CRISPR Therapeutics (CRSP) Citi’s 2026 Virtual Oncology Leadership Summit summary

Oncology program strategy and innovation

• Focus on zugo-cel, an allogeneic CAR-T therapy with advanced edits for potency and persistence, targeting CD19 in hematologic malignancies.

• Key edits include CAR insertion into the TCR locus, beta-2M knockout for immune evasion, and proprietary Regnase-1 and TGF-beta receptor 2 knockouts to enhance durability and cytotoxicity.

• No HLA matching required, enabling broad patient applicability and redosing without immune response issues.

• Standard lymphodepletion regimen is used, matching autologous CAR-T protocols, with improved cell expansion and efficacy.

• Combination with BTK inhibitor pirtobrutinib is being explored to further improve durable complete response rates.

Autoimmune program expansion and vision

• Rapidly enrolling and expanding indications in autoimmune diseases, including lupus, myositis, scleroderma, ITP, and WAIHA.

• Allogeneic CAR-T offers cost, scalability, and convenience advantages over autologous therapies, with sub-$10,000 cost of goods.

• Early data in SLE patients show complete remission and immune reset, with zero disease activity and sustained B-cell depletion.

• Active trials in ITP and WAIHA, targeting high unmet need and less competitive indications for potential first-mover advantage.

• Dose of 100 million cells used across indications, with ongoing evaluation for optimal dosing.

Regulatory and commercialization strategy

• Parallel development in oncology and autoimmune, with regulatory discussions planned to determine registration paths.

• Potential for single-arm trials in relapsed/refractory oncology settings and small trials for autoimmune indications.

• Pricing strategy aims for $200K or lower per treatment, significantly undercutting autologous CAR-Ts and increasing access.

• Plans for lower-cost manufacturing in Asia and other regions to further expand global access.

• Commercial focus includes niche oncology populations and broad autoimmune indications, with potential for $2B+ in revenue.