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

Approval Year

Substance Class Structurally Diverse
Created
by admin
on Sat Dec 16 15:05:58 GMT 2023
Edited
by admin
on Sat Dec 16 15:05:58 GMT 2023
Source Materials Class ORGANISM
Source Materials Type AUTOLOGOUS HUMAN
Source Materials State LIVE
Source Materials Parent
Part BONE MARROW
Fraction Material Type CELLS
Fraction Name Hematopoietic Stem and Progenitor Cells
Record UNII
MOD3RG31RS
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
SIMOLADAGENE AUTOTEMCEL
USAN   INN  
Official Name English
CELL BASED DRUG PRODUCT CONSISTING OF CELLS THAT HAVE BEEN GENETICALLY MODIFIED EX VIVO. IT INCLUDES AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS (HSCS) FROM SEVERE COMBINED IMMUNODEFICIENCY DUE TO ADENOSINE DEAMINASE DEFICIENCY (ADA-SCID) SUBJECTS TRANSDUC
Common Name English
OTL-101
Code English
SIMOLADAGENE AUTOTEMCEL [USAN]
Common Name English
simoladagene autotemcel [INN]
Common Name English
Simoladagene autotemcel [WHO-DD]
Common Name English
Code System Code Type Description
FDA UNII
MOD3RG31RS
Created by admin on Sat Dec 16 15:05:58 GMT 2023 , Edited by admin on Sat Dec 16 15:05:58 GMT 2023
PRIMARY
INN
11042
Created by admin on Sat Dec 16 15:05:58 GMT 2023 , Edited by admin on Sat Dec 16 15:05:58 GMT 2023
PRIMARY
USAN
FG-30
Created by admin on Sat Dec 16 15:05:58 GMT 2023 , Edited by admin on Sat Dec 16 15:05:58 GMT 2023
PRIMARY
NCI_THESAURUS
C171810
Created by admin on Sat Dec 16 15:05:58 GMT 2023 , Edited by admin on Sat Dec 16 15:05:58 GMT 2023
PRIMARY
Related Record Type Details
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
CULTURE Pre-stimulation IMMUNOLOGICAL RECOMBINANT HUMAN THROMBOPOIETIN E2E529W34D
CD34 Selection CELL SEPARATION
CULTURE IMMUNOLOGICAL FLT-3 LIGAND PROTEIN (THR27-PRO185; INSECT GLYCOSYLATION) 4F6H64K1NO
TRANSFECTION LENTIVIRAL TRANSFECTION SIMOLADAGENE AUTOTEMCEL LENTIVIRAL VECTOR DN654H6FDU
RBC depletion CELL SEPARATION
CULTURE Pre-stimulation IMMUNOLOGICAL MUPLESTIM A802907MI4

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
VECTOR EXPRESSED PROTEIN ADENOSINE DEAMINASE XPQ5L1L4WO