Talking about genetic therapies

Members of our involvement group quizzed three experts on CF genetic therapies in a video Question and Answer session. 

The full recording covers a range of topics listed below. The recording is divided into sections (chapters), so you can go straight to the bit of the recording you’d like to hear about, or watch it all the way through.

For some of the sections, we’ve created separate, shorter videos available on this page too.

Who could benefit from genetic therapies?

The researchers explain how groups of people who are unable to benefit from CFTR modulator medicines such as Kaftrio and Kalydeco (also known as Ivacaftor), and those that are on Kaftrio could see improvements with genetic therapies. They discuss whether and how a combination of both genetic therapies and CFTR modulators could bring additional (‘synergistic’) benefits to different types of CF.

(You can also watch this clip from 5m47s in the full recording)

What are the different types of genetic therapies?

The researchers explain that for people with some rare mutations there could be more than one type of genetic therapy that may be beneficial. For people with other rare mutations, there may be fewer options available.

Watch this animation to understand more about CF mutations.

They go on to talk about treatments being developed for: CF stop mutations; ‘ASO’ therapies CF splicing mutations; genetic therapies that could work for everyone, and the pros and cons of each.

They also discuss the difficulties for researchers in designing medicines to get to the right place.

(You can also watch this clip from 9m39s in the full recording)

Who will be able to take part in trials

The researchers explain how people are selected to take part in clinical trials, the requirements are different for each trial. They also discuss whether people may be able to take a medicine later on in its development.

(You can also watch this clip from 28m2s in the full recording)

Within the Q&A recording

  • How will the therapies be given? (19m49s)

    The researchers explain that genetic therapies will be initially given as an inhaled therapy, via a nebulizer, as this is the easiest journey for the medicines to get to the type of lung cells where they need to be. Delivery to other parts of the body in the future will need to be designed specifically, eg specially designed to reach the pancreas or specially to reach the gut etc

  • When will any trials open or the therapies be available? (22m10s)

    At the time of recording the Q&A session in March 2023, the Early Phase network within the Trust’s Trials Accelerator programme had begun discussions about a Vertex mRNA therapy clinical trial and discussions are taking place across Europe for gene therapy trials.

    The researchers explain how early phase trials work, and how recruitment to them might work and suggests people keep checking the Trials Tracker. They also explain how well tested the therapies are before clinical trials start and share how excited they are to be at the clinical trials stage for these therapies.

  • Which organs might genetic therapies benefit? (37m35s)

    The panel discuss whether genetic therapies designed to benefit the lung may lead to improvements in quality of life and to improvements in symptoms outside the lungs.

  • When can we sign up for clinical trials? (42m25s)

    Listen to the panel discussing the practicalities of taking part in a genetic therapy trial, for example, when are the trials going to open for people to sign up, whether some study visits can be done at the participant’s local CF centre, and can anyone take part no matter where you live in the UK.

Genetic therapies 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.

Go to our glossary

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