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ATIDARSAGENE AUTOTEMCEL

UNII:EPP8G99QG4
Formula:
Preferred Substance Name:ATIDARSAGENE AUTOTEMCEL

  • ATIDARSAGENE AUTOTEMCEL [INN]
  • ATIDARSAGENE AUTOTEMCEL [USAN]
  • 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




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