Gene Therapy
Since the CFTR gene was identified in 1989, there has been hope that this knowledge could unlock better treatments, or even a cure, for Cystic Fibrosis, which is caused by a faulty version of the gene. Over the past 10 years, we have funded the UK CF Gene Therapy Consortium (GTC) to develop a gene therapy product which has the potential to 'correct' the faulty gene in the lungs.
Thanks to our investment of more than £30 million, a Phase 2 clinical trial is due to commence in the spring of 2012 to add a healthy, working copy of the gene into the lungs to replace or override the faulty copy.
The GTC's plan is for more than 100 people with CF to take part in this trial, some taking the active gene therapy product, some taking a placebo, once a month for a year. The results will then be analysed to see if patients show improvements in their lung function. If the trial is successful the aim is to then find a pharmaceutical company to take the product into a phase 3 clinical trial from 2015 onwards.
More information about the UK Gene Therapy Consortium can found at www.cfgenetherapy.org.uk






