Seglebegagene Dasniparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 18:38:09 GMT 2023` Edited by `admin` on `Sat Dec 16 18:38:09 GMT 2023`
Source Materials Class	ORGANISM
Source Materials Type	VIRUS
Source Materials State	LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family	Parvoviridae
Organism Genus	Dependoparvovirus
Organism Species	adeno associated virus
Infraspecific Type	SUBSTRAIN
Infraspecific Name	AAVhu68
Part	whole
Record UNII	EXB5VWG5TW
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Seglebegagene Dasniparvovec

Official Name

English

DNA (recombinant replication-defective adeno-associated virus serotype hu68 vector PBGM01 human ubiquitin C promoter plus chimeric chicken β-actin and human β-globin intron plus human β-galactosidase codon-optimized gene GLB1 plus simian virus 40 poly

Common Name

English

seglebegagene dasniparvovec [INN]

Common Name

English

recombinant, non-replicating adeno-associated virus serotype hu68 (rAAVhu68) vector encoding codon-optimised human β-galactosidase (GLB1), under control of the human ubiquitin C promoter plus a chimeric intron, consisting of human β-globin splice donor

Common Name

English

Code System Code Type Description

INN

12225

Created by admin on Sat Dec 16 18:38:09 GMT 2023 , Edited by admin on Sat Dec 16 18:38:09 GMT 2023

PRIMARY

NCI_THESAURUS

C190438

Created by admin on Sat Dec 16 18:38:09 GMT 2023 , Edited by admin on Sat Dec 16 18:38:09 GMT 2023

PRIMARY

CAS

2607029-99-0

Created by admin on Sat Dec 16 18:38:09 GMT 2023 , Edited by admin on Sat Dec 16 18:38:09 GMT 2023

PRIMARY

FDA UNII

EXB5VWG5TW

Created by admin on Sat Dec 16 18:38:09 GMT 2023 , Edited by admin on Sat Dec 16 18:38:09 GMT 2023

PRIMARY

Related Record Type Details


					MEQ84W5J4Q

BETA-GALACTOSIDASE

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					EXB5VWG5TW

Seglebegagene Dasniparvovec

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
TRANSGENE					Seglebegagene dasniparvovec transgene insert	UQX89KA9ST