GILAVEBEXAGENE ANVUPARVOVEC

Approval Year

Substance Class	Mixture Created by `admin` on `Sat Dec 16 18:07:24 UTC 2023` Edited by `admin` on `Sat Dec 16 18:07:24 UTC 2023`
Record UNII	CUC7FB5YWE
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

GILAVEBEXAGENE ANVUPARVOVEC

Official Name

English

AXO-AAV-GM2

Code

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