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

Substance Class Structurally Diverse
Created
by admin
on Wed Apr 02 12:56:47 GMT 2025
Edited
by admin
on Wed Apr 02 12:56:47 GMT 2025
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Source Materials State SELF-INACTIVATING
Organism Family Retroviridae
Organism Genus Lentivirus
Organism Species human immunodeficiency virus 1
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name Self-inactivating LV-PGK promoter-alpha-L-iduronidase (IDUA)
Part whole
Record UNII
2CX3CU3LQY
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
AZAMIDUGENE AUTOTEMCEL VECTOR
Common Name English
SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN ALPHA-L-IDURONIDASE (IDUA) UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK)PROMOTER THE CONSTRUCT IS FLANKED BY 5' AND 3' LONG TERMINAL REPEATS (LTRS) AND ALSO CONTAINS A HIV-1 .PSI. PACKAGIN
Preferred Name English
Code System Code Type Description
FDA UNII
2CX3CU3LQY
Created by admin on Wed Apr 02 12:56:47 GMT 2025 , Edited by admin on Wed Apr 02 12:56:47 GMT 2025
PRIMARY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR AZAMIDUGENE AUTOTEMCEL TRANSGENE INSERT 65HBD4H5PV