CRISPR Therapeutics (CRSP) investor relations material

Key priorities and portfolio overview

• CASGEVY, a commercial product for sickle cell and thalassemia, is showing strong revenue growth and is expected to be a multibillion-dollar opportunity.

• Three assets—CTX310 (ANGPTL3), zugo-cel (allogeneic CAR T), and CTX611 (siRNA for Factor XI)—are advancing toward potential pivotal trials next year.

• Early clinical assets include an advanced editor for A1AT and CTX340 for refractory hypertension, both expected to have data soon.

• The research arm is developing in vivo CAR T and HSC editing platforms, aiming for durable and broad therapeutic applications.

• A strong balance sheet supports advancing the diverse pipeline and expanding global reach.

CASGEVY commercial progress and expansion

• FDA label expansion to children aged two and older adds 5,500 eligible patients, boosting addressable market.

• Quarterly revenues are approaching blockbuster status, with over 100 patient initiations per quarter and strong payer coverage.

• Most bottlenecks in reimbursement, hospital contracting, and manufacturing have been resolved, supporting steady growth.

• Treatment journey from initiation to therapy remains lengthy, but patient assurance and future gentler conditioning agents are expected to further expand uptake.

• In vivo HSC editing is targeted for introduction around 2032–2033, enabling broader global access.

CTX310 (ANGPTL3) clinical development

• ANGPTL3 knockout leads to significant reductions in LDL and triglycerides, with durable effects observed for at least a year.

• Phase I-B will focus on severe hypertriglyceridemia, with data expected by year-end to inform pivotal trial design.

• Dose selection is guided by phase I-A safety and efficacy, with a fixed high dose moving forward.

• ANGPTL3 offers a one-time treatment alternative to siRNA, with potential pharmacoeconomic benefits and broader metabolic improvements.