A groundbreaking inhalable gene therapy is currently undergoing human trials in the UK and Europe with the potential to revolutionize the treatment of cystic fibrosis (CF). CF is a genetic disease caused by defects in the CFTR gene, leading to the buildup of thick mucus in the lungs and digestive system, resulting in serious complications such as lung infections and breathing difficulties.
The new gene therapy, known as BI 3720931, aims to improve lung function and reduce exacerbations in individuals with CF, regardless of their specific mutation type. This novel treatment involves the use of lentiviral vector-based gene therapy, which inserts a functional copy of the CFTR gene into the DNA of airway epithelial cells.
While some CF patients respond well to existing CFTR modulator medications, a significant portion of individuals do not benefit from these treatments. The LENTICLAIR 1 trial, conducted by Boehringer Ingelheim in collaboration with the UK Respiratory Gene Therapy Consortium and OXB, will assess the safety, tolerability, and efficacy of BI 3720931 in 36 men and women with CF across multiple European countries.
Cystic fibrosis is a progressive and lifelong condition affecting an estimated 105,000 individuals worldwide. With over 2,000 known mutations in the CFTR gene, the severity of the disease can vary significantly among patients. Lentiviral vectors, a type of gene therapy utilizing modified lentiviruses to deliver therapeutic genes into cells, hold great promise for innovative treatments in CF.
Professor Eric Alton, leading the trial and coordinating the UK CF Gene Therapy Consortium, expressed optimism about the potential of this gene therapy to provide long-lasting benefits for CF patients, especially those who are not eligible for current treatments. The trial will consist of two phases, evaluating different doses of the therapy for safety and efficacy before moving on to a larger study.
The ultimate goal of the trial is to establish the long-term efficacy and safety of BI 3720931, offering hope for individuals with CF who have limited treatment options. Trial participants will also be enrolled in a follow-up study, LENTICLAIR-ON, to monitor the ongoing effects of the gene therapy.
The completion of this trial in early 2027 will mark a significant milestone in the development of advanced treatments for cystic fibrosis. With the potential for re-dosing if necessary, this gene therapy could significantly improve the quality of life for individuals living with CF, paving the way for future advancements in the field of respiratory gene therapy. For more information on the trial, visit ClinicalTrials.gov.
