NHS to Offer Cutting-Edge Gene Therapy for Sickle Cell Disease

This gene-editing approach was previously approved for treating beta thalassemia, another rare blood disorder, in August 2023.

The National Health Service (NHS) will begin providing a groundbreaking gene therapy aimed at curing sickle cell disease, according to the National Institute for Health and Care Excellence (NICE).

The therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, utilizes the revolutionary CRISPR gene-editing technology. 

It was first approved by Britain’s Medicines and Healthcare Products Regulatory Agency (MHRA) in 2023, making the UK the first country to authorize the treatment.

The treatment will cost the state-funded NHS approximately £1.65 million ($2.1 million) per course and will be available to around 50 patients per year.

How the Gene Therapy Works

Sickle cell disease is a lifelong genetic disorder that affects hemoglobin, the protein responsible for carrying oxygen in red blood cells. 

The therapy involves extracting stem cells from a patient’s bone marrow, editing a gene in a laboratory, and then reintroducing the modified cells into the patient’s body.

This gene-editing approach was previously approved for treating beta thalassemia, another rare blood disorder, in August 2023.

By offering this pioneering treatment, the NHS aims to significantly improve outcomes for patients with these serious blood conditions.

In another recent development, NHS announced that it will introduce AI-powered scanning technology to enhance fracture detection in emergency departments.

This initiative seeks to minimize undiagnosed bone fractures, a frequent error in A&E settings, as highlighted by the National Institute for Health and Care Excellence.

Stay tuned for more such updates on Digital Health News.