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Partners speed development of cystic fibrosis therapy

Researchers and commercial partners agree to advance work on gene therapy for life-threatening lung condition.

Sutton Lung Belt life

Development of a potential treatment for cystic fibrosis, a rare, progressive, life-threatening disease that affects 70,000 people worldwide, is to be supported by steps taken under a collaborative agreement.

The UK Respiratory Gene Therapy Consortium (GTC), led by Imperial College London, includes scientists from the University of Edinburgh’s Roslin Institute and Institute of Genetics and Cancer among the partners in a licensing agreement with pharmaceutical company Boehringer Ingelheim.

The academic organisations in the collaboration – Imperial College London, University of Oxford and University of Edinburgh – have agreed to grant exclusive rights to Boehringer Ingelheim to develop, manufacture, register and commercialise the gene therapy product.

Under the arrangement, which follows more than two decades of research within the GTC, Boehringer Ingelheim will support development of the potential therapy through clinical trials in people, before it potentially becomes available for use in patients.

The gene therapy under development is designed for inhalation, and uses a modified virus to deliver a healthy version of a gene known as CFTR into the relevant cells in patients.

This can correct for defective or absent protein resulting from mutations in the CFTR gene, which causes cystic fibrosis.

Long-term collaboration

The announcement was made by a group of partners that have been working together since 2018 including the IP Group and Oxford BioMedica.

Research to develop the potential drug included studies in sheep lungs carried out at the University of Edinburgh’s Large Animal Research and Imaging Facility, adjacent to the Roslin Institute.

Large animals such as sheep are useful in the study of human diseases. Their lungs are anatomically similar to those of humans and as such give helpful insight that cannot be gained through studies in animals such as mice.

Our goal in working with our GTC collaborators on a gene therapy for cystic fibrosis was always to reach the stage where a commercial partner would seek to take the product forward, through clinical trials, and eventually to deliver benefit for patients in the clinic. We are really excited to have reached this stage, and grateful to our partners, funders and everyone involved in this research.

Dr Gerry McLachlanGroup Leader, Roslin Institute

Related links

Read the press release

UK Respiratory Gene Therapy Consortium

British Society for Gene and Cell Therapy

Dr Gerry McLachlan on cystic fibrosis