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PERSICABTAGENE LEMGEDLEUCEL

UNII:9C6AT9DSU2
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:PERSICABTAGENE LEMGEDLEUCEL
GSRS Full Record:
9C6AT9DSU2
i

  • A hypoimmune, genetically modified, allogeneic CAR T cell therapy targeting CD19. Healthy donor T cells are transduced with a lentiviral vector to incorporate gene sequences to overexpress CD47 and express a CD19-directed CAR. The donor T cells are also gene-edited with CRISPR/Cas12b to disrupt the TRAC, B2M, and CIITA genes
  • PERSICABTAGENE LEMGEDLEUCEL [USAN]
  • Persicabtagene Lemgedleucel
  • SC-291
  • SC291
  • 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 CD19-directed chimeric antigen receptor (CAR). The cells have also been gene-edited using CRISPR/Cas12b (clustered regularly interspaced short palindromic repeats/ CRISPRassociated protein 12b) The cells have also been gene-edited using CRISPR/Cas12b (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 12b) 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, and a mutant Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The CD47-CD19 transgene comprises a codon optimised CD47 coding region, a furin cleavage sequence, a Thosea asigna virus 2A (T2A) ribosomal skip sequence, a CD8α signal peptide, and an anti-CD19 single chain variable fragment (clone FMC63), fused to a CD8α hinge, CD8α transmembrane, 4-1BB co-stimulatory and CD3ζ signaling domain and is under control of the human elongation factor-1 alpha 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 (≥90%) with greater than 35% of the T lymphocytes expressing the CAR transgene, and with ≥70% B2M disrupted, and ≥70% CIITA disrupted cells.
  • persicabtagene lemgedleucel [INN]




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