The Sickle Cell Society welcomes the decision from the Scottish Medicines Consortium (SMC) to recommend the CRISPR gene therapy exa-cel (exagamglogene autotemcel), also known as Casgevy, as a treatment option for people with severe sickle cell disorder in Scotland. Exa-cel involves extracting and modifying a patient’s own blood stem cells before returning them to their body, allowing the modified cells to produce healthy red blood cells and address the symptoms of sickle cell. Historically, the only curative treatment available in Scotland has been a donor stem cell transplant, however not all patients have a matching donor. This decision means Casgevy could provide a ‘functional cure’ for those unable to access a stem cell transplant, offering the potential to regain quality of life.

Casgevy is now available on NHS Scotland for eligible patients aged 12 and over. Individuals who may wish to explore this treatment should discuss it with their healthcare professional to understand whether it is the right option for them.

Before sharing further information about our upcoming webinars, we would like to highlight the words of a patient, reflecting on what developments like this could mean for the sickle cell community:

“As someone living with sickle cell anaemia, I write today with a hope I have seldom felt before, and a gratitude that is difficult to put into words.
The news that groundbreaking gene editing research for sickle cell anaemia and other rare blood conditions is moving forward is not just a scientific headline to me—it is a beam of light. For years, my life and the lives of countless others have been defined by pain, fatigue, and uncertainty. This announcement represents more than a trial; it represents a tangible chance at a future free from the relentless grip of this condition.”

You can read the official SMC decision here:
https://scottishmedicines.org.uk/medicines-advice/exagamglogene-autotemcel-casgevy-full-smc2852/

Lastly please find the relevant Detailed Advice Document here and a link to the Decision Explained public information summary here.

Looking Ahead 

For those who would like to find out more, we are planning to run a series of webinars on Casgevy in the coming weeks. This will include one session delivered in partnership with the UK Thalassaemia Society and clinicians, and another featuring a patient who has received Casgevy, offering a lived experience perspective. If you would like to be kept updated and register your interest in attending, please complete the expression of interest form below.

Sickle Cell Society – Register your interest for Casgvey gene therapy webinars