Ristoglogene Autogetemcel

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 18:18:56 GMT 2025` Edited by `admin` on `Wed Apr 02 18:18:56 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	blood
Fraction Material Type	CELLS
Fraction Name	cd34+ hematopoietic stem and progenitor cells (hspcs)
Record UNII	CEL7E4NG36
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) obtained from peripheral blood by leukapheresis, genetically modified ex vivo by electroporation of a guide RNA (gRNA) that targets the two ?-globin gene (HBG1 and HBG2) promotors and a mes

Preferred Name

English

Ristoglogene Autogetemcel

Official Name

English

ristoglogene autogetemcel [INN]

Common Name

English

Code System Code Type Description

FDA UNII

CEL7E4NG36

Created by admin on Wed Apr 02 18:18:56 GMT 2025 , Edited by admin on Wed Apr 02 18:18:56 GMT 2025

PRIMARY

INN

13397

Created by admin on Wed Apr 02 18:18:56 GMT 2025 , Edited by admin on Wed Apr 02 18:18:56 GMT 2025

PRIMARY

Related Record Type Details


					43F9JP2P5Z

HEMOGLOBIN F

TARGET->CORRECTOR

Amount:

Related Record Type Details


					CEL7E4NG36

Ristoglogene Autogetemcel

ACTIVE MOIETY

Comments:

?75% on-target editing

Amount:

Agent Modifications

Modification Process	Modification Role	Modification Type	Fragment Name	Fragment ID
ELECTROPORATION	GENE EDITING	GENE MODIFICATION	Ristoglogene autogetemcel mRNA	AAF4LU29JC
CULTURE	EXPANSION	BIOLOGICAL
ELECTROPORATION	GENE EDITING	GENE MODIFICATION	Ristoglogene autogetemcel gRNA	5UBM9CGH6K

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
VECTOR EXPRESSED PROTEIN					Ristoglogene autogetemcel expressed adenine base editor 8.8 (ABE8.8)	HDJ8SRJ2YN

Name	Property Type	Referenced Substance
ANTIGEN EXPRESSED	BIOLOGICAL	`ZWL6BH0E0S` HEMATOPOIETIC PROGENITOR CELL ANTIGEN CD34

ANTIGEN EXPRESSED	BIOLOGICAL	`FOR10CZY15` RECEPTOR-TYPE TYROSINE-PROTEIN PHOSPHATASE C