First-in-human trial of an optimised lentiviral vector for cystic fibrosis gene therapy (360G-Wellcome-106878_B_15_Z)

£1,000,187

Cystic Fibrosis (CF) is a genetic condition that shortens patients' lives, usually because of lung disease. A person with CF inherits two faulty copies of a gene called CFTR, one from each parent. Lacking normal CFTR, CF lungs become clogged with sticky mucus and cannot easily get rid of inhaled bacteria and viruses that damage the lungs. The annual cost of treating the 10,000 UK patients is ~£300M. Time-consuming treatments such as antibiotics and physiotherapy slow down, but don’t stop, the lung disease. New drugs can improve lung function in some patients, but effective medicines to treat lung disease in all CF patients are required. A team led by Chris Boyd (Edinburgh), Eric Alton (Imperial College) and Stephen Hyde (Oxford) have made a new gene therapy product designed to meet this need by putting normal CFTR genes into patients' lungs. The new gene therapy was developed from a virus to make it better at getting into nose and lung cells, and we wish to make it suitable for a clinical trial. We will test it for toxicity, and then study whether it can work safely when sprayed into the noses of CF patients. The ultimate aim is to bring this gene therapy into clinical use to treat CF lung disease.

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Grant Details

Amount Awarded 1000187
Applicant Surname Hyde
Approval Committee Health Innovation Challenge Fund
Award Date 2015-04-16T00:00:00+00:00
Financial Year 2014/15
Grant Programme: Title Health Innovation Challenge Fund Award
Internal ID 106878/B/15/Z
Lead Applicant Dr Steven Hyde
Other Applicant(s) Dr Alastair Innes, Dr Alex McConnachie, Dr Chris Boyd, Dr Deborah Gill, Ms Gursharan Randhawa, Prof Eric Alton, Prof Jane Davies
Partnership Name Health Innovation Challenge Fund
Partnership Value 2105107
Planned Dates: End Date 2023-11-30T00:00:00+00:00
Planned Dates: Start Date 2017-04-01T00:00:00+00:00
Recipient Org: Country United Kingdom
Region South East