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SC-U02 is an allogeneic, non-viral genome-targeted chimeric antigen receptor T-cell (CAR-T) therapy developed by Sunnycell Therapeutics for the treatment of B-cell malignancies, specifically relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL). Engineered using CRISPR-Cas9, SC-U02 features a knockout of the T-cell receptor (TCR) gene to prevent graft-versus-host disease (GvHD) and a knockout of the beta-2-microglobulin (B2M) gene to prevent host-versus-graft rejection. To protect the cells from natural killer (NK) cell-mediated rejection caused by the lack of HLA class I expression, an artificial B2M-HLA-E fusion gene is inserted along with the anti-CD19 CAR gene directly into the TRAC locus. This design allows SC-U02 to persist in vivo without requiring CD52-targeted lymphodepletion.
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