Developing effective treatment options for everyone with cystic fibrosis

Blog - 27/02/2026

One of our research goals is to develop effective treatment options for all. To mark Rare Disease Day we recap on some of the research studies that are underway which focus on the development of new treatments for those who have been unable to benefit from current modulators.

Extending access to modulators

The approval of Alfytrek for people with CF who are over 6 years old means that more people with CF may be able to benefit from these medicines. The medicine is available for people with any one of 302 CF variants. Find out more about Alfytrek.

However, this still leaves a significant number of people with CF without an effective treatment, including those with ‘Class 1’ CF variants and those who have CF variants that are so rare that little is known about them and how to treat them. There are also many people with CF who are not able to tolerate these medicines.

We recently reported on the results of the CHOICES clinical trial, which showed that a person’s cellular response in an ‘organoid’ lab test can determine whether someone is likely to respond to a new medicine in a clinical trial. Pioneering research in the US and compassionate use programmes such as that in France are increasing our knowledge of whether very rare mutations may respond to modulators. These results may pave the way for screening to support access to modulators for people with very rare mutations in the future.

Developing treatments for people with Class 1 CF variants

Class 1 CF variants, also known as ‘nonsense’ or ‘stop’ variants, are faults in the CF gene where very little or no CF protein is made. CFTR modulator medicines need CF protein to work.

We’re funding lab-based research to test a new combination of medicines that may mean that people with Class 1 CF variants would be able to make CF protein. Listen to Mark, a PhD student funded through our Karen Menzies PTSuppress Strategic Research Centre, talking about his research at our UKCFC conference, and read more about the aims of the study here.

Research into genetic therapies

Genetic therapies (sometimes known as nucleic acid-based therapies) are medicines that are designed to make working copies of a protein. They are being developed for conditions where there are faults in a gene such as in cystic fibrosis. Genetic therapies offer the potential to treat the lungs of everyone with CF. This includes those who, for whatever reason, are not able to take CFTR modulators such as Kaftrio. This includes people with rare CFTR mutations.

We don’t know when genetic therapies might become available as standard treatments for CF. We’re at the very first stages of testing whether these therapies are safe in people in clinical trials. If these clinical trials go well, it is likely to be many years before they become available.

Look at our Trials Tracker and filter by ‘genetic therapy’ to find out if there are any genetic therapy clinical trials you can take part in.

CF genetic therapies could help cells in the lungs to make working copies of the CF protein, from the CF gene. This means that the health of the lungs would improve. Benefits to CF lung function may or may not lead to improvements in quality of life and to improvements in symptoms outside the lungs.

Find out more about genetic therapies

Understanding the CF protein better

There is potential to design new and innovative medicines to act on the CF protein that will make a bigger difference for more people with cystic fibrosis. These improvements can only be made by increasing our detailed understanding of faulty CFTR proteins and exploring new ways to correct them.

We’re funding research studies to understand more about the shape of the CF protein and how this is affected by different CF variants. Research to understand more about the transport of bicarbonate (rather than chloride) through the CF protein that could improve tailored treatments for people with CF in the future is underway in our ‘Restoring the fizz’ SRC. A research funding partnership with Action Medical Research is supporting a research study to test whether medicines that act to boost the activity of potassium channels could be effective treatments for CF.

All these studies are helping us to achieve our research goal of developing effective treatments for all.

Our research isn't possible without amazing supporters like you.

Please consider making a donation today to help power ground-breaking research, drive campaigns to improve standards of care, and support people with CF and their loved ones every step of the way.

Make a donation