News Flash: Groundbreaking gene therapy approved for sickle cell disease

✨ New Treatment Approved
The National Institute for Health and Care Excellence has officially approved a groundbreaking gene-editing therapy for use in NHS England called exa-cel (also known as Casgevy) for severe sickle cell disease starting today, 31 January 2025. Clinical trials suggest exa-cel can stop painful and unpredictable sickle cell crises.

👥 Who It’s For
Available through NHS England.
It will be available for patients over the age of 12, who experience recurring crises, and are suitable for a stem cell transplant but do not have a matched donor.
Wales is also expected to offer this treatment in the coming months, expanding access further.

🔬 How the Treatment Works
Exa-cel uses Nobel Prize-winning CRISPR technology to edit genes in patients' bone marrow stem cells, enabling the body to produce healthy hemoglobin. It's a one-time therapy that aims to provide a long-term solution.

📌 Location
The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham.

📈 Success Rates
In clinical trials, 96.6% of patients achieved a 'functional cure'. Additionally, nearly all participants avoided hospital stays for over three years after receiving the treatment.

💷 Cost of Treatment
While the treatment is priced at £1.65 million, the NHS has negotiated a special deal with Vertex to make it affordable through the Innovative Medicines Fund. Availability is predicted to initially be around 50 patients per year, with about 1,700 people in England eligible.

❤️ What This Means for You
Imagine fewer painful episodes, less time in the hospital, and more freedom to enjoy everyday activities without constant worry.

🏭 Made in the UK
The therapy is manufactured in the UK, near Edinburgh.

📣 Voices of Support
Amanda Pritchard, NHS Chief Executive, describes the therapy as “a leap in the right direction” and “absolutely transformative.”

Community Leaders: John James from the Sickle Cell Society calls it a “groundbreaking” milestone that brings immense hope to the community.

View the full NICE article here

Read more about this development:

NHS England: NHS England details the implementation of the new gene-editing therapy, highlighting its potential to significantly reduce the burden of severe sickle cell disease.

BBC News: BBC News reports on the groundbreaking approval, featuring expert opinions and patient testimonials about the new treatment's impact.

The Guardian: The Guardian explores the societal implications of the therapy, emphasizing its role in addressing health disparities in sickle cell disease treatment.

Sky News: Sky News covers the first-time offering of the therapy to NHS patients, outlining the expected benefits and future prospects for sickle cell disease management.

Learn more about sickle cell disease with this guide

The report is available for free online on the AllMyHealth website.

View Report Here