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Genetic therapies: what you need to know

Feature - Published Date: 23/05/2023

With the first genetic therapy clinical trials for CF opening in the UK this year, we have created a new online information resource – which includes FAQs, video Q&As and a glossary. We talk to people from across the CF community about what this means to them.

A day at a CF clinical trials centre

Blog - Published Date: 20/05/2023

To celebrate International Clinical Trials Day on 20 May, Georgia from our Clinical Trials Accelerator Platform (CTAP) central coordinating team reports back on a recent visit to the Bristol CF centre.

She met with the Clinical Trials teams in both the children’s and adults’ CF centres to find out what a typical day is like.

Adapting to a new adventure: Alison’s story

Feature - Published Date: 18/05/2023

As part of our new programme of employment support for people with CF, Work Forwards, 39-year-old manager Alison Smith shares her journey into work and talks about how she manages her schedule alongside her CF.

Search result from the Trials Tracker

A study to evaluate the safety and effectiveness of a new triple combination CFTR modulator therapy in children aged 1 to 11 (VX21-121-105)

This trial has been archived

This study aims to evaluate whether a new triple combination CFTR modulator treatment (VX-121/TEZ/D-IVA) is safe and effective in children with cystic fibrosis aged between 1 and 11 years. CFTR modulators help the faulty CFTR protein to function properly. The treatment is made up of three medications: VX-121, tezacaftor and deutivacaftor. Deutivacaftor is a slightly modified version of ivacaftor that is potentially more stable in the body. This means deutivacaftor can be taken once a day instead of twice a day. All participants will be give n an oral tablet of the treatment to take once a day. The researchers...

This study assessed the safety and effectiveness of the new 'vanza triple' in people with CF ages 6 - 11 who have one F508del mutation and one minimal function CFTR mutation. The results of this study show that this new therapy is at least as good at Kaftrio in improving lung function and it is better in reducing sweat chloride levels.

Click below to find out more. Find out more

Trials Tracker ID: TT009226
Age range: Aged 1 to 11 years
Length of participation: 28 weeks
Trial status: Closed with results
Therapeutic approach: Restore CFTR Function

Strategic Research Centre: Pseudomonas

The Cystic Fibrosis Trust's Strategic Research Centre (SRC) investigating better treatments for Pseudomonas Aeruginosa.

“My mental health deserves the same care and respect as my physical health”: Izzie’s mental health story

Blog - Published Date: 17/05/2023

Content Lead, Izzie, talks about her life with CF, its impact on her mental health and how she tries to tackle it.

What is cystic fibrosis?

Cystic fibrosis (CF) is a genetic condition affecting more than 11,000 people in the UK. Find out more about the condition and how it is caused here.

Videos about genetic therapies

A selection of videos talking about genetic therapies.

“More research desperately needs to happen so everyone with CF can benefit from vital treatments ”: Charlotte shares her story

Feature - Published Date: 15/05/2023

With your support this Wear Yellow Day (16 June), we can continue to fund research into life-changing treatments that work for everyone with cystic fibrosis (CF). Together, we can ensure people with CF, like new mum Charlotte, can look forward to a brighter future. Charlotte shares her story.