NHS Approves First Gene-Editing Therapy for Severe Sickle Cell Disease

The groundbreaking treatment, Casgevy, offers hope for a functional cure using Nobel Prize-winning Crispr technology.

Casgevy, also known as exa-cel, is the first Crispr-based therapy approved for NHS use, targeting severe sickle cell disease in patients aged 12 and older.
The therapy edits faulty genes in patients' stem cells, enabling the production of healthy red blood cells and potentially eliminating painful sickle cell crises.
Clinical trials showed nearly all patients avoided hospitalizations for over three years following treatment, highlighting its transformative potential.
Approximately 50 patients per year will initially receive the treatment at NHS centers in London, Manchester, and Birmingham, under a managed access scheme.
Sickle cell disease primarily affects individuals of African and Caribbean descent, and advocates hail the approval as a major step in addressing healthcare inequalities.