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 13:24:07 GMT 2025
Edited
by admin
on Wed Apr 02 13:24:07 GMT 2025
Source Materials Class ORGANISM
Source Materials Type AUTOLOGOUS HUMAN
Source Materials State LIVE
Source Materials Parent
Part blood
Fraction Name cd34+ cells
Record UNII
7BAL95SK9C
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
AUTOLOGOUS CD34+ CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD, TRANSDUCED WITH A SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN ALPHA-L-IDURONIDASE (IDUA) UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK) PROMOTER THE CONSTRUCT IS FLANKE
Preferred Name English
AZAMIDUGENE AUTOTEMCEL
INN  
Official Name English
azamidugene autotemcel [INN]
Common Name English
Code System Code Type Description
NCI_THESAURUS
C184823
Created by admin on Wed Apr 02 13:24:07 GMT 2025 , Edited by admin on Wed Apr 02 13:24:07 GMT 2025
PRIMARY
INN
11773
Created by admin on Wed Apr 02 13:24:07 GMT 2025 , Edited by admin on Wed Apr 02 13:24:07 GMT 2025
PRIMARY
FDA UNII
7BAL95SK9C
Created by admin on Wed Apr 02 13:24:07 GMT 2025 , Edited by admin on Wed Apr 02 13:24:07 GMT 2025
PRIMARY
SMS_ID
100000177556
Created by admin on Wed Apr 02 13:24:07 GMT 2025 , Edited by admin on Wed Apr 02 13:24:07 GMT 2025
PRIMARY
Related Record Type Details
TARGET->ENZYME REPLACEMENT
Related Record Type Details
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
LENTIVIRAL TRANSFECTION TRANSFECTION AZAMIDUGENE AUTOTEMCEL VECTOR 2CX3CU3LQY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR AZAMIDUGENE AUTOTEMCEL TRANSGENE INSERT 65HBD4H5PV
VECTOR EXPRESSED PROTEIN .ALPHA.-L-IDURONIDASE 658E1771KX