CARsgen Therapeutics Holdings Limited announced that it has submitted two separate Investigational New Drug (IND) applications to China’s National Medical Products Administration for its allogeneic BCMA-targeted CAR T-cell therapy, CT0596. The INDs are intended to support two phase Ib clinical trials for the treatment of relapsed or refractory multiple myeloma and primary plasma cell leukaemia.

CT0596 is an allogeneic CAR T-cell therapy developed using CARsgen’s proprietary THANK-u Plus platform. The therapy incorporates multiple gene edits, including knockout of NKG2A, TRAC and B2M, to reduce the risks of graft-versus-host disease and host immune rejection. Additional genetic modifications are designed to inhibit natural killer cell-mediated rejection, with the aim of improving both safety and therapeutic durability.

Investigator-initiated trials evaluating CT0596 have already been conducted in China for relapsed or refractory multiple myeloma and primary plasma cell leukaemia. First-in-human data presented at the 2025 American Society of Hematology Annual Meeting showed a favourable safety profile and early signs of efficacy. As of August 31, 2025, all eight evaluable patients with relapsed or refractory multiple myeloma achieved encouraging clinical responses, with six patients attaining partial response or better, including three complete or stringent complete responses. Cytokine release syndrome was limited to Grade 1 events, with no higher-grade CRS, neurotoxicity, graft-versus-host disease, dose-limiting toxicities or treatment-related deaths reported.

In addition, preliminary clinical data in relapsed or refractory primary plasma cell leukaemia showed that two heavily pretreated patients achieved stringent complete responses following CT0596 treatment.

The submission of the two IND applications marks the start of the registration-directed clinical development phase for CT0596. CARsgen said the programme has the potential to provide a new treatment option for patients with plasma cell malignancies who have limited therapeutic alternatives.

CT0596 continues to be explored across additional plasma cell-driven malignancies and autoimmune diseases. CARsgen is focused on advancing innovative CAR T-cell therapies to address unmet needs in haematological cancers, solid tumours and autoimmune disorders.