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Caribou Biosciences (CRBU) investor relations material

Caribou Biosciences Citi's Biopharma Back to School Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Citi's Biopharma Back to School Conference summary3 Sep, 2025

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.

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Frequently asked questions

Caribou Biosciences Inc. is a clinical-stage biopharmaceutical company at the forefront of genome editing technology. It focuses on developing genome-edited allogeneic cell therapies for the treatment of cancer and other devastating diseases. The company utilizes CRISPR-Cas9 technology to create precise, directed changes in the DNA of living cells, aiming to develop innovative therapies that can address unmet medical needs. Caribou's leading product candidates include allogeneic chimeric antigen receptor T (CAR-T) cell therapies for hematologic malignancies and solid tumors. This approach allows for the development of therapies that are not patient-specific but can be used as "off-the-shelf" treatments, potentially reducing time and cost compared to traditional autologous CAR-T therapies. The company is headquartered in Berkeley, CA, and its shares are listed on the NASDAQ.

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