many of the very same people who played an integral role in discovering the molecules that eventually became our medicines for CF.

Our approach is based on a fundamental understanding of the underlying cause of the disease. But it’s not just discovering a molecule out of hundreds of thousands that might work. It’s about producing it in large enough quantities to test it in countless experiments. It’s about evaluating it in clinical trials with thousands of people who had the courage to volunteer or in some cases taking it back to our laboratories to test it using methods that were developed by our scientists and engineers. It’s about developing a formulation that allows the active ingredient to get to its target. It’s about manufacturing at scale. All of this must come together to ultimately deliver medicines to people with CF. And this is what we’ve done and what we hope to continue to do for everyone living with this disease.

As we recognize CF Awareness Month in the U.S. and CF Awareness Week next month in the U.K alongside the CF community, we celebrate the progress that’s been made to advance the care for people with CF and acknowledge the work that’s still ahead of us. The CF community is our source of inspiration and motivation, and our unwillingness to give up is the mentality we continue to embrace because we’re not done. As we continue our journey in CF, we’re focused on three things:

  1. 1

    Expand access to our existing medicines
    We strive to continue to enable more people to access our existing medicines by expanding regulatory approvals to broader patient populations and enabling access through reimbursement agreements with local government agencies worldwide.

  2. 2

    Discover and test additional combination therapies
    We continue to raise the bar for ourselves by discovering and testing combinations of new molecules for potentially more advanced therapies.

  3. 3

    Investigate genetic therapy approaches
    We continue our work to develop potential treatments that target the underlying cause of the disease for people who need a different treatment approach. We’re investing in our own science and applying it with technology from partners like Affinia Therapeutics, Arbor Biotechnologies, CRISPR Therapeutics and Moderna, Inc.

From our first high throughput screening in 2001 to the first clinical study in people with a specific mutation in 2007 to today, we’ve come a long way. But the story is far from over because we know people are still waiting. As Science’s editor Daniel Koshland wrote in 1989 in conjunction with the discovery of the CFTR gene, “there is a story that does not begin at the beginning or end at the end, but has a very happy middle.” The end is a cure, and this is the shared mission of Vertex, people living with CF, caregivers, health care providers, researchers, regulators and payers alike. Guided by our uncompromising commitment to patients, we are committed to going the distance in CF.

To learn more about our continued efforts in CF and beyond, visit https://www.vrtx.com/research-development/.