GL67A pGM169 is the combination of cationic liposome (GL67A) and plasmid DNA expressing CFTR (pGM169). The Consortium are currently assessing GL67A/pGM169 in our ongoing clinical trial programme.
In July 2006 we began to look for volunteers to help us with the Tracking Study. This involved following a CF patient during a period of lung exacerbation, whilst they were having their IV antibiotics as in-patients (or if they lived locally, at home).
In preparation for the multi-dose trial of gene therapy we designed a study of almost 200 people with cystic fibrosis. This is called the run-in study. The purpose of this study was to help design the multi-dose trial and to choose the patients most likely to be able to demonstrate benefit.
The aim of the Pilot study was to assess the safety of a single dose of the Wave 1 product. Over 30 patients took part in the study and received one dose of the gene therapy. Patients were treated with different quantities of the gene therapy formulation to assess the optimal dose going forward to the multi-dose clinical trial.
Following on from the single dose trial we commenced with a multi-dose trial in 2012. The trial was based at the the Royal Brompton Hospital in London and at the Western General Hospital and Royal Hospital for Sick Children in Edinburgh. We recruited ~130 people with CF (aged 12 years and above) into the trial.
You can download our MD clinical trial protocol from this page
On 30 May 2015 we invited members of the CF community to be first to see the results of the multi dose clinical trial. Now that the data has been published we can now share these presentations on this page.