CARsgen Submits Dual IND Applications for Allogeneic CAR-T Therapy CT0596 Targeting Multiple Myeloma and Plasma Cell Leukemia

SHANGHAI —December 28, 2025— CARsgen Therapeutics Holdings Limited has officially submitted two Investigational New Drug (IND) applications to China’s National Medical Products Administration (NMPA) for its next-generation allogeneic CAR T-cell therapy, CT0596. The filings aim to initiate Phase Ib/II clinical trials for two aggressive blood cancers: relapsed/refractory multiple myeloma (R/R MM) and primary plasma cell leukemia (pPCL).

The THANK-u Plus™ Platform: Solving the Allogeneic Challenge

Unlike traditional autologous therapies that use a patient’s own cells, CT0596 is an “off-the-shelf” (allogeneic) product derived from healthy donors. To overcome the biological barriers of universal transplantation, CARsgen utilized its proprietary THANK-u Plus™ platform, which employs precise gene editing to enhance safety and persistence:

• Preventing GvHD: The TRAC gene is knocked out to eliminate the risk of graft-versus-host disease.

• Evading Rejection: Knockouts of B2M and NKG2A help the cells hide from the host’s immune system.

• NK Cell Shielding: Additional edits block host Natural Killer (NK) cell-mediated rejection, a common failure point for allogeneic products.

Encouraging Clinical Signals

Preliminary data from Investigator-Initiated Trials (IIT), presented at the 2025 American Society of Hematology (ASH) meeting, show significant promise for the BCMA-targeted therapy:

• R/R Multiple Myeloma: In a study of 8 patients, 75% (6/8) achieved a partial response or better. Notably, 3 patients achieved a complete response (CR) or stringent complete response (sCR).

• Plasma Cell Leukemia: Two patients with pPCL—an extremely aggressive and hard-to-treat cancer—both achieved a stringent complete response (sCR) following treatment.

• Safety Profile: The therapy demonstrated a favorable safety profile with no reported cases of neurotoxicity (ICANS), GvHD, or Grade 2+ cytokine release syndrome (CRS).

Future Expansion into Autoimmune Diseases

The submission marks the beginning of the formal registration phase for CT0596. Beyond plasma cell malignancies, CARsgen plans to investigate the therapy’s potential in treating autoimmune diseases mediated by plasma cells, leveraging the product’s rapid “off-the-shelf” availability to provide immediate care for patients with high disease burdens.