Clinical Trial Update

Tuesday, February 4th 2014

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The Guardian, Sunday 18 March 2012Clinical Trial Update. The Wave 1 Clinical Trial (CF gene delivered by liposomes) is going well. We were able to recruit all the patients needed from CF Centres across the UK.

Approximately half the patients have received all the 12 doses of gene therapy or placebo given at monthly intervals.

The remaining will be completing their dosing over the next few months, with the final dose in the final patient due around June. There will then be a period of data analysis, and we hope to present the findings at the North American CF Conference in October. 

There have been 3 safety reviews by an independent external committee, with no unexpected problems noted to date. The trial is double-blind so we, of course, have no knowledge about any efficacy. The project was awarded by the Efficacy and Mechanism Evaluation (EME) Programme and is funded and managed by the National Institute for Health Research (NIHR) on behalf of the MRC-NIHR partnership.


Proposed 3D model of the CFTR protein.


A CFTR Western blot, to confirm protein production in cell culture.