CRISPR Therapeutics AG

CRISPR Therapeutics is a biopharmaceutical company developing gene-based medicines for serious diseases.

Its first approved product is CASGEVY (exagamglogene autotemcel), the first approved CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia.
- Under the Vertex collaboration, Vertex leads global development, manufacturing and commercialization of CASGEVY.
- Vertex and CRISPR share profits and program costs 60/40.
- CRISPR records collaboration revenue and net collaboration expense tied to the program.
- CASGEVY is approved in 39 countries, including for younger children in several markets.

The company's pipeline spans several areas:
- **In vivo liver editing:** Uses a proprietary lipid nanoparticle platform. Programs include CTX310 (ANGPTL3, holding FDA RMAT designations), CTX340 (AGT, for refractory hypertension) and CTX321 (Lp(a)). CTX460 targets alpha-1 antitrypsin deficiency and uses the proprietary SyNTase editing technology.
- **siRNA:** Programs developed with Sirius Therapeutics, led by CTX611, a long-acting Factor XI inhibitor for thromboembolic disorders.
- **Allogeneic CAR-T:** Zugo-cel targets CD19 for autoimmune diseases and B-cell malignancies.
- **In vivo CAR-T:** An early-stage platform.
- **In vivo hematopoietic stem cell editing:** An approach aimed at SCD and TDT.
- **Regenerative medicine:** CTX213, an edited iPSC-derived beta cell replacement candidate for Type 1 diabetes.

The company operates a wholly-owned GMP manufacturing facility in Framingham, Massachusetts. It also collaborates with Lilly on a zugo-cel and pirtobrutinib combination study. Updated Nov 2, 2026