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 18:04:34 GMT 2025
Edited
by admin
on Wed Apr 02 18:04:34 GMT 2025
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 a
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name 4D-A101.CMV173-CFTR?R
Part whole
Record UNII
9SG6QLJ7VJ
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Plencifgene Parvec
INN  
Official Name English
4D-710
Preferred Name English
plencifgene parvec [INN]
Common Name English
recombinant replication-defective adeno-associated virus vector 4D-710 cytomegalovirus promoter CMV173 plus human (1-707)-(760-1480)-cystic fibrosis transmembrane conductance regulator codon-optimized gene CFTR?R plus synthetic polyadenylation signal plu
Common Name English
recombinant, non-replicating adeno-associated virus (AAV) vector (with capsid variant A101) encoding codon-optimised human cystic fibrosis transmembrane conductance regulator (CFTR) with the regulatory domain deleted (CFTR?R), under control of a 173bp cy
Common Name English
Code System Code Type Description
INN
13321
Created by admin on Wed Apr 02 18:04:34 GMT 2025 , Edited by admin on Wed Apr 02 18:04:34 GMT 2025
PRIMARY
CAS
2737363-32-3
Created by admin on Wed Apr 02 18:04:34 GMT 2025 , Edited by admin on Wed Apr 02 18:04:34 GMT 2025
PRIMARY
FDA UNII
9SG6QLJ7VJ
Created by admin on Wed Apr 02 18:04:34 GMT 2025 , Edited by admin on Wed Apr 02 18:04:34 GMT 2025
PRIMARY
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 Plencifgene parvec transgene insert MNG6B6YGE4