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BETIBEGLOGENE AUTOTEMCEL

UNII:MEE8487RTP
Formula:
Preferred Substance Name:BETIBEGLOGENE AUTOTEMCEL

  • AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE
  • AUTOLOGOUS CD34+ ENRICHED CELL POPULATION THAT CONTAINS HEMATOPOIETIC STEM CELLS (HSC'S) TRANSDUCED WITH THE LENTIGLOBIN BB305 LENTIVIRAL VECTOR (LVV) ENCODING FOR THE .BETA.A-T87Q-GLOBIN GENE. THE CD34+ ENRICHED CELL POPULATION CONTAINING HEMATOPOIETIC STEM AND PROGENITOR CELLS IS OBTAINED FROM AUTOLOGOUS PERIPHERAL BLOOD MONONUCLEAR CELLS (PBMCS)
  • AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD OF PATIENTS WITH BETA-THALASSEMIA, TRANSDUCED EX VIVO WITH BETIBEGLOGENE DAROLENTIVEC (116)(78), A SELF-INACTIVATING HUMAN IMMUNODEFICIENCY VIRUS-1 (HIV-1)-DERIVED LENTIVIRAL VECTOR ENCODING A T87Q-MUTATED FORM OF THE HUMAN HEMOGLOBIN SUBUNIT BETA (HBB, BETA-GLOBIN) GENE UNDER THE CONTROL OF A HUMAN .BETA.-GLOBIN PROMOTER AND A 3' .BETA.-GLOBIN ENHANCE
  • BB-305
  • BETIBEGLOGENE AUTOTEMCEL [INN]
  • BETIBEGLOGENE AUTOTEMCEL [USAN]
  • BETIBEGLOGENE AUTOTEMCEL [WHO-DD]
  • LENTIGLOBIN BB305
  • ZYNTEGLO



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