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Genetic therapies - working together for treatments for all

A report published by the Trust today highlights the vital potential of gene editing for new treatments that tackle the cause of cystic fibrosis (CF) for everyone with the condition; and co-funding and collaboration could hold the key.

One of the highlights of this strange year has been getting access to the life-saving drug Kaftrio. CFTR modulator drugs, such as Kaftrio and Kalydeco, work by addressing the underlying cause of cystic fibrosis. While they significantly improve the health of many people with CF, they don’t work for everyone. At the Trust we remain committed to funding innovative and world-leading research that will help everyone with cystic fibrosis to live longer, better quality lives.

Cutting-edge genetic therapy techniques such as gene editing could be used as a treatment for everyone with CF in the next decade. The aim of genetic therapies is 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 of people with CF would be healthier.

Gene editing is evolving at a rapid pace and in October we committed £750,000 to fund a new Strategic Research Centre (SRC) on gene editing to move the research from the lab towards the clinic. We’ll be funding the SRC in partnership with the US Cystic Fibrosis Foundation.

“We know that promoting collaboration and developing partnerships is an effective way for us to make the most of our research budget. This has become more important than ever this year as the challenges brought about by the COVID-19 pandemic have meant we’ve had to make cuts to it”, said Dr Lucy Allen, our Director of Research.

Our research in focus report on gene editing, published today, explains in detail why genetic therapies are needed, how they work and what the research we’re funding aims to find out.

Download the report

If you can, please consider making a donation today so that we can keep funding the research needed to help people with CF live longer, better quality lives.