Clinical trial of inhaled gene therapy for cystic fibrosis

Novel gene therapy could improve outcomes for people affected by the disease.

Global biopharmaceutical company Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) and viral vector manufacturing company OXB have announced the start of a Phase I/II trial of a novel gene therapy that could improve outcomes for people with cystic fibrosis.

The LENTICLAIRTM 1 trial is a first-in-human study of BI 3720931 – an inhaled lentiviral vector-based gene therapy based on research by the GTC, made up of scientists from the Universities of Oxford and Edinburgh and Imperial College London.

The trial specifically focuses on adults with cystic fibrosis (CF) who genetically cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs.

BI 3720931 could potentially address unmet needs by inserting a functional copy of the CFTR gene in the DNA of airway epithelial cells. The aim is to improve lung function and reduce exacerbations for people with CF irrespective of their gene mutations.

A scientist stands working at a lab bench, while another scientist sits working at the bench opposite. Two scientists in white coats work

Collaborative study

Researchers Dr Chris Boyd, of the Institute for Genetics and Cancer, and Dr Gerry McLachlan, of the Roslin Institute, lead the University of Edinburgh's contributions to the GTC. 

This trial will explore the safety of BI 3720931, aiming to help the 10-15 per cent of people who cannot benefit from existing CFTR modulator treatments. 

There is currently no gene therapy in clinic targeting the lungs. Alongside Oxford and Imperial, we have been working on a range of therapies for 24 years, with Boehringer Ingelheim coming on board in 2018 with an interest in CF. We are delighted to be progressing on our journey to clinic.

Dr Chris Boyd Institute for Genetics and Cancer, University of Edinburgh

CF is a hereditary, lifelong disease that progresses in severity over time, and affects more than 100,000 people worldwide.

While the immediate target is patients who are not eligible for CFTR modulators, this novel therapy has the potential to achieve long-lasting CFTR function improvement and disease modification for people with CF irrespective of mutation type and has the potential for re-dosing if needed. 

We are very grateful to our wonderful team, our partners and funders, and those with CF who have and continue to support us in many ways, including taking part in the multiple trials.

Professor Eric Alton National Heart and Lung Institute, Imperial College London

Funders have included the Cystic Fibrosis Trust, Health Innovation Challenge Fund, Wellcome and the National Institutes of Health and Care Research.

In addition to involvement in the Boehringer Ingelheim CF programme, the GTC spinout AlveoGene was launched in 2023 to develop gene therapy for other lung diseases with support from Edinburgh Innovations (EI) and seed funding from a number of sources, including Old College Capital. EI also facilitated the collaboration with Boehringer.

This is a crucial moment in the development of BI 3720931, which we have progressed together with our partners, since 2018. The partnership with the GTC and OXB provides an excellent example of the progress that can be made when diverse organisations and people come together with a long-term, shared goal to create a new paradigm of care.

Paola Casarosa Member of the Board of Managing Directors, Boehringer Ingelheim

** The Roslin Institute receives strategic investment funding from the Biotechnology and Biological Sciences Research Council and it is part of the University of Edinburgh’s Royal (Dick) School of Veterinary Studies. **  

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