Cystic Fibrosis Clinical Trials in the UK (2026)

CF Research: A UK Success Story

The UK has been at the forefront of cystic fibrosis research, from the discovery of the CFTR gene to pioneering gene therapy trials. CFTR modulator therapy (Kaftrio) has transformed outcomes for most patients, but significant unmet needs remain — particularly for rare mutations, advanced lung disease, and extra-pulmonary complications.

Next-Generation CFTR Modulators

While Kaftrio covers approximately 90% of CF mutations, trials for the remaining 10% are active. Next-generation correctors and potentiators aim to address rare mutations, improve efficacy in residual function mutations, and explore triple-combination alternatives with enhanced pharmacokinetics.

Gene Therapy for CF

UK researchers lead the world in CF gene therapy development. Current trials use viral vectors and lipid nanoparticles to deliver functional CFTR gene copies directly to lung cells. While still early-stage, gene therapy could eventually provide a one-time treatment that works regardless of mutation type.

Infection and Inflammation Trials

Chronic lung infections remain the primary cause of morbidity in CF. Active UK trials include novel antibiotics for resistant organisms (including mycobacteria), anti-inflammatory approaches to reduce lung damage, bacteriophage therapy, and immune modulation strategies.

Extra-Pulmonary CF Research

As lung health improves with modulator therapy, other CF complications are receiving more research attention: CF-related diabetes (CFRD), liver disease, bone health, mental health, and fertility. Trials targeting these aspects of CF are expanding.

Joining a CF Trial

Consider your mutation class (and whether you are on a modulator), lung function status, infection history, and age. Many CF trials are conducted through specialist CF centres. Browse our cystic fibrosis condition page for all recruiting UK trials.

Frequently Asked Questions