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 16:18:21 GMT 2023
Edited
by admin
on Sat Dec 16 16:18:21 GMT 2023
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependovirus
Organism Species adeno-associated virus
Author NONE
Infraspecific Type SUBSTRAIN
Infraspecific Name RAAV8G6PASE
Part whole
Record UNII
7LP92X4WPE
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
PARIGLASGENE BRECAPARVOVEC
USAN   INN  
Official Name English
AAV8G6PASE
Code English
DTX401
Code English
pariglasgene brecaparvovec [INN]
Common Name English
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 8 VECTOR DTX401 HUMAN GLUCOSE 6-PHOSPHATASE .ALPHA. CODON-OPTIMIZED GENE G6PC PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS)
Common Name English
PARIGLASGENE BRECAPARVOVEC [USAN]
Common Name English
A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 (RAAV8), ENCODING CODON-OPTIMIZED HUMAN GLUCOSE-6-PHOSPHATASE (G6PC) UNDER THE CONTROL OF THE NATIVE HUMAN G6PC PROMOTER/ENHANCER AND AN SV40 POLYADENYLATION SEQUENCE, CONTAINING A CH
Common Name English
DTX-401
Code English
Code System Code Type Description
NCI_THESAURUS
C177070
Created by admin on Sat Dec 16 16:18:21 GMT 2023 , Edited by admin on Sat Dec 16 16:18:21 GMT 2023
PRIMARY
CAS
2259927-72-3
Created by admin on Sat Dec 16 16:18:21 GMT 2023 , Edited by admin on Sat Dec 16 16:18:21 GMT 2023
PRIMARY
USAN
JK-221
Created by admin on Sat Dec 16 16:18:21 GMT 2023 , Edited by admin on Sat Dec 16 16:18:21 GMT 2023
PRIMARY
INN
11440
Created by admin on Sat Dec 16 16:18:21 GMT 2023 , Edited by admin on Sat Dec 16 16:18:21 GMT 2023
PRIMARY
FDA UNII
7LP92X4WPE
Created by admin on Sat Dec 16 16:18:21 GMT 2023 , Edited by admin on Sat Dec 16 16:18:21 GMT 2023
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY
for the treatment of glycogen storage disease type Ia (GSDIa)

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
VECTOR EXPRESSED PROTEIN GLUCOSE-6-PHOSPHATASE 0RJV840J55