CRISPR Therapeutics AG
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a technology for gene editing which is the process of precisely altering specific sequences of genomic DNA. It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes, as well as develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting allogeneic chimeric antigen receptor T cell for autoimmune indications and oncology. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
What does it do?
CRISPR Therapeutics is a biotech company that uses a gene-editing tool called CRISPR to fix diseases at their root cause — inside your DNA. Think of it like a molecular find-and-replace: scientists can locate a faulty section of genetic code and correct it. Their most advanced work targets sickle cell disease and beta-thalassemia, two painful inherited blood disorders. In late 2023, they received FDA approval for Casgevy, the world's first CRISPR-based medicine, developed alongside Vertex Pharmaceuticals.
Casgevy's approval was a historic moment — it proved that rewriting human DNA to cure disease is no longer science fiction, it's a real product on shelves. Investors are watching CRISPR Therapeutics because it sits at the center of what could become a multi-billion dollar gene therapy market. If even one or two of its pipeline treatments work, the financial payoff could be enormous.
How does it make money?
Right now, CRISPR Therapeutics earns almost no revenue on its own — its financials show $0B in revenue for the latest year, which is normal for early-stage biotechs. Its main income comes from collaboration deals, most importantly a partnership with Vertex Pharmaceuticals, which funds development of Casgevy and shares profits. As Casgevy launches commercially and more patients are treated, CRISPR Therapeutics should start receiving milestone payments and royalties — a cut of each sale — from Vertex. The company is burning roughly $600 million per year while it funds its research pipeline.
Why do investors care?
The core bet is that CRISPR's gene-editing platform works not just once, but across dozens of diseases — from cancer to diabetes to heart disease. Casgevy already proves the concept, and the company has early-stage programs in CAR-T cancer therapy, where your own immune cells are reprogrammed to hunt tumors. For the thesis to play out, Casgevy needs strong commercial uptake, pipeline trials need to show positive data, and the company needs to avoid running out of cash before it reaches profitability. If two or three programs succeed, today's $5B market cap could look very small.
Deep Dive
MemberA full investor briefing on CRISPR Therapeutics AG — history, leadership, risks, and outlook.