CRISPR Therapeutics has earned a place on TIME‘s “Best Inventions of 2024” list, celebrated for transformative gene-editing therapies that tackle complex genetic disorders.

CRISPR’s revolutionary CASGEVY treatment, designed for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), is the first FDA-approved CRISPR-based therapy.

It harnesses CRISPR/Cas9 gene-editing to promote fetal hemoglobin production, significantly easing SCD symptoms by reducing the frequency of painful vaso-occlusive crises. The therapy is now accessible for patients 12 and older in the United States and is under regulatory review in Europe and Saudi Arabia, signaling broader global adoption on the horizon​.

Beyond CASGEVY, CRISPR Therapeutics is advancing a pipeline of therapies targeting cardiovascular and metabolic diseases. New trials include CTX310, which aims to reduce serum lipid levels associated with heart disease, and CTX320, a therapy targeting high levels of lipoprotein(a), a key heart disease risk factor.

Both therapies are being evaluated for safety and efficacy in early-stage clinical trials. By expanding applications of CRISPR/Cas9 technology, CRISPR Therapeutics is poised to bring precision medicine to a wider range of patients with severe genetic diseases​.