Pivodisgene Parvec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 18:44:41 GMT 2025` Edited by `admin` on `Wed Apr 02 18:44:41 GMT 2025`
Source Materials Class	ORGANISM
Source Materials Type	RECOMBINANT VIRUS
Source Materials State	LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family	Parvoviridae
Organism Genus	Dependovirus
Organism Species	adeno-associated dependoparvovirus a
Infraspecific Type	RECOMBINANT VECTOR
Infraspecific Name	AAV8.Spc5-12.Microdystrophin
Part	whole
Record UNII	NVQ25YNH8P
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

RGX-202 (AAV8.Spc5-12.Microdystrophin)

Preferred Name

English

Pivodisgene Parvec

Official Name

English

recombinant, non-replicating adeno-associated virus serotype 8 (rAAV8) vector, encoding a codon-optimised miniaturised version of human dystrophin (microdystrophin variant ABD1-H1-R1-R2-R3-H3-R24-H4-CR-CT194), under control of a synthetic muscle-specific

Common Name

English

pivodisgene parvec [INN]

Common Name

English

Recombinant replication-defective adeno-associated virus 8 vector RGX-202 synthetic promoter Spc5-12 plus human codon-optimized microdystrophin variant ABD1-H1-R1-R2-R3-H3-R24-H4-CR-CT194 plus synthetic polyadenylation signal plus adeno-associated virus

Common Name

English

Code System Code Type Description

FDA UNII

NVQ25YNH8P

Created by admin on Wed Apr 02 18:44:41 GMT 2025 , Edited by admin on Wed Apr 02 18:44:41 GMT 2025

PRIMARY

CAS

2648631-52-9

Created by admin on Wed Apr 02 18:44:41 GMT 2025 , Edited by admin on Wed Apr 02 18:44:41 GMT 2025

PRIMARY

INN

13471

Created by admin on Wed Apr 02 18:44:41 GMT 2025 , Edited by admin on Wed Apr 02 18:44:41 GMT 2025

PRIMARY

Related Record Type Details


					WVT37PK339

Pivodisgene parvec expressed human microdystrophin

EXPRESSED PROTEIN->VECTOR

Amount:


					60RX8439K2

DYSTROPHIN

TARGET->MIMETIC

Amount:

Related Record Type Details


					NVQ25YNH8P

Pivodisgene Parvec

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					Pivodisgene parvec transgene insert	G74YZ2T2M7