CRISPR-CAS9 GENE EDITING THERAPEUTICS: FROM FIRST APPROVAL TO EXPANDING CLINICAL PIPELINE

Abstract

The approval of the first CRISPR-Cas9-based therapy by the United States Food and Drug Administration in December 2023 marked a watershed moment for genome-editing medicine, transforming a laboratory research tool into a validated clinical treatment within roughly a decade of its initial characterization. That therapy, indicated for sickle cell disease and transfusion-dependent beta-thalassemia, works by editing the BCL11A erythroid enhancer in autologous hematopoietic stem cells to reactivate fetal hemoglobin production, correcting the underlying beta-globin defect without direct repair of the mutated gene itself [1,2]. Since this approval, the clinical trial pipeline has expanded considerably beyond hemoglobinopathies: registered trials as of the end of 2024 include CRISPR-Cas9 approaches targeting cancer, chronic viral infection, hereditary transthyretin amyloidosis, hereditary angioedema, and inherited dyslipidemias, illustrating a shift from monogenic blood disorders toward a broader range of genetic and acquired conditions [1].