CRISPR Therapeutics (CRSP) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic Progress and Therapeutic Franchises

• Four franchises advancing: Heme, in vivo gene editing, CAR-T, and diabetes/T1D, with significant milestones and best-in-class results in each.

• CASGEVY achieved global regulatory approvals for SCD and TDT, launched in 10 countries, exceeding $100M in revenue, and expanding into pediatric populations with ongoing regulatory submissions.

• In vivo gene editing platform demonstrates best-in-class editing in hematopoietic stem cells and liver, with >50% editing in NHPs and durable results, plus promising data for CTX310 in hypercholesterolemia.

• CAR-T platform (CTX112/zugo-cel) shows high response rates in oncology and autoimmune diseases, with 70% CR in DLBCL, durable remissions in SLE, and ongoing expansion into new indications.

• Advancing islet cell therapies for type 1 diabetes, with proof-of-concept data and new candidates moving toward the clinic.

Commercial and Clinical Progress

• Over 75 authorized treatment centers globally for CASGEVY, with rapid patient initiation and cell infusions.

• Pediatric pivotal studies showed 100% of children with sufficient follow-up achieved primary endpoints for SCD and TDT.

• Regulatory submissions for pediatric use expected in 1H 2026; included in FDA's CNPV program and CMMI pilot for Medicaid access.

• Partnerships and potential out-licensing considered for pipeline assets as pharma interest in gene and cell therapy increases.

• 2026 positioned as a pivotal year, with key data readouts and regulatory clarity expected to define the next phase.

Innovation in Enabling Technologies

• Targeted conditioning with antibody-drug conjugates for HSC depletion, minimizing off-target toxicity.

• Proprietary delivery systems and optimized editing for durable, redosable gene therapies.

• AI is leveraged in preclinical design, especially for protein and mRNA folding, with future clinical applications anticipated.

• In vivo CAR-T platform advancing with non-viral, targeted LNP delivery and durable T-cell expression.

• Allogeneic CAR-Ts and in vivo approaches aim to reduce costs, with pricing strategies targeting affordability and broad access.