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RUNITRESGENE LEUCEL

UNII:C6LF4ED2YD
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:RUNITRESGENE LEUCEL
GSRS Full Record:
C6LF4ED2YD
i

  • Runitresgene Leucel
  • allogeneic T lymphocytes derived from peripheral blood mononuclear cells (PBMCs) of a haploidentical hematopoietic cell transplantation (HCT) donor that is negative for the HA-2 genotype and/or negative for HLA A*02, engineered by transposon/transposase-mediated gene delivery to express a T cell receptor (TCR) that is specific for a 9-amino acid peptide (YIGEVLVSV) termed HA-2, derived from myosin 1G (MYO1G) and presented on HLA-A*02:01. The TCR comprises α and β subunits separated by a P2A self-cleaving peptide. The transgene also encodes a codon-optimised CD8 co-receptor consisting of α and β chains separated by a P2A self-cleaving peptide, with the CD8 co-receptor separated from the TCR by an additional P2A self-cleaving peptide. Each P2A sequence is preceded by a furin cleavage site. A 16 amino acid epitope encoding a portion of human CD34 is incorporated into the Nterminus of the CD8α chain. The transgene is under the control of a murine stem cell virus (MSCV) promoter, a Woodchuck hepatitis virus post-transcriptional response element and a simian virus 40 (SV40) polyadenylation (polyA) signal. The transposase (Armyworm transposase) is introduced in form of an mRNA that comprises an anti-reverse cap analogue, a Xenopus globin 5' untranslated region (UTR), a Kozak sequence, the transposase open reading frame, and a Xenopus globin 3' UTR. The transgene is introduced via a nanoplasmid DNA and contains inverted terminal repeats (ITRs). The leukapheresis material is electroporated with mRNA transposase as well as the nanoplasmid transposon followed by activation with CD2, CD3 and CD28 agonists. The cells are grown in serum free media supplemented with interleukin 2 (IL-2), interleukin 7 (IL-7) and interleukin 15 (IL-15). The T lymphocytes (≥95%) are positive for the transgene (≥80% TCR+), and release interferon -gamma (IFN-γ) in response
  • runitresgene leucel [INN]




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