Nulabeglogene Autogedtemcel

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 19:10:04 GMT 2023` Edited by `admin` on `Sat Dec 16 19:10:04 GMT 2023`
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

Nulabeglogene Autogedtemcel

Official Name

English

GPH101

Code

English

GPH-101

Code

English

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

Common Name

English

nulabeglogene autogedtemcel [INN]

Common Name

English

NULABEGLOGENE AUTOGEDTEMCEL [USAN]

Common Name

English

Autologous, CRISPR/Cas9-based, gene-editing hematopoietic stem cell therapy (HSCT) targeted at the beta-globin (HBB) gene and designed to correct its mutation responsible for sickle cell disease (that is, designed to convert the mutant HbS allele of the

Common Name

English

Code System Code Type Description

NCI_THESAURUS

C190451

Created by admin on Sat Dec 16 19:10:05 GMT 2023 , Edited by admin on Sat Dec 16 19:10:05 GMT 2023

PRIMARY

FDA UNII

H9VA9H989B

Created by admin on Sat Dec 16 19:10:05 GMT 2023 , Edited by admin on Sat Dec 16 19:10:05 GMT 2023

PRIMARY

USAN

LM-06

Created by admin on Sat Dec 16 19:10:05 GMT 2023 , Edited by admin on Sat Dec 16 19:10:05 GMT 2023

PRIMARY

INN

12025

Created by admin on Sat Dec 16 19:10:05 GMT 2023 , Edited by admin on Sat Dec 16 19:10:05 GMT 2023

PRIMARY

Related Record Type Details


					58L34KZV4N

HEMOGLOBIN SUBUNIT BETA

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					H9VA9H989B

Nulabeglogene Autogedtemcel

ACTIVE MOIETY

Comments:

cell-based gene therapy (sickle cell disease)

Amount:

Agent Modifications

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

Structural Modifications

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