Drug intelligence / Profile preview

BEAM-201

Development stage
Phase 2
Lead developer
Beam Therapeutics
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Editing → Gene Therapies
Administration
Intravenous
01

Overview

BEAM-201 is an investigational allogeneic, multiplex base-edited anti-CD7 chimeric antigen receptor (CAR) T-cell therapy developed for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LL). The therapy uses healthy donor T cells that are genetically modified ex vivo using base editing to introduce four specific single-base mutations in the TRAC, CD7, CD52, and PDCD1 genes. These edits abolish expression of their respective proteins to enable universal compatibility (allogeneic use), prevent fratricide among CAR-T cells by silencing CD7, block endogenous TCR signaling to avoid graft-versus-host disease via TRAC disruption, confer resistance to anti-CD52 antibodies used during lymphodepletion by disrupting CD52, and enhance antitumor activity by eliminating PDCD1-mediated immune checkpoint inhibition. The edited cells are then transduced with a lentivirus encoding an anti-CD7 CAR construct. This approach aims to provide a potent off-the-shelf cell therapy option for aggressive blood cancers expressing CD7[1][5][6].

Other names
CD7-targeted base-edited CAR-TCD-7-targeted base-edited CAR-TCD 7-targeted base-edited CAR-T
02

Targets

PDCD1 (Programmed cell death protein 1 receptor)TRAC (T cell receptor alpha variable 17)CD52 (CD52 Antigen)CD7 (CD7 antigen)

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