CRISPR Therapeutics (CRSP) Corporate presentation summary

Strategic vision and therapeutic franchises

• Focused on four franchises: hemoglobinopathies, CAR T, in vivo gene editing, and type 1 diabetes, with innovation to expand market reach and address unmet needs.

• Global launch of CASGEVY for sickle cell disease and beta-thalassemia, with ongoing expansion into new indications and geographies.

• Advancing in vivo approaches using LNP delivery and developing best-in-class allogeneic cell therapies with novel potency edits.

• Two Phase I programs (CTX310, CTX320) in cardiovascular disease to de-risk the in vivo platform.

• Multi-target siRNA deal with Sirius Therapeutics, including a lead asset in Phase 2 clinical development.

Pipeline and clinical progress

• Broad pipeline includes approved, clinical, and research-stage programs in hemoglobinopathies, cardiovascular, autoimmune, and rare diseases.

• CASGEVY approved in multiple countries for SCD and TDT; manufacturing and commercial agreements in place to support global demand.

• CTX310 Phase I showed up to 50% LDL-C and 55% triglyceride reduction, with a favorable safety profile.

• CTX320 demonstrated durable Lp(a) reduction in NHPs and is advancing in clinical trials for patients with elevated Lp(a).

• Additional programs (CTX340, CTX460) target refractory hypertension and alpha-1 antitrypsin deficiency, showing promising preclinical results.

CAR T and immuno-oncology advancements

• CTX112, an allogeneic CAR T with novel potency edits, is in Phase I trials for autoimmune and oncology indications.

• CTX112 shows efficacy and safety on par with autologous CAR T, with deep B-cell depletion and high response rates, including in post-TCE patients.

• Manufacturing and scalability advantages position CTX112 for global expansion and broader patient access.

• In vivo CAR T research is ongoing, with efforts to optimize LNP composition, targeting, and payload engineering.