Caribou Biosciences (CRBU) investor relations material

Technology platform and clinical programs

• Chardonnay technology enables highly specific CRISPR genome editing, advancing two off-the-shelf CAR T cell therapies for lymphoma (CB10) and multiple myeloma (CB11), both in phase 1 trials with major data readouts expected by year-end.

• CB10 uses PD-1 knockout to prevent CAR T cell exhaustion, aiming for efficacy on par with autologous CAR Ts, with a focus on HLA matching to improve outcomes.

• CB11 targets BCMA in myeloma, employing immune cloaking by knocking out HLA class I and overexpressing HLAE to enhance persistence and reduce NK cell rejection.

• Both programs leverage a manufacturing platform that allows rapid, scalable production and distribution, with no additional scale-up needed for commercial supply.

• The company has paused other programs, prioritizing autoimmune indications for CB10 over AML due to strong oncology data.

Key clinical data and trial design updates

• CB10’s phase 1 study includes a 20-patient cohort with ≥4 HLA allele matches, aiming to confirm retrospective findings that higher HLA matching improves outcomes.

• Data from this cohort, including response durability and pivotal trial strategy, will be shared at a corporate event before year-end, following FDA discussions.

• CB11’s first data release will cover at least 25 patients treated with a higher-dose lymphodepletion protocol, with efficacy benchmarked against bispecifics (60–70% ORR) and plans for dose expansion.

• Both programs emphasize the value of off-the-shelf availability, rapid delivery, and potential to expand access beyond academic centers.

• International enrollment in the Antler study (CB10) includes sites in Israel and Australia, broadening patient diversity and access.

Commercial and operational strategy

• Centralized warehousing and drop-shipping enable community site access without requiring local cryostorage, streamlining logistics for broad adoption.

• Thirteen donor lines can cover over 90% of US lymphoma patients with sufficient HLA matching, supporting scalable supply.

• Current cash reserves fund operations into 2027, but additional capital will be needed for pivotal trials.

• Strategic partnership with Pfizer provides funding, data access, and a right of first negotiation for the myeloma program.

• Market research and operational planning are underway to ensure readiness for commercialization, with a vision to expand cell therapy access to underserved patient populations.