DOMOFENOGENE ZALFAPARVOVEC

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 16:07:56 GMT 2023` Edited by `admin` on `Sat Dec 16 16:07:56 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	AAV5-ApoeE-hAAT-LGi-hPAH-GENEius1
Part	WHOLE
Record UNII	PN77BRS6J9
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

DOMOFENOGENE ZALFAPARVOVEC

Official Name

English

DNA (RECOMBINANT REPLICATION-DEFICIENT ADENO-ASSOCIATED VIRUS 5 VECTOR BMN 307 HUMAN LIVER-SPECIFIC TRANSCRIPTION REGULATORY ELEMENT PLUS HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED GENE HPAH PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT F

Common Name

English

BMN 307 (AN AAV5-PHENYLALANINE HYDROXYLASE (PAH) GENE THERAPY)

Common Name

English

domofenogene zalfaparvovec [INN]

Common Name

English

RECOMBINANT, REPLICATION-INCOMPETENT ADENOASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING CODON-OPTIMIZED HUMAN PHENYLALANINE HYDROXYLASE (HPAH) UNDER CONTROL OF A LIVER-SELECTIVE TRANSCRIPTION REGULATORY ELEMENT (TRE), CONSISTING OF THE APOLIPOPROTEI

Common Name

English

BMN-307

Common Name

English

Code System Code Type Description

CAS

2382896-04-8

Created by admin on Sat Dec 16 16:07:56 GMT 2023 , Edited by admin on Sat Dec 16 16:07:56 GMT 2023

PRIMARY

NCI_THESAURUS

C184853

Created by admin on Sat Dec 16 16:07:56 GMT 2023 , Edited by admin on Sat Dec 16 16:07:56 GMT 2023

PRIMARY

INN

11598

Created by admin on Sat Dec 16 16:07:56 GMT 2023 , Edited by admin on Sat Dec 16 16:07:56 GMT 2023

PRIMARY

FDA UNII

PN77BRS6J9

Created by admin on Sat Dec 16 16:07:56 GMT 2023 , Edited by admin on Sat Dec 16 16:07:56 GMT 2023

PRIMARY

Related Record Type Details


					559J4TS483

PHENYLALANINE-4-HYDROXYLASE

TARGET->ENZYME REPLACEMENT

Amount:

Related Record Type Details


					PN77BRS6J9

DOMOFENOGENE ZALFAPARVOVEC

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
VECTOR EXPRESSED PROTEIN					PHENYLALANINE-4-HYDROXYLASE	559J4TS483