The realization of CRISPR gene therapy.

Casgevy, the inaugural CRISPR-based drug, has been approved by several medical agencies and represents an important milestone in gene therapy. Additional CRISPR-based therapies are currently in clinical trials and could be approved soon. Although there are technological hurdles to overcome, chemical biology has a vital role to play in developing recent breakthroughs in base editing, prime editing, and epigenetic editing into future treatments.