AZAMIDUGENE AUTOTEMCEL

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 19:02:31 GMT 2023` Edited by `admin` on `Sat Dec 16 19:02:31 GMT 2023`
Source Materials Class	ORGANISM
Source Materials Type	AUTOLOGOUS HUMAN
Source Materials State	LIVE
Source Materials Parent	`G0T704E6Z3` HOMO SAPIENS WHOLE
Part	BLOOD
Fraction Name	CD34+ CELLS
Record UNII	7BAL95SK9C
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

AZAMIDUGENE AUTOTEMCEL

Official Name

English

AUTOLOGOUS CD34+ CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD, TRANSDUCED WITH A SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN ALPHA-L-IDURONIDASE (IDUA) UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK) PROMOTER THE CONSTRUCT IS FLANKE

Common Name

English

azamidugene autotemcel [INN]

Common Name

English

Code System Code Type Description

NCI_THESAURUS

C184823

Created by admin on Sat Dec 16 19:02:31 GMT 2023 , Edited by admin on Sat Dec 16 19:02:31 GMT 2023

PRIMARY

INN

11773

Created by admin on Sat Dec 16 19:02:31 GMT 2023 , Edited by admin on Sat Dec 16 19:02:31 GMT 2023

PRIMARY

FDA UNII

7BAL95SK9C

Created by admin on Sat Dec 16 19:02:31 GMT 2023 , Edited by admin on Sat Dec 16 19:02:31 GMT 2023

PRIMARY

Related Record Type Details


					658E1771KX

.ALPHA.-L-IDURONIDASE

TARGET->ENZYME REPLACEMENT

Amount:

Related Record Type Details


					7BAL95SK9C

AZAMIDUGENE AUTOTEMCEL

ACTIVE MOIETY

Amount:

Agent Modifications

Modification Process	Modification Role	Modification Type	Amount	Fragment Name	Fragment ID
LENTIVIRAL TRANSFECTION	TRANSFECTION			AZAMIDUGENE AUTOTEMCEL VECTOR	2CX3CU3LQY

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					AZAMIDUGENE AUTOTEMCEL TRANSGENE INSERT	65HBD4H5PV
VECTOR EXPRESSED PROTEIN					.ALPHA.-L-IDURONIDASE	658E1771KX