A revolutionary inhalable gene therapy, currently undergoing clinical trials across the UK and Europe, aims to enhance lung function in cystic fibrosis (CF) patients, regardless of their genetic mutation. This innovative treatment, known as BI 3720931, leverages lentiviral vectors to introduce a functional CFTR gene into airway epithelial cells. The trial, led by Boehringer Ingelheim in collaboration with leading research institutions, seeks to provide relief to CF patients who do not respond to existing treatments. Initial results are anticipated by early 2027.

Pioneering Gene Therapy Targets CFTR Gene Defects

This new approach represents a significant advancement in treating cystic fibrosis, a chronic condition affecting over 105,000 individuals worldwide. By addressing the root cause of CF—mutations in the CFTR gene—the therapy aims to restore normal cellular function and reduce mucus buildup in the lungs. Unlike current treatments that target specific mutations, this gene therapy is designed to benefit all CF patients, including those with rare or complex mutations.

The lentiviral vector-based therapy works by delivering a healthy copy of the CFTR gene directly into the DNA of airway cells. This method has shown promise in preclinical studies, where it successfully corrected the defective CFTR protein expression. For patients who cannot benefit from CFTR modulator drugs, this novel treatment offers hope for improved lung function and reduced exacerbations. The LENTICLAIR 1 trial will evaluate the safety and efficacy of BI 3720931, with initial findings expected in the coming years. Around 36 participants from various European countries will be involved in this critical phase of testing.

Collaborative Effort Drives Innovative Treatment Forward

The development of this groundbreaking therapy is the result of decades of collaborative research between biopharmaceutical companies and academic institutions. Led by Professor Eric Alton from Imperial College London's National Heart & Lung Institute, the UK CF Gene Therapy Consortium has played a pivotal role in advancing this project. The consortium brings together experts from Edinburgh University, Oxford University, and Imperial College London, focusing on translational respiratory gene therapy.

Professor Jane Davies, the UK Lead Investigator for the trial, emphasized the importance of this new treatment for patients who have limited options. "While CFTR modulators have brought about remarkable health improvements, many patients still lack effective therapies," she noted. "This gene therapy could offer long-lasting benefits and potentially transform the lives of those affected by cystic fibrosis." The trial will proceed in two phases: evaluating safety and tolerability in the first phase, followed by a randomized, double-blind placebo-controlled study to assess clinical efficacy. Participants will also engage in a long-term follow-up study to monitor the therapy's lasting effects.