Nimatpagene Pariparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 18:48:01 UTC 2023` Edited by `admin` on `Sat Dec 16 18:48:01 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 b
Infraspecific Type	RECOMBINANT VECTOR
Infraspecific Name	rAAV3B-ATP7Bmini (AAV2LTR/AAV3Bcapsid-ATP7Bmini)
Part	whole
Record UNII	C4J4L5V7XS
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Nimatpagene Pariparvovec

Common Name

English

Capsomere, recombinant human adeno-associated virus AAV3B viral capsid protein VP1-VP2-VP3 complex (1:2:7), hexacontamer, complex with DNA (recombinant replication-defective adeno-associated virus serotype 3B vector VTX-801 liver-specific human α-1 anti

Common Name

English

nimatpagene pariparvovec [INN]

Common Name

English

VTX-801

Code

English

Recombinant, non-replicating adeno-associated virus serotype 3B (rAAV3B) vector, encoding a shortened version of the human copper-transporting ATPase 2 (ATP7B) (also known as ATPase copper-transporting beta, Wilson disease-associated protein) which is de

Common Name

English

Code System Code Type Description

CAS

2773545-20-1

Created by admin on Sat Dec 16 18:48:01 UTC 2023 , Edited by admin on Sat Dec 16 18:48:01 UTC 2023

PRIMARY

FDA UNII

C4J4L5V7XS

Created by admin on Sat Dec 16 18:48:01 UTC 2023 , Edited by admin on Sat Dec 16 18:48:01 UTC 2023

PRIMARY

INN

12798

Created by admin on Sat Dec 16 18:48:01 UTC 2023 , Edited by admin on Sat Dec 16 18:48:01 UTC 2023

PRIMARY

Related Record Type Details


					C4J4L5V7XS

Nimatpagene Pariparvovec

ACTIVE MOIETY

Comments:

Preclinical; The biological activity of VTX-801 allows restoring defective ATP7B function

Amount:

Structural Modifications

Modification Type	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR	Nimatpagene pariparvovec transgene insert	LLR6QA66HW
VIRAL CAPSID	UNSPECIFIED SUBSTANCE	65f6429a (not in database)
VIRAL CAPSID	UNSPECIFIED SUBSTANCE	a84a61b9 (not in database)
VIRAL CAPSID	UNSPECIFIED SUBSTANCE	72b059a1 (not in database)