CRISPR Therapeutics AG Dossier
Qualitative Analysis
Business overview
CRISPR Therapeutics AG is a leading Swiss-headquartered biopharmaceutical company focused on developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. The company achieved a historic milestone with the regulatory approval of CASGEVY (exagamglogene autotemcel), the world's first CRISPR-based therapy, developed in collaboration with Vertex Pharmaceuticals. CASGEVY is approved for eligible patients with severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) across major global markets including the United States, European Union, Great Britain, Canada, Switzerland, and several Middle Eastern nations. CRISPR Therapeutics is also advancing a broad, diversified pipeline of ex vivo and in vivo therapies spanning hemoglobinopathies, oncology (CAR-T), regenerative medicine, cardiovascular, and autoimmune diseases.
Research as of 19 Jun 2026
Strategic Initiatives
Growth programs, investments, and their expected impact
Accelerating the global launch and adoption of CASGEVY (exagamglogene autotemcel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in partnership with Vertex Pharmaceuticals, alongside expanding the label to pediatric patients aged 5-11.
Expected impact: Establish a robust commercial revenue stream and expand the addressable patient pool across North America, Europe, and the Middle East.
Advancing a diversified portfolio of in vivo gene editing programs leveraging proprietary lipid nanoparticle (LNP) and SyNTase editing platforms, focusing on cardiovascular diseases (CTX310, CTX320/321) and rare genetic disorders (CTX460, CTX340).
Expected impact: Validate once-and-done gene editing therapies for highly prevalent chronic conditions, expanding the company's reach beyond rare hematological diseases.
Developing zugo-cel (zugocaptagene geleucel) across oncology and autoimmune indications, including systemic lupus erythematosus (SLE), and evaluating it in combination with pirtobrutinib in aggressive B-cell lymphomas.
Expected impact: Establish a leading position in the emerging field of CAR-T therapies for autoimmune diseases and aggressive lymphomas.
Mergers, Acquisitions & Partnerships
Recent deals and strategic collaborations
Strategic Partnerships
Establishes a small interfering RNA (siRNA) therapeutic pillar to complement CRISPR's gene-editing platforms, focusing initially on thromboembolic disorders (CTX611/SRSD107) and providing options to license up to two additional siRNA programs.
Terms: CRISPR paid $25 million in upfront cash and made a $70 million equity investment in Sirius (totaling $95 million upfront). The companies share global development costs and profits on a 50/50 basis, with CRISPR leading U.S. commercialization and Sirius leading in Greater China. Sirius is eligible for up to $800 million in milestone payments.
Governs the development, manufacturing, and global commercialization of CASGEVY, the first approved CRISPR-based gene-editing therapy.
Terms: Costs and profits are split on a 60/40 basis, with Vertex holding the 60% majority stake and leading global operations.
Evaluates CRISPR's zugo-cel in combination with Lilly's pirtobrutinib for aggressive B-cell lymphomas, expanding development opportunities in oncology.
Terms: Clinical supply and collaboration terms to evaluate combination therapy.