On December 28, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported that it has submitted two separate Investigational New Drug (IND) applications to the National Medical Products Administration (NMPA) for its allogeneic BCMA-targeted CAR-T cell therapy product, CT0596. The applications seek to initiate two corresponding Phase Ib/Ⅱ clinical trials for the treatment of relapsed/refractory multiple myeloma (R/R MM) and primary plasma cell leukemia (pPCL), respectively.
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CT0596 is an allogeneic CAR T-cell therapy targeting BCMA, developed based on CARsgen’s proprietary THANK-u Plus platform. Through the knockout of genes such as NKG2A, TRAC, and B2M, CT0596 is designed to reduce the risk of graft-versus-host disease (GvHD) and host immune rejection. Additional gene editing further blocks host natural killer (NK) cell-mediated rejection, thereby aiming to enhance the product’s efficacy and safety profile.
Investigator-initiated trials (IIT) for CT0596 have already been conducted in China to explore its clinical potential in treating R/R MM and pPCL. Data from the first-in-human study, presented at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, demonstrated a favorable safety profile and encouraging efficacy signals for CT0596. As of August 31, 2025, all 8 patients with R/R MM who received CT0596 infusion were evaluable for efficacy, with a median follow-up of 4.14 months. Six patients achieved a partial response (PR) or better: 3 achieved complete response/stringent complete response (CR/sCR) (all received full-dose lymphodepletion), 1 achieved very good partial response (VGPR), and 2 achieved PR. Four patients experienced Grade 1 cytokine release syndrome (CRS), with no Grade 2 or higher CRS observed. No immune effector cell-associated neurotoxicity syndrome (ICANS), GvHD, dose-limiting toxicities, treatment discontinuations, or deaths were reported.
Previously, the company also disclosed preliminary clinical data for CT0596 in relapsed/refractory pPCL. Two heavily pretreated pPCL patients with high disease burden and rapid progression both achieved sCR after receiving CT0596 treatment.
The IND applications mark the commencement of the registration clinical development phase for CT0596, which holds the potential to offer a new treatment option for patients with R/R MM and pPCL.
About CT0596
CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen’s proprietary THANK-u Plus platform. It is currently being evaluated in investigator-initiated trials for relapsed/refractory multiple myeloma (R/R MM) or primary plasma cell leukemia (pPCL). CT0596 demonstrated preliminary favorable tolerability and encouraging efficacy signals. Further investigation is planned in additional plasma cell malignancies and autoimmune diseases mediated by plasma cells.
(Press release, Carsgen Therapeutics, DEC 28, 2025, View Source [SID1234661634])