Nulabeglogene Autogedtemcel

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 13:50:38 GMT 2025` Edited by `admin` on `Wed Apr 02 13:50:38 GMT 2025`
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

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