U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Unknown
139594
Name Type Language
Renizgamglogene Autogedtemcel
INN  
Official Name English
An investigational CRISPR/Cas12a-based gene therapy that consists of autologous CD34+ hematopoietic stem cells edited at HBG1 and HG2 promoters for potential use in treating sickle cell disease and β-thalassemia
Common Name English
renizgamglogene autogedtemcel [INN]
Common Name English
autologous human CD34+ hematopoietic stem/precursor cells (HSPCs), obtained from peripheral blood by leukapheresis from plerixafor-mobilized sickle cell disease (SCD) patients or granulocyte colony stimulating factor (G-CSF) and plerixafor-mobilized tran
Common Name English
EDIT 301
Code English
EDIT-301 CD34+ hematopoietic stem cells
Common Name English
EDIT-301
Code English
Code System Code Type Description
INN
12733
Created by admin on Sat Dec 16 19:50:21 GMT 2023 , Edited by admin on Sat Dec 16 19:50:21 GMT 2023
PRIMARY
CAS
2923537-23-7
Created by admin on Sat Dec 16 19:50:21 GMT 2023 , Edited by admin on Sat Dec 16 19:50:21 GMT 2023
PRIMARY
FDA UNII
4A4FLA6DZA
Created by admin on Sat Dec 16 19:50:21 GMT 2023 , Edited by admin on Sat Dec 16 19:50:21 GMT 2023
PRIMARY
NIH
NCATS
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National Center for Advancing Translational Sciences (NCATS),
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