The Inquiry

• Gene therapy now offers potential cure for sickle cell disease, but accessibility remains limited by high costs and specialist infrastructure requirements across the NHS
• Treatment involves modifying patient haematopoietic stem cells ex vivo, requiring referral to designated centres with capacity for complex cellular therapies
• Key challenge is ensuring equitable access across all UK regions and addressing barriers to implementation in routine clinical practice alongside existing standard care pathways

Can gene therapy become accessible for people living with sickle cell disease?

ClinicaliQ summarises and contextualises external updates for clinical awareness, then links to the original publisher for the full article and most current context.