Pozetaldogene Ormesparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 20:18:04 UTC 2023` Edited by `admin` on `Sat Dec 16 20:18:04 UTC 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	SBT101 (rAAV9.CBA.CMV.ABCD1)
Part	WHOLE
Record UNII	R5CJD8SH2N
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Pozetaldogene Ormesparvovec

Official Name

English

Recombinant, non-replicating adeno-associated virus serotype 9 (rAAV9) vector encoding human adenosine triphosphate (ATP)-binding cassette sub-family D member 1 (ABCD1, also known as adrenoleukodystrophy (ALD) protein) under control of a human cytomegalo

Common Name

English

DNA (recombinant replication-defective adeno-associated virus serotype 9 vector SBT101 cytomegalovirus enhancer fusion with chicken β-actin promoter and exon 1plus chimeric chicken β-actin and rabbit β-globin intron plus rabbit β-globin exon 3 plus

Common Name

English

pozetaldogene ormesparvovec [INN]

Common Name

English

SBT-101

Code

English

Code System Code Type Description

CAS

2764626-03-9

Created by admin on Sat Dec 16 20:18:04 UTC 2023 , Edited by admin on Sat Dec 16 20:18:04 UTC 2023

PRIMARY

INN

12672

Created by admin on Sat Dec 16 20:18:04 UTC 2023 , Edited by admin on Sat Dec 16 20:18:04 UTC 2023

PRIMARY

FDA UNII

R5CJD8SH2N

Created by admin on Sat Dec 16 20:18:04 UTC 2023 , Edited by admin on Sat Dec 16 20:18:04 UTC 2023

PRIMARY

Related Record Type Details


					GK3D7Z9R9V

ATP-BINDING CASSETTE SUB-FAMILY D MEMBER 1

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					R5CJD8SH2N

Pozetaldogene Ormesparvovec

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					Pozetaldogene ormesparvovec transgene insert	7QPY3D8XAF