CRISPR Therapeutics (CRSP) Jefferies London Healthcare Conference 2024 summary

Company overview and platform expansion

• Celebrating 10 years, leveraging CRISPR-Cas9 for transformative medicines in rare and common diseases.

• Approved CASGEVY drug launched globally for beta thalassemia and sickle cell disease.

• Pipeline includes CAR T-cells for oncology and autoimmune, in vivo gene editing for cardiovascular risk, and diabetes programs.

• Preclinical data shown for glaucoma, with ongoing innovation in editing technologies.

CASGEVY launch and commercial outlook

• Strong global demand and payer support for CASGEVY, with expansion into Middle East and Europe.

• Activation of treatment centers (ATCs) is progressing, with plans to increase capacity and global availability.

• Early adopters are driven by patient attitudes and severity, with potential to reach 25,000 severe U.S. patients and 30,000 globally.

• Commercial setup for 2025 focuses on label and capacity expansion, anticipating growing demand.

Financials and profitability targets

• Investment in the franchise expected to continue through 2025, aiming for profitability or break-even by 2028.

• Current cash reserves of $2 billion provide a buffer for ongoing clinical and commercial activities.