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

Approval Year

Unknown
149124
Name Type Language
GILAVEBEXAGENE ANVUPARVOVEC
USAN  
Official Name English
AXO-AAV-GM2
Preferred Name English
AXO-AAV-GM2 (RAAVRH8-HEXA/B) IS A ONE-TIME, ADENO-ASSOCIATED VIRUS (AAV) IN VIVO GENE THERAPY FOR THE TREATMENT OF TAY-SACHS AND SANDHOFF DISEASE. THE THERAPY IS DESIGNED TO PROVIDE A FUNCTIONAL COPY OF BOTH HEXA AND HEXB GENES THROUGH A 1:1 FORMULATION
Common Name English
GILAVEBEXAGENE ANVUPARVOVEC [USAN]
Common Name English
COBNABEXAGENE ANVUPARVOVEC
Common Name English
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE RH.8 VECTOR AAVRH8-CB-CI-HEXB CHICKEN .BETA.-ACTIN PROMOTER/CHIMERIC CHICKEN .BETA.-ACTIN AND RABBIT .BETA.-GLOBIN INTRON/HUMAN .BETA.-ACETYLHEXOSAMINIDASE .BETA.-SUBUNIT TRANSGENE WTHEXB/SV40 POLYADENYLAT
Common Name English
Code System Code Type Description
USAN
JK-189
Created by admin on Wed Apr 02 10:10:45 GMT 2025 , Edited by admin on Wed Apr 02 10:10:45 GMT 2025
PRIMARY
INN
12326
Created by admin on Wed Apr 02 10:10:45 GMT 2025 , Edited by admin on Wed Apr 02 10:10:45 GMT 2025
PRIMARY
NCI_THESAURUS
C188561
Created by admin on Wed Apr 02 10:10:45 GMT 2025 , Edited by admin on Wed Apr 02 10:10:45 GMT 2025
PRIMARY
CAS
2364477-18-7
Created by admin on Wed Apr 02 10:10:45 GMT 2025 , Edited by admin on Wed Apr 02 10:10:45 GMT 2025
PRIMARY
FDA UNII
CUC7FB5YWE
Created by admin on Wed Apr 02 10:10:45 GMT 2025 , Edited by admin on Wed Apr 02 10:10:45 GMT 2025
PRIMARY
All of the following components must be present:
COBNABEXAGENE ANVUPARVOVEC HEXA
COBNABEXAGENE ANVUPARVOVEC HEXB
Definition References
NIH
NCATS
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