FIROLIMOGENE AUTOTEMCEL

U.S. Department of Health & Human Services Divider Arrow

National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 11:16:00 GMT 2025` Edited by `admin` on `Wed Apr 02 11:16:00 GMT 2025`
Source Materials Class	ORGANISM
Source Materials Type	AUTOLOGOUS HUMAN
Source Materials State	LIVE GENETICALLY MODIFIED
Source Materials Parent	`G0T704E6Z3` HOMO SAPIENS WHOLE
Part	bone marrow
Fraction Name	cd34+ cells isolated from bone marrow of newly diagnosed x-linked severe combined immunodeficiency (x-scid) patients
Record UNII	XH2XT2GQS6
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

FIROLIMOGENE AUTOTEMCEL

Official Name

English

AUTOLOGOUS CD34+ CELLS ISOLATED FROM BONE MARROW OF NEWLY DIAGNOSED X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (X-SCID) PATIENTS, TRANSDUCED EX VIVO WITH A SELF-INACTIVATING LENTIVIRAL VECTOR, ENCODING CODON-OPTIMIZED HUMAN INTERLEUKIN-2 RECEPTOR SUBUNIT

Preferred Name

English

firolimogene autotemcel [INN]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

759720

Created by admin on Wed Apr 02 11:16:00 GMT 2025 , Edited by admin on Wed Apr 02 11:16:00 GMT 2025

Code System Code Type Description

SMS_ID

300000015441

Created by admin on Wed Apr 02 11:16:00 GMT 2025 , Edited by admin on Wed Apr 02 11:16:00 GMT 2025

PRIMARY

FDA UNII

XH2XT2GQS6

Created by admin on Wed Apr 02 11:16:00 GMT 2025 , Edited by admin on Wed Apr 02 11:16:00 GMT 2025

PRIMARY

INN

11551

Created by admin on Wed Apr 02 11:16:00 GMT 2025 , Edited by admin on Wed Apr 02 11:16:00 GMT 2025

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NCI_THESAURUS

C184824

Created by admin on Wed Apr 02 11:16:00 GMT 2025 , Edited by admin on Wed Apr 02 11:16:00 GMT 2025

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Related Record Type Details


					OX7CIS4H0Z

CYTOKINE RECEPTOR COMMON SUBUNIT GAMMA

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					XH2XT2GQS6

FIROLIMOGENE AUTOTEMCEL

ACTIVE MOIETY

Amount:

Agent Modifications

Modification Process	Modification Role	Modification Type	Amount	Fragment Name	Fragment ID
LENTIVIRAL TRANSFECTION	TRANSFECTION			FIROLIMOGENE AUTOTEMCEL VECTOR	PZ6C5A9U4S

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
VECTOR EXPRESSED PROTEIN					CYTOKINE RECEPTOR COMMON SUBUNIT GAMMA	OX7CIS4H0Z
INSERTED GENE EXPRESSION VECTOR					FIROLIMOGENE AUTOTEMCEL TRANSGENE INSERT	73GRS55F42