Nulabeglogene Autogedtemcel

Investigational

Approval Year

Substance Class	Structurally Diverse
Source Materials Class	ORGANISM
Source Materials Type	AUTOLOGOUS HUMAN
Source Materials State	LIVE ATTENUATED GENETICALLY MODIFIED
Source Materials Parent	`G0T704E6Z3` HOMO SAPIENS WHOLE
Part	blood
Fraction Name	cd34+ hematopoietic stem and progenitor cells from sickle cell patients
Record UNII	H9VA9H989B
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) obtained by apheresis from sickle cell patients, genetically modified ex vivo by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated

Preferred Name

English

Nulabeglogene Autogedtemcel

Official Name

English

GPH101

Code

English

GPH-101

Code

English

nulabeglogene autogedtemcel [INN]

Common Name

English

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Code System

Code

Type

Description

NCI_THESAURUS

C190451

PRIMARY

FDA UNII

H9VA9H989B

PRIMARY

SMS_ID

300000046987

PRIMARY

USAN

LM-06

PRIMARY

INN

12025

PRIMARY

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Related Record

Type

Details


					58L34KZV4N

HEMOGLOBIN SUBUNIT BETA

EXPRESSED PROTEIN->VECTOR

Amount:

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Related Record

Type

Details


					H9VA9H989B

Nulabeglogene Autogedtemcel

ACTIVE MOIETY

Comments:

cell-based gene therapy (sickle cell disease)

Amount:

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Agent Modifications

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Modification Process	Modification Role	Modification Type	Amount	Fragment Name	Fragment ID
	TRANSFECTION			NULABEGLOGENE AUTOGEDTEMCEL AAV6 VECTOR	G2X9QZG8TU
	GENE EDITING			NULABEGLOGENE AUTOGEDTEMCEL SINGLE GUIDE RNA SEQUENCE TARGETING THE FIRST EXON OF THE HUMAN .BETA.-GLOBIN (HBB) GENE	AAC95PP873

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Structural Modifications

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Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					NULABEGLOGENE AUTOGEDTEMCEL TRANSGENE INSERT	KND6MY3R59

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