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Caribou Biosciences Announces the FDA Granted Regenerative Medicine Advanced Therapy (RMAT) Designation to CB-011, an Allogeneic Anti-BCMA CAR-T Cell Therapy
-- RMAT granted based on promising initial clinical data, including previously disclosed recommended dose for expansion data of 92% ORR, 75% ≥CR rate, 91% MRD
About this update from Caribou Biosciences, Inc.
[{"type":"text","content":"-- RMAT granted based on promising initial clinical data, including previously disclosed recommended dose for expansion data of 92% ORR, 75% ≥CR rate, 91% MRD negativity in the 12-patient, BCMA-naïve r/r MM patient cohort -- -- Ongoing dose expansion enrollment in CaMMouflage phase 1 clinical trial includes BCMA-naïve and BCMA-exposed cohorts; initial dose expansion and longer follow up on dose escalation data expected in 2026 -- BERKELEY, Calif., March 31, 2026 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CB-011 for relapsed or refractory multiple myeloma (r/r MM). CB-011, an allogeneic anti-BCMA CAR-T cell therapy, is being evaluated in the company’s ongoing open-label, multicenter CaMMouflage phase 1 clinical trial evaluating patients with r/r MM. “Only one in 10 people with multiple myeloma in the U.S. are able to receive CAR-T cell therapies due to long wait times and manufacturing limitations,” said Adriana Rossi, MD, director of CAR-T and stem cell transplant clinical program at the center of excellence for multiple myeloma at Mount Sinai and an investigator on the CaMMouflage trial. “This highlights a critical gap in access for patients with relapsed or refractory disease. An off-the-shelf CAR-T cell therapy like CB-011 could help bridge that gap by offering a readily available treatment option to a broader group of patients.” As previously reported in November 2025, 48 patients have been treated in the dose escalation portion of the company’s CaMMouflage phase 1 clinical trial. The 450x106 CAR-T cell dose was selected as the recommended dose for expansion (RDE). In dose escalation, 12 BCMA-naïve patients were treated with the RDE; efficacy outcomes from this cohort included a 92% (11/12) overall response rate (ORR), 75% (9/12) ≥ complete response (CR) rate, and 91% (10/11 evaluable) minimal residual disease (MRD) negativity as of a September 24, 2025, data cutoff. CB-011 has demonstrated a manageable safety profile, with no cases of graft-versus-host disease, immune effector cell-associated enterocolitis, parkinsonism, or cranial nerve palsies observed at any dose level. Treatment emergent adver...