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 11:50:51 GMT 2025
Edited
by admin
on Wed Apr 02 11:50:51 GMT 2025
Source Materials Class ORGANISM
Source Materials Type VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependoparvovirus
Organism Species adeno associated virus
Infraspecific Type SUBSTRAIN
Infraspecific Name AAVhu68
Part whole
Record UNII
EXB5VWG5TW
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
DNA (recombinant replication-defective adeno-associated virus serotype hu68 vector PBGM01 human ubiquitin C promoter plus chimeric chicken ?-actin and human ?-globin intron plus human ?-galactosidase codon-optimized gene GLB1 plus simian virus 40 polyade
Preferred Name English
Seglebegagene Dasniparvovec
INN  
Official Name English
seglebegagene dasniparvovec [INN]
Common Name English
recombinant, non-replicating adeno-associated virus serotype hu68 (rAAVhu68) vector encoding codon-optimised human ?-galactosidase (GLB1), under control of the human ubiquitin C promoter plus a chimeric intron, consisting of human ?-globin splice donor a
Common Name English
Code System Code Type Description
INN
12225
Created by admin on Wed Apr 02 11:50:51 GMT 2025 , Edited by admin on Wed Apr 02 11:50:51 GMT 2025
PRIMARY
NCI_THESAURUS
C190438
Created by admin on Wed Apr 02 11:50:51 GMT 2025 , Edited by admin on Wed Apr 02 11:50:51 GMT 2025
PRIMARY
SMS_ID
300000021716
Created by admin on Wed Apr 02 11:50:51 GMT 2025 , Edited by admin on Wed Apr 02 11:50:51 GMT 2025
PRIMARY
CAS
2607029-99-0
Created by admin on Wed Apr 02 11:50:51 GMT 2025 , Edited by admin on Wed Apr 02 11:50:51 GMT 2025
PRIMARY
FDA UNII
EXB5VWG5TW
Created by admin on Wed Apr 02 11:50:51 GMT 2025 , Edited by admin on Wed Apr 02 11:50:51 GMT 2025
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
TRANSGENE Seglebegagene dasniparvovec transgene insert UQX89KA9ST