Casgevy: New CRISPR-Cas9 based Therapy for Sickle Cell Disease and β-Thalassemia
Casgevy is a trademark name for a novel gene therapy that uses CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia, two inherited blood disorders that affect millions of people worldwide. CRISPR-Cas9 is a powerful tool that can edit the DNA of living cells with high precision and efficiency. Casgevy aims to correct the genetic defect that causes these diseases by restoring the production of fetal hemoglobin, a form of hemoglobin that is normally present only in newborns and can prevent the harmful effects of sickle hemoglobin or β-globin deficiency.