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 01:11:30 GMT 2025
Edited
by admin
on Wed Apr 02 01:11:30 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 ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) EXPRESSING THE PADUA VARIANT (R338L) OF HUMAN COAGULATION FACTOR IX
Part whole
Record UNII
Z5XCD5Q9RL
Record Status Validated (UNII)
Record Version
  • Download
Related Record Type
Name Type Language
Etranacogene Dezaparvovec
INN   USAN  
Official Name English
AAV5-hFIXco-Padua
Preferred Name English
etranacogene dezaparvovec [INN]
Common Name English
ETRANACOGENE DEZAPARVOVEC [USAN]
Common Name English
Etranacogene dezaparvovec [WHO-DD]
Common Name English
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) EXPRESSING THE PADUA VARIANT (R338L) OF HUMAN COAGULAN FACTOR IX (F9, FACTOR IX, OR FIX), UNDER THE CONTROL OF THE LIVER-SPECIFIC PROMOTER (LP1)THE GENOMIC INVERTED TERMINAL REPEATS (IT
Systematic Name English
AMT-061
Code English
Code System Code Type Description
CAS
2156583-26-3
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
INN
10963
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
FDA UNII
Z5XCD5Q9RL
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
NCI_THESAURUS
C171798
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
USAN
JK-202
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
SMS_ID
300000005896
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
DAILYMED
Z5XCD5Q9RL
Created by admin on Wed Apr 02 01:11:30 GMT 2025 , Edited by admin on Wed Apr 02 01:11:30 GMT 2025
PRIMARY
Related Record Type Details
SUB_CONCEPT->SUBSTANCE
TARGET->GENE REPLACEMENT
EXPRESSED PROTEIN->VECTOR
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 ETRANACOGENE DEZAPARVOVEC DNA INSERT EI983WK411