CRISPR Therapeutics (CRSP) investor relations material

Strategic focus and pipeline overview

• Emphasis on expanding cardiovascular and autoimmune franchises, with new modalities like siRNA and in vivo CAR-T under evaluation.

• Five clinical trial readouts across three programs expected in the second half of the year, including CTX611 (siRNA, Factor XI), CTX310 (ANGPTL3), and allogeneic CD19 zugo-cel for autoimmune and oncology indications.

• Pivotal trials for these programs are anticipated to begin in 2027, with ongoing assessment of best-in-class opportunities in in vivo CAR-T.

• Strategic shift toward therapeutic area (TA) focus and diversification of modalities, leveraging partnerships like Sirius Therapeutics.

Clinical development and data readouts

• CTX611 phase II data in total knee arthroplasty expected in the second half, with focus on thromboembolic rates and dosing regimens.

• CTX310 phase I-A update will provide longer follow-up on ANGPTL3 reduction, with phase I-B targeting specific indications such as severe hypertriglyceridemia.

• Allogeneic CD19 zugo-cel data will include over 20 patients across autoimmune indications, aiming to match autologous therapy efficacy and safety.

• Next-generation Lp(a) asset CTX321 development will be informed by the HORIZON trial, with focus on high-threshold cohorts.

Market opportunities and strategic decisions

• Factor XI program targets multiple indications, with market opportunities ranging from $1B (cancer-associated thrombosis) to $20B (atrial fibrillation), depending on upcoming trial results.

• Cardiovascular assets may be advanced independently or through partnerships, depending on breadth of development and market potential.

• Allogeneic CAR-T positioned for high-severity autoimmune and CNS indications, with low cost of goods and potential for multi-billion dollar markets.