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 12:16:20 GMT 2025
Edited
by admin
on Wed Apr 02 12:16:20 GMT 2025
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependoparvovirus
Organism Species adeno-associated dependoparvovirus a
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name rAAV9/CMV/HEXB-P2A-HEXA
Part whole
Record UNII
XX923BN5NY
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ENGABEXAGENE CINCESPARVOVEC
Common Name English
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-101 SYNTHETIC CAG PROMOTER PLUS HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SUBUNIT .BETA. PRECURSOR FUSION PROTEIN WITH PORCINE TESCHOVIRUS 2A PEPTIDE FUSION PROTEIN WITH HUMAN .BETA.-N-ACETYLHEXOSAMINIDASE SU
Preferred Name English
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (RAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN .BETA.-HEXOSAMINIDASE SUBUNITS .BETA. AND .ALPHA., JOINED BY A PORCINE TESCHOVIRUS 2A PEPTIDE LINKER, UNDER CONTROL OF A SYNTHETIC PROMOTER (CYT
Common Name English
engabexagene cincesparvovec [INN]
Common Name English
Code System Code Type Description
INN
12045
Created by admin on Wed Apr 02 12:16:20 GMT 2025 , Edited by admin on Wed Apr 02 12:16:20 GMT 2025
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FDA UNII
XX923BN5NY
Created by admin on Wed Apr 02 12:16:20 GMT 2025 , Edited by admin on Wed Apr 02 12:16:20 GMT 2025
PRIMARY
NCI_THESAURUS
C188657
Created by admin on Wed Apr 02 12:16:20 GMT 2025 , Edited by admin on Wed Apr 02 12:16:20 GMT 2025
PRIMARY
CAS
2567881-77-8
Created by admin on Wed Apr 02 12:16:20 GMT 2025 , Edited by admin on Wed Apr 02 12:16:20 GMT 2025
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SMS_ID
300000032081
Created by admin on Wed Apr 02 12:16:20 GMT 2025 , Edited by admin on Wed Apr 02 12:16:20 GMT 2025
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Related Record Type Details
TARGET->ENZYME REPLACEMENT
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR ENGABEXAGENE CINCESPARVOVEC TRANSGENE INSERT RN9AXF4YZA