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LASMECABTAGENE TIMGEDLEUCEL

UNII:5GP3N2YX96
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:LASMECABTAGENE TIMGEDLEUCEL
GSRS Full Record:
5GP3N2YX96
i

  • LASMECABTAGENE TIMGEDLEUCEL [USAN]
  • Lasmecabtagene Timgedleucel
  • UCART-22
  • UCART22
  • UCART22 is defined as allogeneic, non-alloreactive T-cells, transduced with a selfinactivating (SIN) recombinant lentiviral vector to express an anti-CD22 CAR and RQR8, and genetically edited with TALEN® (Transcription Activator Like Effector Nucleases) to disrupt the CD52 and the T cell receptor alpha constant (TRAC) genes
  • allogeneic T lymphocytes obtained from peripheral blood mononuclear cells by leukapheresis of healthy donors, transduced with a selfinactivating, non-replicating lentiviral vector encoding a depletion ligand and a chimeric antigen receptor (CAR) targeting CD22. The cells are also genetically modified using transcription activator-like (TAL) effector nucleases, which are transiently delivered into the cell as mRNAs to disrupt the T cell receptor alpha constant (TRAC) and CD52 genes. The expressed transgene comprises the RQR8 depletion ligand, a T2A ribosomal skip motif and an anti-CD22 CAR, under control of the human elongation factor-1 alpha (EF-1α) promoter. The RQR8 ligand is a 136 amino acid artificial cell surface protein that combines the antibody-binding epitopes from human CD20 (R) and human CD34 (Q) in the order of R, Q and R, followed by a CD8α hinge and transmembrane domain. The CAR comprises an anti-CD22 single chain variable fragment (scFv), a CD8α hinge and transmembrane domain, and 4-1BB (CD137) and CD3ζ (CD247) intracellular costimulatory domains. The construct is flanked by 5' and 3' long terminal repeats (LTRs) and also contains a ψ packaging signal, a truncated gag, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and a mutated Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The vector is pseudotyped with vesicular stomatitis virus (VSV) glycoprotein G. Proposed INN: List 131 WHO Drug Information, Vol. 38, No. 2, 2024 362 The leukapheresis material is activated by CD3 and CD28 agonists and transduced with the vector. Residual TCRαβ+ lymphocytes are removed by magnetic depletion, then cultured in media containing interleukin 2 (IL-2). The suspension consists primarily of T lymphocytes with >30% CAR+ and ≤3.0% TCRαβ+ T lymphocytes. The cells are cytotoxic and secrete pro-inflammatory cytokines in response to CD22-expressing cells.
  • lasmecabtagene timgedleucel [INN]




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