U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Substance Class Structurally Diverse
Created
by admin
on Wed Apr 02 07:40:03 GMT 2025
Edited
by admin
on Wed Apr 02 07:40:03 GMT 2025
Source Materials Class ORGANISM
Source Materials Type HUMAN
Source Materials State LIVE
Source Materials Parent
Part blood
Fraction Material Type cells
Fraction Name hematopoietic stem and progenitor cells (hspcs)
Record UNII
2GNP7PE0CG
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Dazagamglogene Autogedtemcel
INN  
Official Name English
Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) collected by leukapheresis and genetically modified ex vivo by a pair of zinc finger nucleases (ZFN) directed against the genomic sequence of the erythroid-specific enhancer (ESE) located i
Preferred Name English
dazagamglogene autogedtemcel [INN]
Common Name English
Code System Code Type Description
NCI_THESAURUS
C203029
Created by admin on Wed Apr 02 07:40:03 GMT 2025 , Edited by admin on Wed Apr 02 07:40:03 GMT 2025
PRIMARY
FDA UNII
2GNP7PE0CG
Created by admin on Wed Apr 02 07:40:03 GMT 2025 , Edited by admin on Wed Apr 02 07:40:03 GMT 2025
PRIMARY
INN
12741
Created by admin on Wed Apr 02 07:40:03 GMT 2025 , Edited by admin on Wed Apr 02 07:40:03 GMT 2025
PRIMARY
Related Record Type Details
ACTIVE MOIETY
Severe Sickle Cell Disease

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
CULTURE EXPANSION CELL SEPARATION UNSPECIFIED SUBSTANCE 9cd8c8f9
TRANSFECTION FOLLOWED BY EXPANSION AND RELEASE FROM MAGNETIC BEADS TRANSFECTION Gene delivery by mRNA electroporation mRENH1 1DSV7GF306
TRANSFECTION FOLLOWED BY EXPANSION AND RELEASE FROM MAGNETIC BEADS TRANSFECTION Gene delivery by mRNA electroporation mRENH2 RB6ZJ0EJ1O