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

Strategic Overview and Portfolio Highlights

• Four main franchises: hemoglobinopathies (CASGEVY), allogeneic CAR-Ts, in vivo gene editing, and diabetes cell therapies, aiming to develop transformative gene-based medicines for serious diseases.

• Portfolio includes one approved therapy, five clinical programs, and ten preclinical programs, expanding into both common and rare diseases.

• Focus on building a sustainable, industry-leading genomic medicines company with operationalized in-house manufacturing and a strong balance sheet of ~$1.9 billion as of end 2024.

• Serial innovation in enabling technologies, such as targeted conditioning, in vivo HSC editing, and plug-and-play LNP/mRNA platforms, broadens patient access and supports pipeline growth.

• Diabetes franchise advances engineered islet cell therapies, with multiple parallel programs and collaborations to address severe insulin-dependent diabetes.

Key Product and Pipeline Developments

• CASGEVY, the first approved gene-editing therapy, targets sickle cell disease and beta thalassemia, approved in eight jurisdictions with over 50 authorized treatment centers and >50 patients initiating cell collection by end of 2024.

• Allogeneic CAR-T programs (CTX112, CTX131, GPC3) show strong efficacy and safety, with rapid manufacturing, lower costs, and promising results in oncology and autoimmune diseases.

• In vivo gene editing programs (CTX310, CTX320, CTX340, CTX450) target cardiovascular and rare diseases, with durable, single-dose effects demonstrated in preclinical and early clinical studies.

• Manufacturing brought in-house, reducing CAR-T cost of goods to $10,000 per dose, enabling global clinical trials and commercial viability in emerging markets.

• Additional preclinical programs targeting hypertension and acute hepatic porphyrias are progressing toward the clinic.

Clinical and Commercial Milestones

• 2024 and 2025 are described as inflection years, with multiple clinical readouts expected across all franchises, including quarterly CASGEVY updates, CTX310/CTX320 cardiovascular data in 1H 2025, and CTX112 oncology/autoimmune update mid-2025.

• CASGEVY launch trajectory remains strong, with favorable market access, first GCC patient reimbursed at ~$2M, and expansion into the Middle East nearly doubling the anticipated market.

• Investments made to expand manufacturing and meet global demand for disease-modifying therapies.

• Platform engine expected to generate 1–2 new IND/CTAs per year, supporting continued pipeline growth.

• Path to profitability supported by commercial drug revenue, robust pipeline, and opportunistic business development.