SHANGHAI, Dec. 28, 2025 — CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a firm concentrating on the development of innovative CAR T-cell therapies, declared that it has sent 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 aim to start 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.
CT0596 is an allogeneic CAR T-cell therapy that targets BCMA and is developed based on CARsgen’s proprietary THANK-u Plus
platform. By knocking out genes such as NKG2A, TRAC, and B2M, CT0596 is designed to lower the risk of graft-versus-host disease (GvHD) and host immune rejection. Additional gene editing further blocks host natural killer (NK) cell-mediated rejection, thus aiming to improve the product’s efficacy and safety profile.
Investigator-initiated trials (IIT) for CT0596 have already been carried out 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) Annual Meeting, showed 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 assessable 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), and no Grade 2 or higher CRS was observed. No immune effector cell – associated neurotoxicity syndrome (ICANS), GvHD, dose – limiting toxicities, treatment discontinuations, or deaths were reported.
Previously, the company also revealed 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 start of the registration clinical development phase for CT0596, which has 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 for additional plasma cell malignancies and autoimmune diseases mediated by plasma cells.
About CARsgen Therapeutics Holdings Limited
CARsgen is a biopharmaceutical company that focuses on developing innovative CAR T – cell therapies to meet the unmet clinical needs, including but not limited to hematologic malignancies, solid tumors, and autoimmune diseases. CARsgen has established end – to – end capabilities for CAR T – cell research and development, covering target discovery, preclinical research, product clinical development, and commercial – scale production. CARsgen has developed novel in – house technologies and a product pipeline with global rights to tackle the challenges faced by existing CAR T – cell therapies. Efforts include improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen’s mission is to become a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases curable.
Forward – looking Statements
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SOURCE CARsgen Therapeutics