A new partnership bringing together academic and pharmaceutical expertise has been established to develop a gene therapy for cystic fibrosis patients.

Researchers from the UK Cystic Fibrosis Gene Therapy Consortium (GTC), which includes Imperial College London and the Universities of Oxford and Edinburgh, will join with Boehringer Ingelheim and Oxford BioMedica to develop a new viral vector-based gene therapy to take forward to clinical trials.

The partnership builds on pioneering research carried out by the GTC, with contributions from Professor Eric Alton, Professor Uta Griesenbach, Professor Jane Davies and Tracy Harman of Imperial’s National Heart & Lung Institute. It also follows on from early clinical trials of gene therapies for CF in the UK which have shown encouraging results. 

Fixing faulty genes

Cystic fibrosis (CF) is the most common lethal inherited disease in the UK and is caused by defects in a single gene, called CFTR. The disease causes the build-up of thick sticky mucus in the lungs and digestive tract, making patients more prone to lung infections and other complications and reducing life expectancy considerably. CF is estimated to affect 70,000 people worldwide, with the majority of those located in the US and Europe.

This partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice Professor Eric Alton National Heart & Lung Institute

Gene therapy treatments for CF aim to correct the underlying genetic defect, using non-viral or viral vectors to deliver functioning copies of the gene into the patient’s cells.

As part of the new partnership, researchers from academia and industry will share their expertise to develop a new inhaled lentivirus-based gene therapy to take forward to clinical trials.

The hope is that this type of long-term treatment could help CF patients to reach a normal life expectancy and significantly improve their quality of life.

Joint expertise

Professor Alton, coordinator of the GTC, said: “We have, for the last 17 years, vigorously sought to establish whether gene therapy can become a clinically viable option for patients with CF.

“From the beginning the GTC identified that this goal would require incremental increases in knowledge. We have, therefore, built on our non-viral gene therapy experience to develop a new viral vector-based product which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust.

“The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems.”

Imperial's technology commercialisation company, Imperial Innovations, is acting as the lead on the technology transfer for the partnership.

Andrew Tingey, Director of Healthcare Licensing at Imperial Innovations, said: “The combination of expertise and resources realised by this deal will give the project a unique opportunity to develop an advanced therapy that could significantly impact the lives of thousands of people living with cystic fibrosis, and we are delighted to have played the lead role in securing this partnership.”

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