Public Meeting, 30 May 2015
On 30 May 2015 we invited members of the CF community to and event at Imperial College. We felt it was important that this group was the first to publicly see the results of the multi dose clinical trial.
Now that the data has been published we can now share these presentation videos on this page.
Main Results Presentation. Professor Eric Alton (50 min)
- 02.40: Why was there a delay in releasing the data?
- 05.10: Gene therapy background
- 09.00: Clinical programme overview
- 20.20: The MD trial
- 25.45: How the trial was analysed
- 27.50: Adverse events
- 31.25: Primary outcome results FEV1
- 34.10: Secondary outcome results
- 38.50: Results stratified by baseline FEV1
- 44.10: What do these results mean?
- 48.50: Thanks
Q&A Session. Professor Eric Alton & CF Trust Chief Executive Ed Owen (44 min)
What is Gene Therapy? (2008): CF gene therapy aims to insert a fully functional CFTR gene into the lung airway cells of people with the condition. Two main types of vector are used to deliver this new copy of the gene. A viral vector, engineered from viral particles or non-viral vectors, plasmid DNA molecules condensed by other chemical entities. More...
