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LUMOCABTAGENE GELEUCEL

UNII:2JR5UJR7B3
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:LUMOCABTAGENE GELEUCEL
GSRS Full Record:
2JR5UJR7B3
i

  • Lumocabtagene Geleucel
  • allogeneic CD4+/CD8+ T lymphocytes obtained from peripheral blood mononuclear cells by leukapheresis of healthy donors, transduced with a lentiviral vector to overexpress CD47 and a CD22-directed chimeric antigen receptor (CAR). The cells have also been gene-edited using clustered regularly interspaced short palindromic repeats (CRISPR)-Cas12b nuclease introduced as mRNA in combination with single guide RNAs (sgRNAs) to disrupt the T cell receptor alpha constant (TRAC), beta-2 microglobulin (B2M), and class II major histocompatibility complex transactivator (CIITA) gene loci. The lentivirus vector genome is flanked by 5' and 3' long terminal repeats (LTRs) and contains a human immunodeficiency virus (HIV) packaging signal, HIV gag, HIV envelope, Rev response element (RRE), and central polypurine tract/central termination sequences as well as a mutant Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE). The CD47-CD22 transgene comprises a codon optimised CD47 coding region, a furin cleavage sequence, a Thosea asigna virus 2A (T2A) ribosomal skip sequence, a granulocyte-macrophage colony-stimulating factor receptor (GMCSFR) signal peptide, an anti-CD22 single chain variable fragment (based uponclone m971), fused to a CD8α hinge, CD8α transmembrane, 4-1BB co-stimulatory and CD3ζ signalling domain, and is under control of the human elongation factor 1α promoter (EF-1α). The leukapheresis material is enriched for CD4/CD8 T lymphocytes by positive immunoselection, activated by CD3 and CD28 agonists and subject to gene editing. The cells are then expanded in media supplemented with serum replacement and interleukin 2 (IL-2). The cell suspension consists of T lymphocytes (CD4+ and CD8+; ≥90%) with ≥25% of the T lymphocytes expressing the CAR transgene, and with ≥70% B2M disrupted, and ≥70% CIITA disrupted cells.
  • lumocabtagene geleucel [INN]




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