CARsgen's Allogeneic CD38 CAR-T Therapy Begins Dosing in Investigator-Led Trial

SHANGHAI, Jan. 20, 2025 – CARsgen Therapeutics Holdings Limited, a biopharmaceutical firm listed under the stock code 2171.HK, has made significant progress in the field of CAR T-cell therapies. With a focus on treating hematologic malignancies and solid tumors, the company has announced the administration of the first dose of KJ-C2320 in a clinical trial. This trial is investigator-initiated and is taking place in China, marking a pivotal step in the evaluation of KJ-C2320 for patients suffering from relapsed or refractory acute myeloid leukemia.

KJ-C2320 is an allogeneic CAR T-cell therapy that targets CD38, a protein found on the surface of certain cancerous cells. This treatment is being developed using the THANK-uCAR® platform, which aims to enhance the efficacy and safety of CAR T-cell therapies. The THANK-uCAR® framework represents CARsgen's proprietary technology focusing on allogeneic CAR-T cells. The approach involves modifying donor-derived T cells to boost their expansion and persistence, addressing some of the significant challenges inherent in allogeneic therapies.

The THANK-uCAR® technology works by targeting the genetic makeup of T cells to prevent graft versus host disease (GvHD) and host versus graft response (HvGR). By disrupting the genes responsible for T-cell receptor expression and human leukocyte antigen class I (HLA-I), the scientists aim to eliminate adverse immune responses. However, the absence of HLA-I can lead to natural killer (NK) cells attacking these modified T cells, which poses a challenge to treatment durability.

To combat this, CARsgen has engineered the TCR-/B2M- T cells to include a chimeric antigen receptor (CAR) that identifies and counters NKG2A-positive NK cells. This strategic adaptation allows the modified CAR T cells to resist attacks from the patient's own NK cells, promoting their survival and efficacy. Clinical trials have shown that the BCMA CAR-T therapy, developed through the THANK-uCAR® platform, can expand within patients achieving complete responses. This indicates that the therapy's effectiveness is on par with autologous CAR T-cell therapies, while also demonstrating a controllable safety profile and promising results.

CARsgen Therapeutics Holdings Limited operates extensively both in China and the United States, establishing itself as a leader in the field of CAR T-cell therapy research and development. The company has built a robust platform that spans the entire development process, from target discovery to clinical trials and large-scale production. They are actively addressing the current limitations of CAR T-cell therapies, such as safety concerns, the challenge of treating solid tumors effectively, and the high cost of treatment.

CARsgen’s overarching mission is to establish itself as a global leader in biopharmaceuticals by providing innovative cell therapies that are not only effective but also cost-efficient. Their vision extends to making cancer a curable disease, ultimately transforming the landscape of cancer treatment worldwide. With the ongoing trials and the strategic use of their THANK-uCAR® platform, CARsgen continues to make strides towards this ambitious goal, offering hope for improved cancer therapies on a global scale.