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 Wed Apr 02 08:40:19 GMT 2025
Edited
by admin
on Wed Apr 02 08:40:19 GMT 2025
Source Materials Class ORGANISM
Source Materials Type VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependovirus
Organism Species adeno-associated virus
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name rAAV2 4D-C102.CAG-cohGLA
Part whole
Record UNII
K24R94BC3K
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Duvalgagene Otiparvovec
INN  
Official Name English
4D-310
Preferred Name English
DUVALGAGENE OTIPARVOVEC [USAN]
Common Name English
duvalgagene otiparvovec [INN]
Common Name English
4D-C102
Code English
DNA (recombinant replication-defective adeno-associated virus 2 vector 4D-310 cytomegalovirus immediate-early enhancer element plus chicken ?-actin exon 1 plus intron 1 plus rabbit ?-globin splice acceptor plus human ?-galactosidase A codon-optimized gen
Common Name English
recombinant, non-replicating adeno-associated virus serotype 2 variant 4D-C102 (rAAV2 [4D-C102]) vector encoding codon optimized human alpha-galactosidase A (GLA) under control of a CAG promoter (cytomegalovirus (CMV) immediate-early enhancer/chicken ?-a
Common Name English
Code System Code Type Description
INN
12386
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
FDA UNII
K24R94BC3K
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
CAS
2768102-81-2
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
NCI_THESAURUS
C206902
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
USAN
NO-23
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
SMS_ID
300000039586
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
PRIMARY
INN
12860
Created by admin on Wed Apr 02 08:40:19 GMT 2025 , Edited by admin on Wed Apr 02 08:40:19 GMT 2025
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
GENE_EXPRESSION VECTOR UNSPECIFIED SUBSTANCE
5b3d6d1e
(not in database)
VIRAL CAPSID UNSPECIFIED SUBSTANCE
784ba655
(not in database)
VIRAL CAPSID UNSPECIFIED SUBSTANCE
177d3744
(not in database)