CF genetic therapies

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CF genetic therapies

Find out what they are, whether they might benefit you and learn more about genetic therapy clinical trials.

Genetic therapies are a new type of treatment that are being developed for cystic fibrosis (CF). They work in a completely different way to current medicines for CF such as Kaftrio or Kalydeco. We hope that people with CF in the UK may have the opportunity to take part in genetic therapy clinical trials in the next few years.

We’ve created these resources to help explain what genetic therapies are, who they might benefit and what taking part in a genetic therapy clinical trial involves. 

Five things to know about genetic therapies

  1. Genetic therapies make working copies of a protein. They are being designed for conditions where there are variants in a gene, such as CF. For CF, they would make working copies of the CFTR protein. 
  2. Genetic therapies offer the potential to treat 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.
  3. 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.
  4. The first CF genetic therapy trials will only recruit a small number of people. We hope that more genetic therapy trials will be available over time. The first trials are likely to recruit people with rare gene variants. If these are successful, then trials for everyone with CF could happen in the future.
  5. Current genetic therapies in clinical trials are aimed at the lungs. They aim to deliver the treatment as an inhaled therapy, with the aim of making the lungs of people with CF much healthier. We don’t yet know if or when we will see genetic therapies that target other areas of the body, such as the gut.

What’s in the genetic therapy resources?

  • Glossary

    We’ve put together some definitions of words that you may come across as you learn more about genetic therapies. Where a word is in bold text, there is also a separate explanation of the word in this glossary.

    You may find it helpful to view the glossary alongside other pages in these resources. To do this either long click (if you're on a phone) or right click (if you're on a computer) on the link below and choose 'open in a separate tab'. 

    Go to our glossary

  • Talking about genetic therapies

    Members of our involvement group quizzed three experts on CF genetic therapies in a video Question and Answer session. You can watch the full recording (which is divided into sections), or some shorter edits of some of the topics discussed.

    Watch Q&As on genetic therapies

  • Frequently Asked Questions (FAQs)

    We’ve created written answers to some of the questions you may have.

    Our general FAQ pages covers questions about how genetic therapies work, the differences between genetic therapies and CFTR modulator medicines such as Kaftrio and research the Trust is funding in this area.

    General FAQs

    Our genetic therapy clinical trials FAQs includes questions about who can take part, what would be involved, what it means to take part and how long the trials will last.

    FAQs on clinical trials

  • Training resources for professionals

    We’ve compiled a list of resources for CF professionals – or anyone – to learn more about genetic therapies and Advanced Therapeutic Medicinal Product (ATMP) Trials. The resources range from a 10 minute read and short video clips to recordings of 1 hour webinars.

    Download list of professional resources

Find out more about CF genetic therapies

CF Professionals training resources on genetic therapies

We’ve compiled a list of resources for CF professionals – or anyone – to learn more about genetic therapies and Advanced Therapeutic Medicinal Product (ATMP) trials. They range from a 10 minute read and short video clips to recordings of 1 hour webinars.

FAQs on genetic therapy clinical trials

Read our Frequently Asked Questions on genetic therapy clinical trials, topics range from can I take part, to what will be involved.

Research in focus on genetic therapies

Our Research in focus report on genetic therapies contains an overview of why we need genetic therapies for CF, how they may work and progress made so far.

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