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 20:01:04 GMT 2025
Edited
by admin
on Wed Apr 02 20:01:04 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 rAAV843/codon-optimised FIX
Part whole
Record UNII
XHE4MDR8C6
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Dalnacogene Ponparvovec
INN  
Official Name English
DNA (recombinant replication-defective adeno-associated virus 2 vector recombinant liver-specific promoter Lxp2.1 plus human DNA-dependent RNA polymerase II gene intron 1 plus human blood-coagulation factor IX Padua codon-optimized gene 5?-region plus hu
Preferred Name English
rAAV843 vector
Common Name English
recombinant, self-complementary, non-replicating adenoassociated virus, synthetic liver-tropic serotype 843 (rAAV843) vector, encoding a codon-optimised (CpG reduced) gain of function variant (Padua variant; R338L) of human coagulation factor IX (FIX), u
Common Name English
dalnacogene ponparvovec [INN]
Common Name English
Code System Code Type Description
FDA UNII
XHE4MDR8C6
Created by admin on Wed Apr 02 20:01:04 GMT 2025 , Edited by admin on Wed Apr 02 20:01:04 GMT 2025
PRIMARY
INN
12774
Created by admin on Wed Apr 02 20:01:04 GMT 2025 , Edited by admin on Wed Apr 02 20:01:04 GMT 2025
PRIMARY
CAS
2770683-67-3
Created by admin on Wed Apr 02 20:01:04 GMT 2025 , Edited by admin on Wed Apr 02 20:01:04 GMT 2025
PRIMARY
NCI_THESAURUS
C211819
Created by admin on Wed Apr 02 20:01:04 GMT 2025 , Edited by admin on Wed Apr 02 20:01:04 GMT 2025
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY
gene therapy (hemophilia B)

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR Dalnacogene ponparvovec transgene insert HX2RYV9JXP