U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Unknown
149124
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
NIH
NCATS
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