Gene Therapy Multi Dose Clinical Trial Update

Tuesday, November 4th 2014

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As you will know we had hoped to announce the outcome of the Multidose Gene Therapy Trial at the upcoming North American CF Conference (NACFC).

The UK CF Gene Therapy Consortium (GTC) only received the first batch of quality-controlled raw data a few days ago, representing perhaps less than half the total information that we will need to look through.

We now have thousands of pieces of data to analyse and interpret, something that is just not plausible to do in a credible way in a few days. The CF community, and the GTC, have spent a considerable time reaching this stage, and it would be unthinkable to rush through this to try and produce a conclusion that may not stand up to later in-depth scrutiny.

We will, therefore, not be presenting at the NACFC and I, with several members of the GTC, will remain in the UK to push forward the data analysis as rapidly as possible so that we can publish the data as soon as possible. We are obviously disappointed by the delay, and will update the website as soon as we have further information.


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


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