Caribou Biosciences Inc. (NASDAQ: CRBU) announced that the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to CB-011 for relapsed or refractory multiple myeloma. The designation was based on clinical data from the company's ongoing CaMMouflage phase 1 trial.

CB-011 is an allogeneic anti-BCMA CAR-T cell therapy designed as an off-the-shelf treatment. In the dose escalation portion of the trial, 48 patients have been treated across multiple dose levels. The company selected 450x106 CAR-T cells as the recommended dose for expansion.

Among 12 BCMA-naïve patients treated at the recommended dose, the therapy achieved a 92% overall response rate and 75% complete response rate, with 91% of evaluable patients showing minimal residual disease negativity as of September 2025 data cutoff.

The trial reported a manageable safety profile with no cases of graft-versus-host disease, immune effector cell-associated enterocolitis, parkinsonism, or cranial nerve palsies observed. The most common treatment-related adverse events in patients receiving the selected lymphodepletion regimen included neutropenia (80%), anemia (60%), and thrombocytopenia (49%).

RMAT designation is designed to expedite development and review processes for therapies addressing unmet medical needs in serious conditions. The designation provides benefits including eligibility for priority reviews and accelerated approval if criteria are met.

The company expects to report initial dose expansion data and longer follow-up results from dose escalation in 2026. The ongoing dose expansion includes both BCMA-naïve and BCMA-exposed patient cohorts.