CRISPR Therapeutics (CRSP) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic Overview and Portfolio

• Achieved approval for CASGEVY, the first gene-editing therapy, now available in eight jurisdictions with over 50 authorized treatment centers and more than 50 patient cell collections in the first year.

• Portfolio includes five clinical programs and ten preclinical programs across oncology, autoimmune, cardiovascular, rare diseases, and regenerative medicine.

• Four therapeutic franchises: hemoglobinopathies, CAR T, in vivo gene editing, and type 1 diabetes.

• Manufacturing brought in-house, reducing cost of goods and enabling global expansion, with commercial partnerships and pricing strategies for emerging markets.

• Strong balance sheet with ~$1.9 billion in cash as of end 2024, low burn rate, and significant interest income supporting operations and R&D.

Hemoglobinopathies and CASGEVY Launch

• CASGEVY addresses sickle cell disease and beta thalassemia, targeting a global patient population exceeding one million.

• Launch features a gradual ramp-up due to the complex, elective molecular surgery process, with compounded growth expected as more centers activate.

• Broad system support includes U.S. CMS access model, NHS pricing agreements, and expansion into the Middle East, nearly doubling the anticipated market.

• First GCC patient reimbursed at ~$2M, with manufacturing agreements and commercial partnerships supporting global access.

• Ongoing innovation focuses on improved conditioning agents and in vivo HSC delivery to expand access globally.

CAR-T and Allogeneic Cell Therapy Advances

• Allogeneic CAR-T programs (CTX112, CTX131, GPC3-targeted) show high potency, rapid off-the-shelf availability, and strong response rates, including in patients refractory to T cell engagers.

• CTX112 demonstrates a 50% complete response rate in heavily pretreated cancer patients, with superior cell expansion and safety profile compared to autologous CAR-Ts.

• CTX112 (anti-CD19 allogeneic CAR T) shows 100% ORR in post-TCE patients, with safety, scalability, and manufacturing advantages.

• Manufacturing improvements and novel edits (e.g., Regnase-1, TGF-β) enable scalable, cost-effective production with no observed GVHD and lower toxicity.

• Allogeneic CAR-Ts offer advantages in autoimmune diseases, allowing treatment without discontinuing existing therapies.