World-First Sickle Cell Treatment comes to Manchester – Bringing Life-Changing Care Closer to Home

Top doctors, patients and community groups are celebrating the news that people in Manchester with Sickle Cell Disease can now receive life-changing treatment on their doorstep as a world-first gene therapy becomes available at Manchester Royal Infirmary and Royal Manchester Children’s Hospital.

The revolutionary one-off gene-editing therapy, Exagamglogene Autotemcel (exa-cel), has been approved by the National Institute for Health and Care Excellence (NICE) and will be available to both adults and children at the hospitals, making Manchester one of just a handful of centres in the UK offering the breakthrough treatment.

Dr Joseph Sharif, Consultant Haematologist at Manchester Royal Infirmary, said: “This is a huge step forward for people living with Sickle Cell Disease. Exa-cel is a cutting-edge treatment that helps the body produce healthy red blood cells, stopping the painful and dangerous complications of the disease. The fact that patients in Manchester will not have to travel long distances for this world-class care is fantastic news for our community.”

Sickle Cell Disease, which mainly affects Black African and Black Caribbean communities, can cause severe pain, strokes, organ damage, and heart failure. Around 15,000 people in the UK live with the condition, which has a major impact on their daily lives.

The Caribbean and African Health Network (CAHN), a national organisation based in Manchester, has welcomed the news. Charles Kwaku-Odoi, Chief Executive of CAHN, said: “For too long, patients have had to travel to cities such as London or further afield to receive specialist care. This approval offers real hope and moves us towards greater health equality.”

Manchester is already at the forefront of genomic medicine, having successfully treated the first patients in the UK with exa-cel (also known as Casgevy) for thalassaemia last year.

Mark Cubbon, Trust Chief Executive for MFT, said: “This news really does bring a fantastic start to 2025. Manchester is leading the way in pioneering, personalised treatments, and this is another example of how we are providing world-class care right here in the city.”

Clinical trials showed that 96.6% of patients treated with exa-cel no longer suffered from painful Sickle Cell crises, and nearly 98% remained hospital-free for more than three years after treatment, offering a substantial benefit to quality of life.