Caribou Biosciences (CRBU) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company overview and technology platform

• Focuses on next-generation CRISPR genome editing to develop off-the-shelf allogeneic CAR-T cell therapies for oncology and autoimmune diseases.

• Proprietary chRDNA technology improves genome editing specificity and enables efficient multiplex editing while maintaining genomic integrity.

• Pipeline includes three allogeneic CAR-Ts in four phase 1 trials.

Lead asset CB-010: Clinical progress and strategy

• CB-010 is the first allogeneic CD19 CAR-T in the clinic with a PD-1 knockout, aiming to enhance antitumor activity.

• ASCO update included data from 46 patients, with dose expansion focused on second-line large B cell lymphoma (LBCL).

• 80 million CAR-T cells selected as the recommended phase II dose based on efficacy, with no significant safety differences across doses.

• HLA matching analysis revealed patients with ≥4/12 allele matches had median progression-free survival of 14.4 months, compared to less than 3 months for others.

• Modeling suggests 13 batches can serve ~90% of eligible patients, supporting an all-comer pivotal trial in 2H next year.

Safety, efficacy, and competitive positioning

• CB-010 shows efficacy and safety comparable or favorable to autologous CAR-Ts, with no grade 3+ CRS and lower rates of severe ICANS.

• Treated patients often have more aggressive disease and higher risk profiles than typical auto CAR-T recipients.

• Deeper lymphodepletion is used due to allo rejection risk, yet safety remains strong.

• Outpatient administration now FDA-approved, aiming to expand access beyond major centers.