NEWS: November 2024

The Consortium, comprising scientific and clinical teams from Imperial College, Oxford and Edinburgh Universities have worked together for the last 25 years to bring forward the best approaches to delivering gene therapy for CF.

 

Funded through the CF Trust, Wellcome, Medical Research Council, NIHR, Flutterby and other charities the Consortium delivered the world’s first proof-of-concept in 2014 that gene therapy can benefit lung function in people with CF using fat globules (also sometimes called liposomes or lipid nanoparticles) as a vector to deliver the CF gene into the lungs.

 

However, the benefits were too small to enable the development of a registered treatment for patients and the Consortium continued to work at pace on the next generation approach to delivering the CF gene into the lungs of people with CF. They identified a novel viral vector, which in preclinical testing was both safe and considerably more effective than the liposomes.

 

In 2018, the Consortium linked up with the global biopharmaceutical company Boehringer Ingelheim to push forward this second approach and the vector was licensed to Boehringer Ingelheim in 2021. Since then, this partnership has rapidly pushed through the required toxicology, manufacturing and regulatory hurdles and we are delighted to announce that recruitment into a trial using this viral vector has now started; details can be found at Home | ClinicalTrials.gov (NCT06515002).

 

The Consortium wish to thank all those who have so generously supported the programme, through the many years of round-the-clock working that it inevitably takes to deliver two completely new pharmaceutical products and we look forward with excitement to the results from the upcoming trial.

 

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