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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 our researchers 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.

A quote from a Vertex scientist looking through a microscope that says "We're inspired by the CF community to go the distance in CF through scientific innovation and beyond"


As we recognize CF Awareness Month in the U.S.and CF Awareness Weeknext monthin the U.K. alongside the CF community,we celebratethe progress that’s been made to advance the care for people with CF andacknowledgethework 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 wecontinue to embrace becausewe’re not done.As we continue our journey in CF, we’re focused on three things:


1. Expand access to our existingmedicines
We strive to continue toenable more people toaccess our existing medicinesby expandingregulatory approvals to broaderpatient populations andenablingaccess through reimbursement agreementswith local government agenciesworldwide.

2. Discover and test additional combinationtherapies
We continue to raise the bar for ourselves by discovering and testing combinations of new molecules with the goal of bringing new therapies to patients.

3. Investigate alternative approaches
We continueourwork to developpotentialtreatments that target the underlying cause of the disease for people who need a different treatment approach.We’re investing in our own science andcollaborating alongside external partners to develop other approaches.


Fromour firsthigh-throughput screeningin 2001 to the first clinical study in people with a specific mutationin 2007 to today,we’ve come a long way. Butthestory isfar from overbecause we know people arestillwaiting.AsScience’s editor DanielKoshlandwrote in 1989 in conjunction with the discovery of theCFTRgene, “there is a story that does not begin at the beginning or end at theend, buthas a very happy middle.”The end is a cure,andthis is the shared mission ofVertex, people living with CF, caregivers, health care providers,researchers, regulators and payersalike.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 /research-development/.