The UKCFGTC is pleased to announce that we have received £2.7M to undertake a Phase 1/2a nose trial in CF patients using our Wave 2 product, delivering the CFTR gene using a novel lentivirus. This latest support, which builds on many years of gene therapy funding from the Cystic Fibrosis Trust, the National Institute for Health Research (NIHR) and the Medical Research Council (MRC), has been awarded by the Wellcome Trust/Department of Health’s Health Innovation Challenge Fund (HICF).
At the same time the Cystic Fibrosis Trust have awarded an additional £0.5M to continue to support the scientific work underpinning this latest trial over the next two years.
We aim to recruit 24 patients into the Phase 1/2a nose trial which will last around 9 months. The study will assess safety, and any changes in molecular endpoints, to provide evidence for the efficacy of the lentivirus. The start point of the trial will depend on the time required for manufacture of the Wave 2 product for clinical delivery; we will further update on timelines once these manufacturing data are available.
We are now focusing our research and development efforts on Wave 2, which has proved to be considerably more efficient than the Wave 1 product (delivering the CFTR gene via liposomes). However, the latter, which led to a stabilisation of lung function significantly different to the decline seen in a placebo group, continues to be discussed with potential commercial partners. We will update further on the outcome of these discussions as soon as possible.
9 months ago
The Consortium are pleased to announce the publication of the results from our multi dose gene therapy clinical trial in Lancet Respiratory Medicine.
One hundred and thirty six patients aged 12 and above were randomly assigned to either 5ml of nebulised pGM169/GL67A (gene therapy) or saline (placebo) at monthly intervals over 1 year. Lung function was evaluated using a common clinical measure FEV1.
The clinical trial reached its primary endpoint with patients who received therapy having a significant, if modest benefit in lung function compared with those receiving a placebo. After a year of treatment, in the 62 patients who received the gene therapy, FEV1 was 3.7% greater compared to placebo.
The trial is the first ever to show that repeated doses of a gene therapy can have a meaningful effect on the disease and change the lung function of patients.
More details here.
2 years ago
The Consortium groups are based in three of the leading UK universities. Prior to the formation of the Consortium each group had conducted at least one clinical trial for gene therapy. Therefore, our researchers have been at the forefront of this field for over 20 years.Learn More
Our research is focussed on the development of gene therapies for CF. Taking research ideas from the lab to the clinic is beyond the reach of most academic groups. The Consortium is different as we have aligned our different groups to focus on their key talents to ensure that we continue to progress.Learn More
Since 2004 UKCFGTC members have published over 100 papers in peer reviewed journals and have presented many posters and presentations at international conferences. Where possible we will provide pdfs of publications. However, most of our conference posters and presentations are available.Learn More