EXCISION BIOTHERAPEUTICS

CRISPR technology, including CRISPR-Cas9, CRISPR-Cas12, and CRISPR-Cas13, is revolutionizing genome editing. FDA approved the first CRISPR-Cas9 drug, Casgevy®, for sickle cell disease and beta thalassemia. Other applications include treating urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, cardiovascular diseases, type 1 diabetes, systemic lupus erythematosus, HIV, and blood cancers. Challenges include cost, regulatory standards, and ethical considerations.