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

Approval Year

2023
446
Substance Class Structurally Diverse
Created
by admin
on Wed Apr 02 04:33:47 GMT 2025
Edited
by admin
on Wed Apr 02 04:33:47 GMT 2025
Source Materials Class ORGANISM
Source Materials Type AUTOLOGOUS HUMAN
Source Materials State LIVE
Source Materials Parent
HOMO SAPIENS WHOLE
Part bone-marrow
Fraction Name hematopoietic stem and progenitor cells (hspc)
Record UNII
S53L777GM8
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
EXAGAMGLOGENE AUTOTEMCEL
USAN   INN  
Official Name English
CTX-001
Preferred Name English
CTX001
Code English
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA)
Common Name English
Exagamglogene autotemcel [WHO-DD]
Common Name English
EXAGAMGLOGENE AUTOTEMCEL [USAN]
Common Name English
exagamglogene autotemcel [INN]
Common Name English
Code System Code Type Description
NCI_THESAURUS
C169042
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
SMS_ID
100000183615
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
FDA UNII
S53L777GM8
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
USAN
KL-107
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
INN
11560
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
DRUG BANK
DB15572
Created by admin on Wed Apr 02 04:33:47 GMT 2025 , Edited by admin on Wed Apr 02 04:33:47 GMT 2025
PRIMARY
Related Record Type Details
EXAGAMGLOGENE AUTOTEMCEL
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
CULTURE CD34+ ENRICHMENT IMMUNOLOGICAL

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
CELLULAR PROTEIN EXPRESSION INCREASED HEMOGLOBIN F 43F9JP2P5Z
NIH
NCATS
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