NHS patients across England living with severe thalassaemia, a condition that requires regular blood transfusions to survive, could now be some of the the first in the world to benefit from a potentially life-changing one-off gene-editing treatment. Exagamglogene-autotemcel (or exa-cel), utilising Nobel Prize-winning CRISPR technology, is now available on the NHS thanks to a commercial agreement with its manufacturer, Vertex, and a positive recommendation from the National Institute for Health and Care Excellence (NICE). This groundbreaking treatment edits stem cells to allow functioning haemoglobin to be produced. As Kirthana shares in the story below: "I’m only 21 and the idea of depending on transfusions for quite literally the rest of my life is daunting." - the potential for this therapy to free patients from lifelong transfusions marks a significant advancement in modern medicine. In a week marred by ugly events across the UK, this hopeful and positive story on a treatment for a condition that disproportionately impacts those from minority ethnic backgrounds has rightly received wall-to-wall print and broadcast this morning, despite the busy news cycle. As ever, a huge thanks to colleagues for helping bring this story to life, particularly Carl Alexander, Chris Woodhall and Kelli Hooks, whose expertise in partnership working and pitching to media have helped get this the coverage it deserves. https://lnkd.in/eJB5ukQV
Suzy Heafield
2mo
Amazing news 👏
Head Of Fundraising at My Wish Charity
2moMy son carries the Thalassaemia gene and there is so little spoken about this condition, this is amazing news. Hope you are well Tom. x