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 15:13:29 GMT 2023
Edited
by admin
on Sat Dec 16 15:13:29 GMT 2023
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 rAAV Rep2-CapS3-hFIX(R338L)
Part WHOLE
Record UNII
KT8AC75KUX
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
VERBRINACOGENE SETPARVOVEC
INN   USAN  
Official Name English
FLT180A
Code English
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS AAVS3-FLT180A VECTOR HUMAN BLOOD-COAGULATION FACTOR IX PADUA-SPECIFYING)
Common Name English
FLT-180A
Code English
verbrinacogene setparvovec [INN]
Common Name English
VERBRINACOGENE SETPARVOVEC [USAN]
Common Name English
A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS OF A MODIFIED LIVER-TROPIC SEROTYPE (S3) (RAAV REP2-CAPS3) VECTOR, ENCODING A GAIN-OF-FUNCTION VARIANT (R338LPADUA) OF CODON-OPTIMISED HUMAN COAGULATION FACTOR IX (HFIX) UNDER THE CONTROL OF A LIVER-SP
Common Name English
Code System Code Type Description
FDA UNII
KT8AC75KUX
Created by admin on Sat Dec 16 15:13:29 GMT 2023 , Edited by admin on Sat Dec 16 15:13:29 GMT 2023
PRIMARY
INN
11278
Created by admin on Sat Dec 16 15:13:29 GMT 2023 , Edited by admin on Sat Dec 16 15:13:29 GMT 2023
PRIMARY
CAS
2364604-87-3
Created by admin on Sat Dec 16 15:13:29 GMT 2023 , Edited by admin on Sat Dec 16 15:13:29 GMT 2023
PRIMARY
USAN
HI-191
Created by admin on Sat Dec 16 15:13:29 GMT 2023 , Edited by admin on Sat Dec 16 15:13:29 GMT 2023
PRIMARY
NCI_THESAURUS
C175872
Created by admin on Sat Dec 16 15:13:29 GMT 2023 , Edited by admin on Sat Dec 16 15:13:29 GMT 2023
PRIMARY
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR VERBRINACOGENE SETPARVOVEC TRANSGENE INSERT FEA6RKL4OX