Vertex Pharmaceuticals has pioneered CASGEVY™ (exagamglogene autotemcel [exa-cel]), a groundbreaking CRISPR/Cas9 gene-edited therapy designed to address sickle cell disease (SCD) and transfusion-dependant beta thalassaemia (TDT).
This innovative treatment marks a significant advancement in the field, offering a novel approach to the management and potential cure of these blood disorders.
CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy tailored for eligible individuals grappling with sickle cell disease (SCD) or transfusion-dependant beta thalassaemia (TDT). This innovative approach involves the precise double-strand break editing of a patient's own haematopoietic stem and progenitor cells at the erythroid-specific enhancer region of the BCL11A gene.
The therapy aims to enhance the presence of foetal haemoglobin (HbF), the oxygen-carrying haemoglobin naturally occurring during foetal development, thereby mitigating vaso-occlusive crises (VOCs) in SCD patients and reducing transfusion requirements in those with TDT.
CASGEVY has demonstrated efficacy in lessening or eliminating VOCs in SCD patients and alleviating transfusion needs for individuals with TDT.
Sickle Cell Disease (SCD) is a severe, progressive, and life-limiting genetic disorder. Individuals with SCD often experience health-related quality of life scores that fall significantly below those of the general population, leading to a substantial utilisation of healthcare resources. This debilitating condition primarily impacts red blood cells, crucial for transporting oxygen to all organs and tissues throughout the body.
CASGEVY has achieved a groundbreaking milestone as the inaugural medicine to be granted breakthrough designation. This significant approval opens up the potential for individuals grappling with sickle cell disease or transfusion-dependant beta thalassaemia to access a revolutionary one-time therapy for their conditions.
