CRISPR Therapeutics (CRSP +2.00%) was co-founded by Emmanuelle Charpentier in 2013. The company's mission is "developing transformative gene-based medicines for serious human diseases."
CRISPR Therapeutics and its big partner, Vertex Pharmaceuticals (VRTX -1.20%), became the first to win regulatory approval for a CRISPR gene-editing therapy in December 2023, with Casgevy for treating sickle cell disease. The two companies won U.S. Food and Drug Administration (FDA) approval the following month for Casgevy to treat transfusion-dependent beta-thalassemia.
The commercial launch of Casgevy continues to gain momentum, with Vertex leading the charge. Meanwhile, CRISPR Therapeutics is advancing several other gene-editing programs.
The company's pipeline includes an allogeneic chimeric antigen receptor T cell (CAR-T) therapy in clinical testing -- CTX112. Allogeneic (meaning involving genetically distinct tissues or cells) CAR-T therapies are often referred to as "off-the-shelf" because they don't require a patient's own cells to be genetically edited; instead, they use cells from healthy donors.
CRISPR Therapeutics is evaluating several in vivo gene-editing therapies, two of which are in clinical studies. The company is also conducting clinical testing of an experimental therapy for treating Type 1 diabetes.
3. Beam Therapeutics