CRISPR Therapeutics (CRSP) H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025 summary

Strategic partnerships and business development

• Announced a 50/50 co-development and co-commercialization deal with Sirius Therapeutics for an siRNA platform targeting Factor XI, with US commercialization led internally and China by Sirius.

• Sirius deal adds two additional siRNA targets with licensing rights to be disclosed soon, expanding the company's modality toolbox.

• Actively seeking further business development opportunities, leveraging a buyer's market and positive feedback on recent deals.

• Anticipates three or four multi-billion dollar products from the wholly owned pipeline within 12-18 months.

• Emphasizes a history of value-creating partnerships, notably with Vertex, and plans to continue this approach.

Pipeline progress and clinical updates

• Casgevy, the first approved CRISPR product, is commercialized in eight countries for sickle cell and beta thalassemia, with Vertex leading commercialization.

• Top-line Phase I data from CTX310 (in vivo LNP+mRNA platform) showed 82% triglyceride and 81% LDL knockdown, with more data expected in the second half of the year.

• CD19 allogeneic CAR-T programs in autoimmune and oncology are progressing, with updates expected mid-year.

• Diabetes and solid tumor programs are advancing, with multiple readouts anticipated over the next 12-18 months.

• CTX320 (LPA target) in vivo data is expected soon, with a focus on achieving durable, one-time gene editing effects in cardiovascular indications.

Financial position and commercialization outlook

• Maintains a strong financial position with $1.6 billion in cash, supporting a capital-efficient approach to growth.

• Casgevy launch is progressing well, with 65 activated treatment centers (ATCs), 90 cell collections, and $14 million in revenue as of Q1.

• Targeting 75 ATCs to optimize coverage, with significant growth expected in the Middle East and intra-ATC expansion driving revenue inflection.

• Manufacturing investments are underway to support increasing demand, with a multi-billion dollar market opportunity anticipated.

• Focused on expanding patient access and scaling up operations without immediate capital constraints.