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CARsgen Therapeutics Reveals Promising Results for CT0596

In recent developments, CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK) has unveiled preliminary clinical data for its innovative therapy, CT0596, a BCMA-targeted CAR-T treatment. This therapy, designed to treat patients with relapsed or refractory multiple myeloma and plasma cell leukemia, has showcased a favorable safety and efficacy profile, raising hopes for those suffering from these challenging conditions.

Overview of CT0596

CT0596 was developed using CARsgen's proprietary THANK-u Plus™ platform, which enhances the performance of allogeneic CAR-T therapies. This technology focuses on maximizing the therapeutic efficacy of CAR-T treatments, particularly in patients with varying expressions of NKG2A, a receptor that can influence immune responses. The ongoing exploratory study aims to evaluate the safety and early efficacy of CT0596, providing essential insights into its potential impact on patient care.

Preliminary Clinical Findings

As of May 6, 2025, the clinical trial included eight patients who had undergone at least three prior lines of therapy for their conditions. After treatment with CT0596, following a lymphodepletion regimen, notable findings emerged:

• - No dose-limiting toxicities (DLTs) or severe adverse events were reported, highlighting the therapy's tolerability.
• - Among the five patients evaluated at the four-week mark, 60% achieved stringent complete response (sCR) or complete response (CR), while 80% showed minimal residual disease negativity in the bone marrow.
• - Early assessments at day 14 indicated substantial reductions in measurable lesions by 92% and 65% for two patients, respectively.
• - The promising data suggests that patients who responded positively have continued to do so over the four-month follow-up period.

The absence of treatment discontinuation due to adverse effects further underscores the safety of CT0596, marking it as a potential option for patients with limited treatment alternatives.

Implications for Future Research

Given these encouraging early results, CARsgen is keen to extend the application of CT0596 beyond relapsed/refractory multiple myeloma to address other plasma cell malignancies and autoimmune diseases that stem from autoreactive plasma cells. The company is planning to present more detailed data in upcoming scientific conferences, reflecting its commitment to transparency and ongoing research in the field.

In addition, CARsgen aims to submit an Investigational New Drug (IND) application for CT0596 later in 2025, marking a significant step toward making this potential treatment available to patients.

About the Technology

The THANK-u Plus™ platform represents an evolution in CAR-T technology, designed specifically to facilitate robust immune responses regardless of individual cellular characteristics. Data suggests that this platform not only improves the success of CAR-T cell expansion but also enhances antitumor activity, particularly in cooperation with natural killer (NK) cells. This adaptability signifies a promising frontier in the development of diverse CAR-T therapies with the potential to improve patient outcomes.

CARsgen's Mission

CARsgen Therapeutics is dedicated to addressing unmet clinical needs in the realm of hematologic malignancies, solid tumors, and autoimmune diseases through innovative CAR T-cell therapies. The firm has established comprehensive capabilities in CAR T-cell research, development, and commercialization, aiming to yield therapies that are safer, more effective, and more accessible to patients worldwide. Their vision is to be recognized as a global leader in biopharmaceutical innovation, ultimately offering curative options for a variety of cancers and diseases.

For further information about CARsgen, please visit CARsgen Therapeutics.