Pharma powerhouse CARsgen Therapeutics Holdings has announced a major step forward in its fight against hard-to-treat blood cancers. 

The pharma company has submitted two Investigational New Drug (IND) applications to China’s National Medical Products Administration (NMPA) for its allogeneic BCMA-targeted CAR-T therapy, CT0596.  

The applications aim to launch Phase Ib/II trials for relapsed/refractory multiple myeloma (R/R MM) and primary plasma cell leukemia (pPCL), respectively. 

CT0596, developed on CARsgen’s proprietary THANK-u Plus platform, uses advanced gene editing to reduce the risk of graft-versus-host disease (GvHD) and host immune rejection. Genes including NKG2A, TRAC, and B2M are knocked out, while additional edits block natural killer (NK) cell-mediated rejection—enhancing both safety and efficacy. 

Early clinical data from investigator-initiated trials (IIT) in China show promising results.  

“As of August 31, 2025, all 8 patients with R/R MM who received CT0596 infusion were evaluable for efficacy, with a median follow-up of 4.14 months. Six patients achieved a partial response (PR) or better: 3 achieved complete response/stringent complete response (CR/sCR) (all received full-dose lymphodepletion), 1 achieved very good partial response (VGPR), and 2 achieved PR.  

"Four patients experienced Grade 1 cytokine release syndrome (CRS), with no Grade 2 or higher CRS observed. No immune effector cell-associated neurotoxicity syndrome (ICANS), GvHD, dose-limiting toxicities, treatment discontinuations, or deaths were reported.” 

Preliminary results in relapsed/refractory pPCL are equally encouraging. “Two heavily pretreated pPCL patients with high disease burden and rapid progression both achieved sCR after receiving CT0596 treatment.”