FDA approves Vertex/CRISPR Gene Therapy for Inherited Blood Disorders
FDA approves Vertex/CRISPR Gene Therapy for Inherited Blood Disorders
In a monumental leap for gene therapy and a beacon of hope for millions suffering from life-threatening blood disorders, the US Food and Drug Administration (FDA) has granted approval to Vertex Pharmaceuticals and CRISPR Therapeutics' revolutionary treatment - Exa-cel (casgenevo). This breakthrough represents a new era in treating genetic diseases, paving the way for personalized, curative therapies once relegated to the realm of science fiction.
Understanding the Blood Disorder Battleground:
Inherited blood disorders, such as transfusion-dependent beta-thalassemia and sickle cell disease, affect millions worldwide. These debilitating conditions are caused by genetic mutations that impair the production or function ...
