ENGABEXAGENE CINCESPARVOVEC

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 18:47:34 UTC 2023` Edited by `admin` on `Sat Dec 16 18:47:34 UTC 2023`
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

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Name

Type

Language

ENGABEXAGENE CINCESPARVOVEC

Common 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

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

Common Name

English

Code System Code Type Description

INN

12045

Created by admin on Sat Dec 16 18:47:35 UTC 2023 , Edited by admin on Sat Dec 16 18:47:35 UTC 2023

PRIMARY

FDA UNII

XX923BN5NY

Created by admin on Sat Dec 16 18:47:35 UTC 2023 , Edited by admin on Sat Dec 16 18:47:35 UTC 2023

PRIMARY

NCI_THESAURUS

C188657

Created by admin on Sat Dec 16 18:47:35 UTC 2023 , Edited by admin on Sat Dec 16 18:47:35 UTC 2023

PRIMARY

CAS

2567881-77-8

Created by admin on Sat Dec 16 18:47:35 UTC 2023 , Edited by admin on Sat Dec 16 18:47:35 UTC 2023

PRIMARY

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HEXOSAMINIDASE A

TARGET->ENZYME REPLACEMENT

Amount:


					AER35W4AMJ

ENGABEXAGENE CINCESPARVOVEC UNPROCESSED PROTEIN

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					XX923BN5NY

ENGABEXAGENE CINCESPARVOVEC

ACTIVE MOIETY

Amount:

Structural Modifications

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