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 15:14:36 GMT 2023
Edited
by admin
on Sat Dec 16 15:14:36 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 ADENO-ASSOCIATED VIRUS SEROTYPE 8 (AAV8) EXPRESSING THE HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) PROTEIN-TBG/AMBP-head-to-head dimer
Part WHOLE
Record UNII
20E4R0BF12
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
AVALOTCAGENE ONTAPARVOVEC
USAN   INN  
Official Name English
A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/8 (RAAV REP2-CAP8) VECTOR, ENCODING CODON-OPTIMIZED, WILD-TYPE HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) UNDER THE CONTROL OF A HYBRID HUMAN THYROXINE-BINDING GLOBULIN (TBG) / HUMAN ALPHA 1-MICROGL
Common Name English
DTX-301 (ORNITHINE-TRANSCARBAMYLASE-GENE-THERAPY)
Common Name English
DTX301
Code English
avalotcagene ontaparvovec [INN]
Common Name English
AVALOTCAGENE ONTAPARVOVEC [USAN]
Common Name English
KFB12DX3PF
Code English
SCAAV8OTC
Common Name English
DTX-301
Code English
Code System Code Type Description
FDA UNII
20E4R0BF12
Created by admin on Sat Dec 16 15:14:37 GMT 2023 , Edited by admin on Sat Dec 16 15:14:37 GMT 2023
PRIMARY
USAN
HI-242
Created by admin on Sat Dec 16 15:14:37 GMT 2023 , Edited by admin on Sat Dec 16 15:14:37 GMT 2023
PRIMARY
NCI_THESAURUS
C171792
Created by admin on Sat Dec 16 15:14:37 GMT 2023 , Edited by admin on Sat Dec 16 15:14:37 GMT 2023
PRIMARY
CAS
2227000-30-6
Created by admin on Sat Dec 16 15:14:37 GMT 2023 , Edited by admin on Sat Dec 16 15:14:37 GMT 2023
PRIMARY
INN
11153
Created by admin on Sat Dec 16 15:14:37 GMT 2023 , Edited by admin on Sat Dec 16 15:14:37 GMT 2023
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
TARGET->ENZYME REPLACEMENT
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR AVALOTCAGENE ONTAPARVOVEC TRANSGENE INSERT 6I3TA646W6