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Approval Year

Unknown
139594
Name Type Language
Nulabeglogene Autogedtemcel
USAN   INN  
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
NIH
NCATS
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