PD-1 knockdown BCMA CAR-T cells

PD-1 knockdown BCMA CAR-T cells are a genetically engineered T-cell immunotherapy designed to treat relapsed or refractory multiple myeloma. This therapy incorporates a **short hairpin RNA cassette** targeting PD-1 (programmed cell death protein 1) into a BCMA-targeting chimeric antigen receptor construct with an **OX-40 costimulatory domain**. The engineered cells are designed to overcome T-cell exhaustion by reducing PD-1 expression, which is a major inhibitory checkpoint receptor that impairs CAR-T cell function in the immunosuppressive tumor microenvironment. By knocking down PD-1, these cells demonstrate **reduced T-cell exhaustion**, **increased memory T-cell formation**, and **superior antitumor activity** compared to conventional BCMA CAR-T cells, particularly against tumors expressing PD-L1. In preclinical studies, PD-1KD BCMA CAR-T cells showed enhanced cytotoxicity, improved cytokine production, and better proliferation when confronted with PD-L1-overexpressing tumor cells. The therapy has shown promising results in phase 1 clinical trials with an **85.7% overall response rate** in relapsed or refractory multiple myeloma patients, with manageable safety profile including mild to moderate cytokine release syndrome and no neurological toxicity.

Other names

PD-1KD BCMA CAR-T cellsPD1KD BCMA CAR-T cellsPD 1KD BCMA CAR-T cellsPD-1 KD BCMA CAR-TPD1 KD BCMA CAR-TPD 1 KD BCMA CAR-T