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 16:37:22 GMT 2023
Edited
by admin
on Sat Dec 16 16:37:22 GMT 2023
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependoparvovirus
Organism Species adeno-associated dependoparvovirus a
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 8 (AAV8) VECTOR EXPRESSING HUMAN MYOTUBULARIN1 (MTM1), UNDER THE CONTROL OF THE HUMAN DESMIN GENE PROMOTER
Part WHOLE
Record UNII
F6LOJ6ER4S
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
RESAMIRIGENE BILPARVOVEC
INN   USAN  
Official Name English
AT-132
Code English
RESAMIRIGENE BILPARVOVEC [USAN]
Common Name English
resamirigene bilparvovec [INN]
Common Name English
AT001 (GENE THERAPY)
Code English
AT-001 (GENE THERAPY)
Code English
AT132
Code English
A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 8 (AAV8) VECTOR EXPRESSING HUMAN MYOTUBULARIN 1 (MTM1), UNDER THE CONTROL OF THE HUMAN DESMIN GENE PROMOTER
Systematic Name English
Code System Code Type Description
USAN
EF-155
Created by admin on Sat Dec 16 16:37:22 GMT 2023 , Edited by admin on Sat Dec 16 16:37:22 GMT 2023
PRIMARY
CAS
2085240-27-1
Created by admin on Sat Dec 16 16:37:22 GMT 2023 , Edited by admin on Sat Dec 16 16:37:22 GMT 2023
PRIMARY
NCI_THESAURUS
C177083
Created by admin on Sat Dec 16 16:37:22 GMT 2023 , Edited by admin on Sat Dec 16 16:37:22 GMT 2023
PRIMARY
FDA UNII
F6LOJ6ER4S
Created by admin on Sat Dec 16 16:37:22 GMT 2023 , Edited by admin on Sat Dec 16 16:37:22 GMT 2023
PRIMARY
INN
10920
Created by admin on Sat Dec 16 16:37:22 GMT 2023 , Edited by admin on Sat Dec 16 16:37:22 GMT 2023
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR RESAMIRIGENE BILPARVOVEC VECTOR DNA CI7K1G5HG4