ZAFTUCLENEGENE PIRUPARVOVEC

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 18:10:02 GMT 2023` Edited by `admin` on `Sat Dec 16 18:10:02 GMT 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	TSHA-118 (scAAV9-β-actin hybrid promoter-PPT1 precursor)
Part	WHOLE
Record UNII	R5X46L79RV
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ZAFTUCLENEGENE PIRUPARVOVEC

Official Name

English

DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-118 CHICKEN .BETA.-ACTIN HYBRID PROMOTER PLUS HUMAN ACYL PROTEIN THIOESTER HYDROLASE PPT1 PRECURSOR TRANSGENE PLUS BOVINE GROWTH HORMONE POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TER

Common Name

English

zaftuclenegene piruparvovec [INN]

Common Name

English

RECOMBINANT, SELF-COMPLEMENTARY (DIMERIC), NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (SCAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN PALMITOYL-PROTEIN THIOESTERASE 1 (PPT1, CLN1), UNDER THE CONTROL OF A SYNTHETIC PROMOTER CONSISTING OF ACYTOMEGAL

Common Name

English

Code System Code Type Description

FDA UNII

R5X46L79RV

Created by admin on Sat Dec 16 18:10:02 GMT 2023 , Edited by admin on Sat Dec 16 18:10:02 GMT 2023

PRIMARY

INN

12046

Created by admin on Sat Dec 16 18:10:02 GMT 2023 , Edited by admin on Sat Dec 16 18:10:02 GMT 2023

PRIMARY

NCI_THESAURUS

C188620

Created by admin on Sat Dec 16 18:10:02 GMT 2023 , Edited by admin on Sat Dec 16 18:10:02 GMT 2023

PRIMARY

CAS

2567881-78-9

Created by admin on Sat Dec 16 18:10:02 GMT 2023 , Edited by admin on Sat Dec 16 18:10:02 GMT 2023

PRIMARY

Related Record Type Details


					G93VK52MZZ

PALMITOYL-PROTEIN THIOESTERASE 1

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					R5X46L79RV

ZAFTUCLENEGENE PIRUPARVOVEC

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					ZAFTUCLENEGENE PIRUPARVOVEC TRANSGENE INSERT	UA46HCC58W