2023 Was a Breakthrough Year for Gene Editing

In 2023, CRISPR made significant strides in gene editing, obtaining clinical approval in the UK for treating sickle cell anemia and beta-thalassemia. The FDA in the US also greenlighted the therapy for sickle cell, with a decision on beta-thalassemia expected in March. The approval marked a major breakthrough, utilizing the newly approved gene editor, Casgevy, to correct genetic errors in stem cells, offering a potential cure for genetic diseases. Despite its success, CRISPR has challenges, such as the risk of dangerous mutations.

The CRISPR recipe, comprising a cutting protein and a guide RNA, has been diversified, and AI has played a role in optimizing gene editing tools and discovering new CRISPR variants. Base editing, a less invasive technique, and prime editing, known for precision, are emerging as next-generation technologies. Base editing has shown promise in a one-time treatment for high cholesterol. CRISPR’s expansion into the epigenome, controlling gene activation, offers potential applications in treating chronic infections. With ongoing clinical trials and innovations like prime editing, CRISPR continues to redefine biotechnology.