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 18:39:23 GMT 2023
Edited
by admin
on Sat Dec 16 18:39:23 GMT 2023
Source Materials Class ORGANISM
Source Materials Type AUTOLOGOUS HUMAN
Source Materials State LIVE GENETICALLY MODIFIED
Source Materials Parent
Part BONE MARROW
Fraction Name hematopoietic stem and progenitor cells
Record UNII
EPP8G99QG4
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ATIDARSAGENE AUTOTEMCEL
INN   USAN  
Official Name English
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS TRANSDUCED EX VIVO WITH A REPLICATION-DEFECTIVE LENTIVIRAL VECTOR ENCODING HUMAN ARYLSULFATASE A (ARSA
Common Name English
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPC), OBTAINED FROM BONE MARROW (BM) OR MOBILISED PERIPHERAL BLOOD (MPB), TRANSDUCED EX VIVO WITH A REPLICATION-DEFECTIVE PSEUDOTYPED HIV-1 BASED LENTIVIRAL VECTOR ENCODING THE HUMAN ARYLSULFATAS
Common Name English
atidarsagene autotemcel [INN]
Common Name English
ATIDARSAGENE AUTOTEMCEL [USAN]
Common Name English
OTL-200
Code English
Atidarsagene autotemcel [WHO-DD]
Common Name English
Code System Code Type Description
FDA UNII
EPP8G99QG4
Created by admin on Sat Dec 16 18:39:23 GMT 2023 , Edited by admin on Sat Dec 16 18:39:23 GMT 2023
PRIMARY
INN
11282
Created by admin on Sat Dec 16 18:39:23 GMT 2023 , Edited by admin on Sat Dec 16 18:39:23 GMT 2023
PRIMARY
USAN
JK-136
Created by admin on Sat Dec 16 18:39:23 GMT 2023 , Edited by admin on Sat Dec 16 18:39:23 GMT 2023
PRIMARY
NCI_THESAURUS
C180638
Created by admin on Sat Dec 16 18:39:23 GMT 2023 , Edited by admin on Sat Dec 16 18:39:23 GMT 2023
PRIMARY
Related Record Type Details
TARGET->ENZYME REPLACEMENT
Related Record Type Details
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
CULTURE CD34+ ENRICHMENT BIOLOGICAL UNSPECIFIED SUBSTANCE 9cd8c8f9
LENTIVIRAL TRANSFECTION TRANSFECTION BIOLOGICAL UNSPECIFIED SUBSTANCE f1852827

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR ATIDARSAGENE AUTOTEMCEL PROVIRUS SEQUENCE KZU38SJ6BG
VECTOR EXPRESSED PROTEIN ATIDARSAGENE AUTOTEMCEL HUMAN ARYLSULFATASE A PREPROTEIN VG44W4V6XH
VECTOR EXPRESSED PROTEIN ARYLSULFATASE A EJS32ZO2L4
Name Property Type Amount Referenced Substance Defining Parameters References
ANTIGEN EXPRESSED BIOLOGICAL
ANTIGEN EXPRESSED BIOLOGICAL
ANTIGEN NOT EXPRESSED BIOLOGICAL
ANTIGEN EXPRESSED BIOLOGICAL
AM9TU3H9K5
Unknown Structure
PROMININ-1
(not present in database)
ANTIGEN NOT EXPRESSED BIOLOGICAL
ANTIGEN NOT EXPRESSED BIOLOGICAL
ANTIGEN EXPRESSED BIOLOGICAL
ANTIGEN NOT EXPRESSED BIOLOGICAL
ANTIGEN EXPRESSED BIOLOGICAL