Pharma Focus Asia

Vertex Pharmaceuticals and CRISPR Therapeutics Unveil Breakthrough CASGEVY™ Gene-Edited Therapy for Sickle Cell Disease

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. 

Cytiva - Supor Prime filtersMFA + MMA 2024Asia Healthcare Week 2024CPHI Korea 2024CHEMICAL INDONESIA 2024World Orphan Drug Congress Europe 2024INALAB 2024Thermo Fisher - Drug Discovery and the impact of mAbsAdvanced Therapies USA 2024ISPE Singapore Affiliate Conference & Exhibition 20242024 PDA Cell and Gene Pharmaceutical Products Conference 2024 PDA Aseptic Manufacturing Excellence Conference2024 PDA Aseptic Processing of Biopharmaceuticals Conference3rd World ADC Asia 2024