Category: Genetic therapy

Traditionally, cystic fibrosis (CF) has been managed with treatments that address symptoms rather than the underlying cause. The emergence of Trikafta has revolutionized this approach, providing a targeted therapy that directly addresses the defective protein, significantly improving lung function and overall health for many with CF.

In December 2023, the U.S. Food and Drug Administration (FDA) approved the world's first CRISPR-based therapy, Casgevy, marking a historic shift in treating genetic blood disorders. This groundbreaking approach directly modifies a patient's own cells, which is central to understanding what is the active ingredient in Casgevy.