Funding new research into genetic therapies to address your top CF research priority
Today the American CF charity Emily’s Entourage (EE) announced a $220,000 (~£180,200) research grant awarded to Professor Lorraine Martin, at Queen’s University Belfast to test whether a new genetic therapy approach, which has been developed by the Irish Biotech company OmniSpirant, successfully produces working copies of CF protein in airway cells grown in the lab. The Trust is proud to be contributing a further £26,000 towards this research project through our Venture and Innovation Award scheme.
The aim of CF genetic therapies is to allow the production of a working CF protein within the lungs of people with CF. This would lead to an improvement in symptoms for all people with CF, including those who cannot benefit from or tolerate CFTR modulators such as Kaftrio.
More about this research study
Some genetic therapies need to be packaged in a protective ‘envelope’, sometimes known as a ‘vector’, to deliver the medicine to the right place. How to make safe and effective ‘envelopes’ for genetic therapies is currently a hot topic of CF research around the world.
Researchers at OmniSpirant have developed a novel ‘envelope’ for a CF genetic therapy called an extracellular vesicle or EV. Professor Martin will be testing the effectiveness of EVs to deliver a CF genetic therapy to lung cells in the lab, and to see if the medicine results in the production of working copies of the CF protein.
We funded Prof Martin to do some initial testing of OmniSpirant’s genetic therapy, so we’re excited to be collaborating with Emily’s Entourage to support the next stage of its development. We know that this area of research is the CF community’s top priority, and we’ll be following progress with interest.
Dr Lucy Allen, Director of Research and Healthcare Data
“By addressing the underlying cause of CF, the gene therapy technology developed by OmniSpirant in collaboration with Professor Martin’s lab has the potential to treat the majority of people with CF regardless of genetic mutation, including those with nonsense and rare mutations,” said Chandra Ghose, PhD, Emily Entourage (EE)'s chief scientific officer. “This is a significant grant for EE. It is our first time co-funding a grant with Cystic Fibrosis Trust. In addition, it represents the diversification of our grant funding mechanisms, including for public/private partnerships, which are designed to accelerate therapeutic development for the final 10%.”
Read more about this research award in a press release from Emily's Entourage.
Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,900 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.
Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives - and we won’t stop until everyone can live without limits imposed by CF.
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