Рапкабтаген 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 T cell chimeric antigen receptor targeting CD19. The expressed transgene comprises a CD8α leader sequence, a murine anti-CD19 single chain variable fragment (scFv) derived from the mouse hybridoma FMC63, a CD8α hinge and transmembrane region, and a 4- 1BB (CD137) and CD3ζ (TCRζ) signaling domain 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, activated by CD3 and CD28 agonists and transduced with the vector. Without further cell propagation, the T cells are washed, formulated for infusion, and cryopreserved. The substance is composed of ≥80% T cells and ≤1% B cells, with a mixture of transgene positive (>3.4%) and negative T cells. The CD4+ and CD8+ naïve T cell subsets (CD45RA+CCR7+) present in the leukapheresis material are largely retained.

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