Our Work
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Together against CF we have successfully:
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Start of the product evaluation study to assess the effectiveness of multiple gene transfer agents (GTAs) in pre-clinical studies.
In preparation for the multi-dose trial, we designed a study of almost 200 people with cystic fibrosis. 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.
We recruited ~130 people with CF (aged 12 years and above) into the trial. The trial was a blinded, placebo-controlled study, meaning that each participant had a 50:50 chance of receiving gene therapy or 'dummy' treatment, with neither participants nor investigators knowing which.
Introduction to Gene Therapy
Introduction to CF
Why use Gene Therapy to treat CF?
Treating other diseases in our portfolio
Thank you for your support