Caribou Biosciences (CRBU) 7th Annual Evercore ISI HealthCONx Conference summary

Company overview and technology

• Focused on developing off-the-shelf CAR-T cell therapies using proprietary chRDNA CRISPR genome editing technology for hematologic malignancies and autoimmune diseases.

• Three ongoing phase I clinical studies: non-Hodgkin lymphoma (CB-010), multiple myeloma (CB-011), and AML (CB-012), with three data readouts planned in the first half of next year.

• Committed to advancing to a pivotal trial in second-line large B-cell lymphoma by end of next year, pending confirmatory data.

• chRDNA platform enables multiple precise edits, supporting complex engineering strategies for different indications.

Editing strategies and clinical rationale

• CB-010 uses PD-1 knockout to enhance anti-tumor activity in lymphoma, showing promising efficacy and durability, especially with partial HLA matching.

• CB-011 for myeloma employs immune cloaking by removing Class I presentation and expressing HLA-E to prevent immune rejection.

• CB-012 for AML combines PD-1 knockout and immune cloaking, requiring five edits, demonstrating the platform's editing efficiency.

• HLA matching strategy in CB-010 correlates with improved pharmacokinetics and efficacy, with a four-plus match threshold providing outcomes rivaling autologous CAR-T therapies.

• Best match strategy being built for CB-011 and CB-012 as data accumulates.

Clinical data and development plans

• ANTLER trial in third-line and later lymphoma showed a 44% six-month CR rate, outperforming autologous CAR-T therapies in similar settings.

• FDA approved move to second-line setting, where confirmatory 20-patient cohort is enrolling with data expected in the first half of next year.

• If confirmatory data is positive, a pivotal phase three trial will be initiated by end of next year.

• CB-010 is also advancing into autoimmune indications, starting with lupus, leveraging oncology data showing deep B-cell aplasia and similar duration to academic benchmarks.

• IND cleared for lupus study, with parallel cohorts for lupus nephritis and extrarenal lupus, and HLA matching will be used.