SIMOLADAGENE AUTOTEMCEL

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

National Institutes of Health Divider Arrow

NCATS

Investigational

Investigational

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 03:47:50 GMT 2025` Edited by `admin` on `Wed Apr 02 03:47:50 GMT 2025`
Source Materials Class	ORGANISM
Source Materials Type	AUTOLOGOUS HUMAN
Source Materials State	LIVE
Source Materials Parent	`G0T704E6Z3` HOMO SAPIENS WHOLE
Part	bone marrow
Fraction Material Type	cells
Fraction Name	hematopoietic stem and progenitor cells
Record UNII	MOD3RG31RS
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

OTL-101

Preferred Name

English

SIMOLADAGENE AUTOTEMCEL

Official Name

English

CELL BASED DRUG PRODUCT CONSISTING OF CELLS THAT HAVE BEEN GENETICALLY MODIFIED EX VIVO. IT INCLUDES AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS (HSCS) FROM SEVERE COMBINED IMMUNODEFICIENCY DUE TO ADENOSINE DEAMINASE DEFICIENCY (ADA-SCID) SUBJECTS TRANSDUC

Common Name

English

SIMOLADAGENE AUTOTEMCEL [USAN]

Common Name

English

simoladagene autotemcel [INN]

Common Name

English

Simoladagene autotemcel [WHO-DD]

Common Name

English

Code System Code Type Description

FDA UNII

MOD3RG31RS

Created by admin on Wed Apr 02 03:47:50 GMT 2025 , Edited by admin on Wed Apr 02 03:47:50 GMT 2025

PRIMARY

SMS_ID

100000174710

Created by admin on Wed Apr 02 03:47:50 GMT 2025 , Edited by admin on Wed Apr 02 03:47:50 GMT 2025

PRIMARY

INN

11042

Created by admin on Wed Apr 02 03:47:50 GMT 2025 , Edited by admin on Wed Apr 02 03:47:50 GMT 2025

PRIMARY

USAN

FG-30

Created by admin on Wed Apr 02 03:47:50 GMT 2025 , Edited by admin on Wed Apr 02 03:47:50 GMT 2025

PRIMARY

NCI_THESAURUS

C171810

Created by admin on Wed Apr 02 03:47:50 GMT 2025 , Edited by admin on Wed Apr 02 03:47:50 GMT 2025

PRIMARY

Related Record Type Details


					MOD3RG31RS

SIMOLADAGENE AUTOTEMCEL

ACTIVE MOIETY

Agent Modifications

Modification Process	Modification Role	Modification Type	Amount	Fragment Name	Fragment ID
CULTURE	Pre-stimulation	IMMUNOLOGICAL		RECOMBINANT HUMAN THROMBOPOIETIN	E2E529W34D
	CD34 Selection	CELL SEPARATION
CULTURE		IMMUNOLOGICAL		FLT-3 LIGAND PROTEIN (THR27-PRO185; INSECT GLYCOSYLATION)	4F6H64K1NO
	TRANSFECTION	LENTIVIRAL TRANSFECTION		SIMOLADAGENE AUTOTEMCEL LENTIVIRAL VECTOR	DN654H6FDU
	RBC depletion	CELL SEPARATION
CULTURE	Pre-stimulation	IMMUNOLOGICAL		MUPLESTIM	A802907MI4

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
VECTOR EXPRESSED PROTEIN					ADENOSINE DEAMINASE	XPQ5L1L4WO