Nefonadogene Vonzuparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 19:50:54 GMT 2025` Edited by `admin` on `Wed Apr 02 19:50:54 GMT 2025`
Source Materials Class	ORGANISM
Source Materials Type	RECOMBINANT VIRUS
Source Materials State	LIVE GENETICALLY MODIFIED NON-REPLICATING
Organism Family	Parvoviridae
Organism Genus	Dependoparvovirus
Organism Species	adeno-associated dependoparvovirus a
Infraspecific Type	RECOMBINANT VECTOR
Infraspecific Name	rAAV2/COX10/ND1
Part	whole
Record UNII	FBB99Y9WCY
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Nefonadogene Vonzuparvovec

Official Name

English

DNA (recombinant replication-defective adeno-associated virus 2 vector NFS02 human cytomegalovirus enhancer plus human cytomegalovirus promoter plus Macacine herpesvirus 3 chimeric intron plus human heme A farnesyltransferase gene COX10 mitochondrial tar

Preferred Name

English

nefonadogene vonzuparvovec [INN]

Common Name

English

recombinant, non-replicating adeno-associated virus serotype 2 (rAAV2) vector, encoding a mitochondrial targeting sequence from the human heme A farnesyltransferase gene (COX10) fused to codon-optimised human mitochondrial NADH dehydrogenase subunit 1 (M

Common Name

English

Code System Code Type Description

CAS

2779554-99-1

Created by admin on Wed Apr 02 19:50:54 GMT 2025 , Edited by admin on Wed Apr 02 19:50:54 GMT 2025

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FDA UNII

FBB99Y9WCY

Created by admin on Wed Apr 02 19:50:54 GMT 2025 , Edited by admin on Wed Apr 02 19:50:54 GMT 2025

PRIMARY

INN

12868

Created by admin on Wed Apr 02 19:50:54 GMT 2025 , Edited by admin on Wed Apr 02 19:50:54 GMT 2025

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NCI_THESAURUS

C211770

Created by admin on Wed Apr 02 19:50:54 GMT 2025 , Edited by admin on Wed Apr 02 19:50:54 GMT 2025

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Related Record Type Details


					GD3X9G2REP

Nefonadogene vonzuparvovec expressed COX10/human mitochondrial NADH dehydrogenase subunit 1 fusion protein

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					FBB99Y9WCY

Nefonadogene Vonzuparvovec

ACTIVE MOIETY

Comments:

gene therapy (Leber's hereditary optic neuropathy)

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR		UNKNOWN			Nefonadogene vonzuparvovec transgene insert	V7ZD8WVA5S