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 the LENTICLAIR^TM 1 trial - a Phase I/II trial of a novel gene therapy that could improve outcomes for people with cystic fibrosis.

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

The UK Respiratory Gene Therapy Consortium (GTC) is made up of scientists from Imperial College London and the Universities of Oxford and Edinburgh, including the Institute of Genetics and Cancer’s Dr Chris Boyd and The Roslin Institute’s Dr Gerry McLachlan.

The LENTICLAIR^TM 1 trial is a first-in-human study of BI 3720931 – an inhaled lentiviral vector-based gene therapy based on research by the GTC.

The trial specifically focuses on adults with 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.

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 approved CFTR gene therapy in clinical practice. 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.

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.

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.

Clinical trial of inhaled gene therapy for cystic fibrosis

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