Tidagixagene Derxeparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 19:26:56 GMT 2023` Edited by `admin` on `Sat Dec 16 19:26:56 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	scAAV9/Synthetic JeT promoter/human codon-optimized gigaxonin/AAV2 inverted terminal repeats
Part	whole
Record UNII	Y7ARS22SWQ
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Tidagixagene Derxeparvovec

Official Name

English

TSHA-120

Common Name

English

DNA (recombinant adeno-associated virus 9 vector TSHA-120 synthetic JeT promoter plus human gigaxonin transgene plus synthetic polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying)

Common Name

English

recombinant, self-complementary (dimeric), non-replicating adeno-associated virus serotype 9 (scAAV9) vector encoding codon-optimised human gigaxonin (giant axonal neuropathy [GAN] gene, Kelch-like protein 16) under the control of the artificial JeT prom

Common Name

English

tidagixagene derxeparvovec [INN]

Common Name

English

Code System Code Type Description

FDA UNII

Y7ARS22SWQ

Created by admin on Sat Dec 16 19:26:56 GMT 2023 , Edited by admin on Sat Dec 16 19:26:56 GMT 2023

PRIMARY

CAS

2644621-85-0

Created by admin on Sat Dec 16 19:26:56 GMT 2023 , Edited by admin on Sat Dec 16 19:26:56 GMT 2023

PRIMARY

NCI_THESAURUS

C190499

Created by admin on Sat Dec 16 19:26:56 GMT 2023 , Edited by admin on Sat Dec 16 19:26:56 GMT 2023

PRIMARY

INN

12310

Created by admin on Sat Dec 16 19:26:56 GMT 2023 , Edited by admin on Sat Dec 16 19:26:56 GMT 2023

PRIMARY

Related Record Type Details


					E3R8GD2S69

Gigaxonin

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					Y7ARS22SWQ

Tidagixagene Derxeparvovec

ACTIVE MOIETY

Comments:

gene therapy (giant axonal neuropathy)

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					Tidagixagene derxeparvovec transgene insert	P3Z5DV7R24