#China #Shanghai #CAR-T therapy #CARsgen Therapeutics #CT0596

CARsgen Therapeutics Submits IND Applications for CT0596

In a pivotal move for oncology treatment, CARsgen Therapeutics Holdings Limited (stock code: 2171.HK) has recently filed two Investigational New Drug (IND) applications with China's National Medical Products Administration (NMPA). These applications are aimed at advancing their allogeneic BCMA CAR-T cell therapy product, CT0596, into clinical trials for treating relapsed/refractory multiple myeloma (R/R MM) and primary plasma cell leukemia (pPCL). This bold step is seen as a significant leap forward in the development of innovative cancer therapies, specifically designed to target stubborn blood cancers that have proven resistant to existing treatments.

The Significance of CT0596

CT0596 represents a novel approach to CAR-T cell therapy, utilizing CARsgen’s proprietary THANK-u Plus™ platform. This innovative therapy is genetically engineered to express a CAR that targets B-cell maturation antigen (BCMA), a key protein involved in the survival of malignant plasma cells. The engineering process includes knocking out genes such as NKG2A, TRAC, and B2M, which significantly reduces the chances of graft-versus-host disease (GvHD) and immune rejection by the host. In addition, further genetic modifications have been implemented to inhibit natural killer (NK) cell responses, promising improved efficacy and a reduced risk of complications.

Clinical Trials and Data

The clinical potential of CT0596 has been explored through investigator-initiated trials in China. As highlighted in findings presented at the 2025 American Society of Hematology (ASH) Annual Meeting, the initial results are promising. In the first human studies, all eight patients suffering from R/R MM who received the CT0596 infusion were analyzed for efficacy after a median follow-up of 4.14 months. Remarkably, six patients demonstrated partial responses, with three reaching complete response or stringent complete response status. The data suggest a favorable safety profile; only four patients experienced Grade 1 cytokine release syndrome (CRS), with no serious adverse effects reported, such as immune effector cell-associated neurotoxicity syndrome (ICANS) or treatment discontinuations.

Moreover, preliminary data from those with R/R pPCL indicates that two heavily pre-treated patients achieved stringent complete responses after receiving CT0596, underscoring its potential effectiveness in difficult-to-treat populations.

A New Chapter in Cancer Treatment

The submission of these IND applications marks an important milestone in CT0596’s registration development phase. This product could provide new hope for patients grappling with R/R MM and pPCL—diseases that have limited treatment options and poor prognosis. By targeting BCMA effectively while minimizing potential side effects, CARsgen positions CT0596 as a leading candidate in the realm of CAR-T therapies.

About CARsgen Therapeutics

Founded with the mission of addressing unmet clinical needs, CARsgen Therapeutics specializes in CAR T-cell therapy innovations to treat hematologic malignancies and solid tumors as well as autoimmune diseases. The company has developed comprehensive capabilities encompassing target discovery, preclinical research, and clinical development, alongside scalable production methods for its CAR T-cell products. With a robust pipeline to tackle existing challenges within CAR-T therapy, CARsgen aims to become a global leader in biopharmaceuticals, potentially implementing novel therapies that can pave the way for curing cancer and other severe diseases.

Through its continuous efforts and groundbreaking products, CARsgen Therapeutics demonstrates an unwavering commitment to advancing treatment options and improving outcomes for patients worldwide. As it moves forward with CT0596, the biopharmaceutical community watches closely for the therapeutic potential that this innovation may unveil.