Пликсакабтаген аутолейцел

Rec. INN (наименование, зарегистрированное ВОЗ)

autologous T cells obtained from peripheral blood mononuclear cells by leukapheresis, transduced with a self-inactivating, non-replicating lentiviral vector, encoding a chimeric antigen receptor (CAR) targeting CD19. The expressed transgene comprises a granulocyte-macrophage colony stimulating factor receptor subunit alpha (CSF2RA) signal peptide, a murine anti-CD19 single chain variable fragment (scFv) derived from mouse hybridoma FMC63, consisting of a variable light chain (VL), a 15 amino acid flexible glycine/serine spacer (G4S)3, and a variable heavy-chain (VH), followed by a short linker sequence and the invariant human CD3ε TCR subunit coding sequence, and is under control of the elongation factor 1 alpha (EF1α) promoter. The construct is flanked by 5' and 3' long terminal repeats (LTRs) and also contains a ψ packaging signal, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and an optimized Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE).
The leukapheresis material is enriched for CD4/CD8 T cells by positive immunoselection and then activated and expanded through CD3 and CD28 receptor stimulation.бикЮFollowing activation, the T cells are transduced with the vector and expanded in media supplemented with human male AB serum and cytokines. The cells comprise of >80% CD3+ cells, >70% viable total cells, >5% viable CD3+/transgene+ cells and a mixture of CD4+ and CD8+ cells.

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