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 15:30:34 GMT 2023
Edited
by admin
on Sat Dec 16 15:30:34 GMT 2023
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependoparvovirus
Organism Species adeno-associated dependoparvovirus
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name AAV5-ENCODING A MICRORNA TARGETED TO EXON 1 OF THE HUMAN HUNTINGTIN GENE
Part WHOLE
Record UNII
VB2ZC1P6P4
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
IFEZUNTIRGENE INILPARVOVEC
INN  
Official Name English
DNA (RECOMBINANT REPLICATION-DEFICIENT ADENO-ASSOCIATED VIRUS 5 VECTOR AMT-130 CHICKEN .BETA.-ACTIN PROMOTER PLUS HUMAN HUNTINGTIN MRNA-TARGETING MICRORNA MIHTT-SPECIFYING)
Common Name English
A RECOMBINANT, REPLICATION DEFICIENT ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING A MICRORNA (MIRNA) TARGETING THE EXON 1 OF HUMAN HUNTINGTIN PROTEIN (HTT) MRNA (MIHTT). THE EXPRESSION CASSETTE CONSISTS OF A CHIMERIC CHICKEN BETA-ACTIN PROMOT
Common Name English
ifezuntirgene inilparvovec [INN]
Common Name English
Code System Code Type Description
CLINICAL_TRIALS.GOV
NCT04120493
Created by admin on Sat Dec 16 15:30:34 GMT 2023 , Edited by admin on Sat Dec 16 15:30:34 GMT 2023
NON-SPECIFIC SUBSTITUTION
FDA UNII
VB2ZC1P6P4
Created by admin on Sat Dec 16 15:30:34 GMT 2023 , Edited by admin on Sat Dec 16 15:30:34 GMT 2023
PRIMARY
CAS
2407582-19-6
Created by admin on Sat Dec 16 15:30:34 GMT 2023 , Edited by admin on Sat Dec 16 15:30:34 GMT 2023
PRIMARY
INN
11711
Created by admin on Sat Dec 16 15:30:34 GMT 2023 , Edited by admin on Sat Dec 16 15:30:34 GMT 2023
PRIMARY
NCI_THESAURUS
C184855
Created by admin on Sat Dec 16 15:30:34 GMT 2023 , Edited by admin on Sat Dec 16 15:30:34 GMT 2023
PRIMARY
Related Record Type Details
TARGET -> INHIBITOR
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR IFEZUNTIRGENE INILPARVOVEC TRANSGENE INSERT 28QQ2R92JD