Arvenacogene Sanparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 20:19:51 GMT 2025` Edited by `admin` on `Wed Apr 02 20:19:51 GMT 2025`
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	rAAV5-codon-optimized FIX (Padua variant, R338L)
Part	whole
Record UNII	KNP55LBJ2N
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Capsomere, recombinant adeno-associated virus 5 viral capsid protein VP1-VP2-VP3 complex (1:1:10), hexacontamer, complex with DNA (recombinant adeno-associated virus vector pAAV-hFIXco-v2 Mus musculus transtyretin gene promoter plus human ?-globin gene i

Preferred Name

English

Arvenacogene Sanparvovec

Official Name

English

recombinant non-replicating adeno-associated virus serotype 5 (AAV5) vector encoding a codon-optimized gain of function variant (Padua variant, R338L) of human coagulation factor IX (FIX), under the control of a murine liver-specific transthyretin (TTR)

Common Name

English

arvenacogene sanparvovec [INN]

Common Name

English

Code System Code Type Description

NCI_THESAURUS

C211781

Created by admin on Wed Apr 02 20:19:51 GMT 2025 , Edited by admin on Wed Apr 02 20:19:51 GMT 2025

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CAS

2891724-48-2

Created by admin on Wed Apr 02 20:19:51 GMT 2025 , Edited by admin on Wed Apr 02 20:19:51 GMT 2025

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FDA UNII

KNP55LBJ2N

Created by admin on Wed Apr 02 20:19:51 GMT 2025 , Edited by admin on Wed Apr 02 20:19:51 GMT 2025

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INN

13234

Created by admin on Wed Apr 02 20:19:51 GMT 2025 , Edited by admin on Wed Apr 02 20:19:51 GMT 2025

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Related Record Type Details


					6UZ594565X

Coagulation factor IX human, (338-Leucine)-

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					KNP55LBJ2N

Arvenacogene Sanparvovec

ACTIVE MOIETY

Comments:

gene therapy (hemophilia B)

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
GENE_EXPRESSION VECTOR					Arvenacogene sanparvovec transgene insert	X96BYJ9M32