Recruitment has begun for a major gene therapy trial for people with cystic fibrosis in the UK
Gene therapies are one type of genetic therapy that could be an effective treatment for everyone with cystic fibrosis in the future, including people who are unable to benefit from CFTR modulator medicines such as Kaftrio or the Vanzacaftor triple.
“My son Xander has a rare mutation. He doesn’t currently benefit from existing modulators. So I wish that there will be future breakthroughs that will help everyone living with CF, including genetic therapies. This is exciting news and shows that research is heading in the right direction,” commented CF dad, Duane.
An important part of developing a gene therapy medicine is designing a way to deliver the gene therapy to a precise location inside the cells that line the lungs. This is done by designing an envelope or ‘vector’ that contains the gene therapy.
The GTC gene therapy uses a novel viral vector. This is the second vector the team have developed, after the benefits of a gene therapy using a liposome vector did not show a large enough effect in people with CF when tested in clinical trials in 2014. The development of this new gene therapy medicine known as BI 3720931 was supported by a partnership with Boehringer Ingelheim.
We are delighted that our research funding has helped pave the way for this first stage (phase I) clinical trial to begin. Thank you to everyone in the CF community that works so hard to fundraise for us. Without you, it would not be possible for milestones like this to be achieved. It is a really important step but we know there is a lot of hard work to do before gene therapies are part of routine treatment and we are looking forward to progress in the months and years ahead.
David Ramsden, CEO of Cystic Fibrosis Trust
Over 20 years of gene therapy research
The GTC, comprising scientific and clinical teams from Imperial College London, the University of Oxford and the University of Edinburgh have been working together to develop gene therapy treatments for cystic fibrosis since 2001. Many other companies and charities have invested in their research.
In 2018, the Consortium linked up with the global biopharmaceutical company Boehringer Ingelheim and in 2021 the Consortium’s novel viral vector was licensed to them. After a further four years of research and overcoming manufacturing and regulatory hurdles, trials of their Lenticlair™ gene therapy using this vector have recently begun.
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 known as liposomes 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 people with CF. Next, they identified a novel viral vector, which in preclinical testing was both safe and considerably more effective than the liposomes. The clinical trial of Lenticlair™ uses the viral vector.
“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,” said Professor Eric Alton, Professor of Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London on behalf of the Consortium.
More information on the Lenticlair™ clinical trial can be found in our Trials Tracker. You can find out more about genetic therapies, including what they are and how they work, in the genetic therapies resources section of our website.