Vertex Pharmaceuticals and CRISPR Therapeutics have collaborated to develop a groundbreaking gene-edited therapy known as CASGEVY™ (exagamglogene autotemcel) specifically designed to address sickle cell disease and transfusion-dependant beta thalassaemia.
CASGEVY™ is a genetically modified autologous CD34+ cell population enriched with human haematopoietic stem and progenitor cells. These cells undergo ex vivo editing by CRISPR/Cas9 technology, specifically targeting the erythroid-specific enhancer region within the BCL11A gene.
CASGEVY is intended for treating transfusion-dependant beta thalassaemia in patients aged 12 and above, when haematopoietic stem cell transplantation is a suitable option, yet a human leukocyte antigen matched related haematopoietic stem cell donor is not accessible.
Sickle cell disease (SCD) is an inherited blood disorder impacting red blood cells, crucial for carrying oxygen throughout the body's organs and tissues. It leads to severe pain, organ damage, and a reduced lifespan due to the presence of misshapen or "sickled" blood cells.
Individuals with sickle cell disease (SCD) often endure painful blockages in their blood vessels, referred to as vaso-occlusive crises (VOCs). These can result in acute chest syndrome, stroke, jaundice, and symptoms associated with heart failure.
The approval of CASGEVY represents the world's pioneering regulatory authorisation for a CRISPR-based therapy. This milestone authorisation introduces a novel option for eligible patients eagerly anticipating innovative therapies, providing a promising avenue for treatment.
