Gene Therapy Funding Update

Tuesday, August 29th 2017

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LV_diagram.jpgThe 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.

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Further Information


 

Mouse lung large airway (cell nuclei blue) transduced with an adenoviral vector (green).

 

E.coli from a large scale industrial production of our clinical trial plasmid pGM169.

 

A cake that only some of us got to enjoy!

 

Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.