Off-the-Shelf CAR-T Therapy CB-011 Shows High Response Rates in Multiple Myeloma; Broader CAR-T Advances Presented at 2026 Tandem Meetings

CB-011, the first allogeneic anti-BCMA chimeric antigen receptor (CAR) T-cell therapy incorporating immune cloaking, achieved a high overall response rate in heavily pretreated patients with relapsed or refractory multiple myeloma in the phase I CaMMouflage trial. Updated data presented at the 2026 Tandem Meetings Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR showed an approximately 92% overall response rate with strong complete response rates and no graft-versus-host disease (GVHD).

CB-011 is manufactured using Cas12a CRISPR hybrid RNA-DNA genome-editing technology. Its immune-cloaking strategy includes beta-2 microglobulin knockout to reduce T-cell–mediated rejection and insertion of a beta-2 microglobulin HLA-E fusion peptide to blunt natural killer cell–mediated rejection. As the HLA class I surface proteins are removed, CB-011 essentially mimics a 6/12 HLA match. The therapy uses healthy donors, and each manufacturing batch can produce multiple doses, eliminating wait time between eligibility determination and lymphodepletion.

In dose-escalation results from the CaMMouflage trial, 48 patients were treated. Among the 35 patients who received the selected lymphodepletion regimen—cyclophosphamide at 500 mg/m² and fludarabine at 30 mg/m² daily for 3 days, followed by CB-011 at 450 million CAR T cells—30 were BCMA-naive. The overall response rate in these BCMA-naive patients was 70%, with 43% achieving a complete response or stringent complete response. The rate of very good partial response or better was 50%, and 57% of 21 evaluable patients achieved minimal residual disease (MRD) negativity. No dose-limiting toxicities were reported.

Study investigators noted deep and durable responses with a very manageable safety profile and rapid recovery of the native immune system. A survey conducted during the trial found that the off-the-shelf nature of the product, the lack of manufacturing wait time, and the deep and promising responses were the top three reasons for rapid patient accrual.

The phase I CaMMouflage study enrolled patients with multiple myeloma who had received at least three prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Patients who had received prior CAR T-cell therapy and/or a BCMA-targeted agent within the previous 3 months were ineligible.

Broader CAR-T Landscape at Tandem Meetings

Multiple next-generation CAR-T therapies were highlighted at the 2026 Tandem Meetings across hematologic cancers.

In aggressive B-cell lymphoma, EB-103, a CD19 TCR-mimetic CAR-T construct, achieved 100% overall and complete response rates at its recommended dose in a phase 1 trial (NCT06343311), with predominantly grade 1/2 cytokine release syndrome (CRS) and no high-grade neurotoxicity.

Dual-target CD19/CD20 CAR T-cell therapy KITE-753 showed high complete response rates and no grade 3 or higher CRS or immune effector cell-associated neurotoxicity syndrome (ICANS) in a phase 1 study (NCT04989803), with a rapid manufacturing timeline of approximately 13 days.

In chronic lymphocytic leukemia (CLL), the LV20.19 CAR-T construct delivered deep responses in heavily pretreated patients, with high MRD negativity and long progression-free survival in a phase 1/2 trial (NCT04186520). However, a new inflammatory syndrome, immune effector cell–associated hemophagocytic syndrome–like syndrome (IEC-HS), was observed.

Beyond CAR-T, autologous repolarized macrophage therapy RB-1355 demonstrated systemic activity in heavily pretreated lymphomas with rapid manufacturing and minimal toxicity in a phase 1 trial.

NXC-201 Reports 95% Complete Response Rate in AL Amyloidosis

In a separate development, Immix Biopharma announced that its sterically-optimized BCMA-targeted CAR-T cell therapy NXC-201 achieved a 95% complete response rate (19 of 20 patients) in the NEXICART-2 phase 2 trial for relapsed/refractory AL amyloidosis. All four MRD-negative patients previously presented at ASH 2025 have since converted to complete response, and all complete responses were reached within one year of follow-up post-dosing. No relapses have been observed to date for patients who have reached complete response. The median number of prior lines of therapy was four.

NXC-201 has been awarded Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy designation by the U.S. FDA, and Orphan Drug Designation by the FDA and the European Medicines Agency. The NEXICART-2 trial is a 45-patient study with a registrational design. The next update is expected in late September 2026, with 1-year follow-up data anticipated by end of March 2027 to support a Biologics License Application submission and commercial launch.