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

CARsgen Unveils Results for Allogeneic CAR-T Therapy CT0596 at ASH 2025

Date: December 7, 2025
Location: Shanghai, China

At the 67th Annual Meeting of the American Society of Hematology (ASH) held in 2025, CARsgen Therapeutics Holdings Limited proudly presented significant data from their latest clinical trial concerning the allogeneic BCMA-targeted CAR T-cell therapy, defined as CT0596. This therapy is particularly designed for addressing relapsed and refractory multiple myeloma (R/R MM), a type of cancer that has shown resistance to existing treatment avenues.

The presentation, which featured the poster titled "A First-in-Human Study of CT0596, an Allogeneic CAR T-Cell Therapy Targeting BCMA, in Patients with Relapsed/Refractory Multiple Myeloma", encapsulated the findings of a clinical trial aimed at evaluating the safety and efficacy of CT0596 in patients who had encountered multiple lines of prior therapies. The trial enrolled eight patients during the dose-escalation phase, and these individuals had undergone a median of 4.5 previous treatments, with some having experienced up to nine.

A noteworthy facet of the trial is that it aimed to assimilate a diverse patient demographic, with no restrictions pertaining to NKG2A expression levels—a consideration that could expand treatment applicability across various patient profiles. Regarding the lymphodepletion protocol, six patients underwent full-dose lymphodepletion utilizing fludarabine and cyclophosphamide, while the remaining two were on a reduced regimen.

The administration of CT0596 varied across dosage levels, with distributions of 1.5×10⁸, 3×10⁸, and 4.5×10⁸ CAR-T cells, wherein one participant received two infusions. By the data cut-off on August 31, 2025, all eight patients had reached meaningful points of evaluation, with a median follow-up duration soaring to 4.14 months.

Of particular interest were the results of the efficacy assessment. Out of the eight treated patients, six effectively achieved at least a partial response (PR), with three individuals reaching either a complete response (CR) or stringent complete response (sCR). Most can be credited to the subgroup receiving the full-dose lymphodepletion, as five patients from this cohort achieved favorable outcomes. Remarkably, six patients in this group achieved minimal residual disease (MRD) negativity by the fourth week post-treatment, demonstrating promising results in killing off cancerous cells.

Continuing the success narrative, Patient 01 maintained a sustained sCR, while Patient 04 witnessed a PR along with the resolution of extramedullary disease after the second CT0596 infusion. Patient responses also indicated a significant CAR-T cell expansion across the board.

The safety profile of CT0596 appears to be manageable, highlighted by only four cases of Grade 1 cytokine release syndrome (CRS). Evaluations confirmed that no instances of Grade 2 or higher CRS, immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GVHD) were observed. Moreover, the trial reported no dose-limiting toxicities, discontinuations of treatment, or fatalities.

Currently, the study remains in the exploratory phase regarding dosage, with the lymphodepletion regimen finalized but inquiries for higher cell doses still ongoing to identify the recommended dosage. Looking ahead to 2026, CARsgen aims to initiate a Phase 1b registrational study for CT0596, a critical next step in validating this promising treatment.

About CT0596
CT0596 employs CARsgen's proprietary THANK-u Plus™ platform, showcasing its innovative approach to tackling hematologic malignancies. Preliminary results suggest this therapy not only boasts an encouraging efficacy signal but also demonstrates favorable tolerability. Future investigations are planned to evaluate its impacts on additional plasma cell malignancies and autoimmune disorders stemming from autoreactive plasma cells. Moreover, CARsgen is set to file an Investigational New Drug (IND) application in the latter half of 2025, paving the way for CT0596's entry into subsequent phases of clinical exploration.

About CARsgen Therapeutics Holdings Limited
CARsgen Therapeutics is at the forefront of biopharmaceutical innovation, developing advanced CAR T-cell therapies targeted at unmet clinical needs across hematologic malignancies, solid tumors, and autoimmune diseases. With a comprehensive end-to-end framework for CAR T-cell development, CARsgen engineers novel technologies and curates a robust product pipeline aimed at improving safety, enhancing efficacy, and making treatment more accessible.**