CF genetic therapies
Genetic therapies are a type of treatment that are being developed for cystic fibrosis. They work in a completely different way to current medicines for CF. 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, how they work, who they might benefit and what taking part in a genetic therapies clinical trial may mean for you.
We’ve created them now, so that people with CF and their CF teams can find out more about them as clinical trials of genetic therapies are discussed.
Five things to know about genetic therapies
Before you explore all of the different resources in this section, here are five things to know about genetic therapies.
Where a word is written in bold, we’ve explained it in our genetic therapies glossary.
- Genetic therapies are a type of treatment that are being developed for cystic fibrosis, they work in a completely different way to current medicines for CF such as Kaftrio or Kalydeco.
- Genetic therapies offer the potential of being able to treat everyone with CF, including those who, for whatever reason, are not able to take CFTR modulators such as Kaftrio, this includes people with rare CFTR mutations.
- We hope that people with CF with rare mutations in the UK may have the opportunity to take part in genetic therapy clinical trials in the next few years. Each individual trial will have different requirements for taking part. If these are successful then trials for everyone with CF could happen in the future.
- There are many different types of genetic therapies, some will be suitable for all types of CF (eg gene therapy and RNA therapy), and some will only work for people with specific forms of CF (specific genotypes of CF).
- We don’t know when genetic therapies are likely to become available for people with 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.
What’s in the genetic therapy resources?
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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'.
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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.
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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.
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.
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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.
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.