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ATIDARSAGENE AUTOTEMCEL

UNII:EPP8G99QG4
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:ATIDARSAGENE AUTOTEMCEL
GSRS Full Record:
EPP8G99QG4
i

  • ATIDARSAGENE AUTOTEMCEL [USAN]
  • Atidarsagene Autotemcel
  • Atidarsagene autotemcel [WHO-DD]
  • Autologous CD34+ cells, containing hematopoietic stem cells (HSCs), transduced with a LVV encoding the human arylsulfatase A (ARSA) gene
  • Autologous CD34+ hematopoietic stem and progenitor cells transduced ex vivo with a replication-incompetent lentiviral vector encoding human arylsulfatase A (ARSA). Autologous CD34+ hematopoietic stem and progenitor cells (HSPC), obtained from bone marrow (BM) or mobilized peripheral blood (mPB), transduced ex vivo with a replication-incompetent pseudotyped HIV-I based lentiviral vector encoding the human arylsulfatase A (ARSA) transgene under the control of a human phosphoglycerate kinase (PGK) promoter. The vector is pseudotyped with the vesicular stomatitis vims (VSV) envelope glycoprotein. The vector genome also contains a mutated Woodchuck hepatitis vims post-transcriptional regulatory element (WPRE), a fragment of the HIV-I gag, the HIV-I Rev response element (RRE), the HIV-1 central polypurine tract (cPPT) and a fragment of nef. The CD34+ cell population corresponds to an heterogenous population of stem and progenitor cells, some expressing the CD90 and/or CD133 markers.
  • OTL-200
  • atidarsagene autotemcel [INN]




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