Development of gene therapy treatment for cystic fibrosis moves to the next stage

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The development of gene therapy treatment for cystic fibrosis will be taking a step forward it has been announced today. Find out more about what it means and how the Cystic Fibrosis Trust was involved.

The company Boehringer Ingelheim (BI) will take the lead on a gene therapy treatment developed by the UK CF Gene Therapy Consortium (GTC), moving its development for cystic fibrosis on to the next stage. In technical terms, this means that BI will own the intellectual property for the product and will lead on the future development of the treatment, working closely in partnership with the GTC.

The GTC, a group of researchers based at Imperial College London, the University of Oxford and the University of Edinburgh have been working together to develop gene therapy treatments for cystic fibrosis. The pioneering group was set up with funding from the Cystic Fibrosis Trust in 2001. Many other companies and charities have subsequently invested in their research. In 2018 a new three-way partnership was announced where the GTC joined forces with pharmaceutical company Boehringer Ingelheim (BI) and the gene and cell therapy company Oxford Biomedica.

David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said “It’s great news that Boehringer Ingelheim have committed to the next stage of the development of a gene therapy treatment for people with cystic fibrosis. This is an important step as it brings hope to the whole CF community and in particular to those who don’t benefit from the currently available medicines. All of those who have helped us to invest long term in the work of the UK CF Gene Therapy Consortium should be proud of what they have made possible.”

Lizzy, whose son has CF, added: "The gene therapy research news is positive for the whole CF community. Perhaps more so for those in the 10% of people with CF for whom the new CTFR modulators do not work for. The fact that gene therapy will treat the underlying cause of CF, regardless of the gene mutation, is so exciting. The road to delivering a safe and effective gene therapy treatment for people with CF has been a long and expensive one. Let’s hope this is the home straight!"

How do CF gene therapy treatments work?

CF gene therapy treatments are still in development. Their aim is to deliver healthy copies of the CF gene (known as CFTR) to the lung cells to allow a healthy, working copy of the CFTR protein to be produced. If there are healthy copies of the CFTR protein, this means that the lungs would be healthier, reducing the symptoms of cystic fibrosis.

Why has it taken so long to get this far?

Gene therapies are difficult to develop. Working out how to get the new copies of the CF gene into the right cells is particularly hard. The GTC have tried two different ways of ‘wrapping up’ the CF gene, to safely and effectively deliver the treatment inside lung cells. Clinical trials of their first product (known as ‘Wave 1’) showed that gene therapy treatments could work to treat CF, but improvements were needed with the ‘wrapper’. A different ‘wrapper’ was then used for their Wave 2 product. It is the Wave 2 product (now known as that BI 3720931) that BI will now take the lead on developing.

How you have helped us get to this point

Your support has enabled the Trust to invest substantial sums in the GTC over many years, ensuring its work in both products could continue while searching for a major partner with the resources to take one of the products forwards.

You can read more about this announcement in the press release.

An overview of the latest research we’re funding in this area is available in our ‘Research in focus report on genetic therapies’ published in December 2020.

Please help us to continue to fund research like this by making a donation today.

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