Posted by Michael Wonder on 01 Feb 2025
Groundbreaking one-off gene therapy approved for severe sickle cell disease
31 January 2025 - People in England with severe sickle cell disease will be among the first to receive treatment using revolutionary CRISPR gene editing technology, following publication of our final draft guidance today.
Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel for use in the NHS in England, providing a potential cure for some people with severe sickle cell disease.
