Completed Lungs & Breathing Digestion, Kidneys & Other Organs

First-in-human trial of an optimised lentiviral vector for cystic fibrosis gene therapy

Funder Wellcome Charity-funded
Associated organisations Imperial College London · University of Edinburgh · University of Oxford Europe PMC affiliations are not treated as award recipients or mapped locations.
Funding £2.8M
Period Apr 2017 — Dec 2022

In plain English

AI plain-English summary

A new gene therapy, built from a modified virus, is about to be sprayed into the noses of cystic fibrosis patients for the first time to test whether it can deliver working copies of the faulty CFTR gene directly into lung cells. Cystic fibrosis is a genetic condition that clogs the lungs with sticky mucus, trapping bacteria and causing progressive damage. Existing treatments—antibiotics, physiotherapy, and drugs that work only for some patients—slow the disease but do not stop it. Treating the 10,000 UK patients costs roughly £300 million annually. This project addresses the need for a therapy that works for everyone, regardless of their specific genetic mutation. The team has optimised a lentiviral vector to better penetrate nose and lung cells. After toxicity testing, they will spray the therapy into the noses of CF patients to assess safety. If successful, this could lead to a repeatable, inhaled gene therapy that halts or reverses lung disease in all CF patients—changing the standard of care from managing decline to correcting the underlying cause.

View original technical description
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.

View the original record at the funder ↗

Researchers

Chris Boyd (EPMC Awardee)Eric W.F.W. Alton (EPMC Awardee)Steven Hyde (EPMC Awardee)

Related Research

Grants with similar aims, by meaning.

Development of a novel, potent, safe, long-lasting lentivirus-based gene therapy for cystic fibrosis
A randomised double-blind placebo controlled Phase 2B clinical trial of repeated application of gene therapy in patients with Cystic Fibrosis
Development of in vitro transcribed mRNA therapeutics for cystic fibrosis
Multiplexed in vivo optimisation of non-toxic gene transfer agents.
Refinement and assessment of a novel adenovirus targeting platform for application to human gene therapy

Original classification

Health Innovation Challenge Fund Award

Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.