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SOFICABTAGENE GELEUCEL

UNII:Z75Y8V4FFA
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:SOFICABTAGENE GELEUCEL
GSRS Full Record:
Z75Y8V4FFA
i

  • Soficabtagene Geleucel
  • allogeneic CD4+ and CD8+ T lymphocytes derived from leukapheresis material of healthy donors, genetically modified using CRISPR/Cas9 gene editing to knockout the expression of endogenous CD7 and TRAC using inactivating, non-replicating lentivirus vector encoding an anti-CD7 chimeric antigen receptor (CAR) comprising a CD8 leader sequence, anti-CD7 single-chain variable fragment (scFv)(clone TH69), CD8 hinge, CD28 transmembrane domain, 4-1BB costimulatory domain and CD3ζ intracellular signalling domain, under control of the elongation factor 1 alpha (EF-1α) promoter. The transgene also encodes a truncated human CD34 extracellular domain separated from the CAR by a 2A peptide derived from porcine teschovirus-1. The CRISPR/Cas9 and gRNAs are introduced as a ribonucleoprotein (RNP) complex by electroporation. The lentivirus vector is flanked by 5' and 3' LTRs and also contains a viral packaging sequence ψ, partial gag, a Rev response element (RRE), a central polypurine tract/central termination sequence (cPPT/CTS) and a synthetic identification (ID) sequence. The vector is pseudotyped with VSV G envelope glycoprotein. The leukapheresis material is enriched for CD4+ and CD8+ T lymphocytes by positive immunoselection prior to activation with CD3 and CD28 agonists in growth media containing interleukin 7 (IL-7) and interleukin 15 (IL-15). The cells are gene-edited and then transduced with the lentiviral vector and expanded in growth media containing human AB serum, IL-7, and IL-15. The cell suspension consists of T lymphocytes (≥98.0% CD45+, ≤1.0% CD7+, ≤8.0% CD3+) with greater than 30% of the T lymphocytes expressing the CAR transgene and >70% (CD7) and >85% (TRAC) on-target gene editing. The transduced T lymphocytes demonstrate cytotoxicity against CD7+ Tlymphoblastic target cells
  • soficabtagene geleucel [INN]




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