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 14:47:05 GMT 2023
Edited
by admin
on Sat Dec 16 14:47:05 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 a
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) EXPRESSING THE PADUA VARIANT (R338L) OF HUMAN COAGULATION FACTOR IX
Part WHOLE
Record UNII
413EU9081Y
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Fidanacogene Elaparvovec
INN  
Official Name English
SPK-9001
Code English
PF-06838435
Common Name English
SPK-1001
Code English
fidanacogene elaparvovec [INN]
Common Name English
FIDANACOGENE ELAPARVOVEC [USAN]
Common Name English
SPK-FIX PADUA
Common Name English
A NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) EXPRESSING THE PADUA VARIANT (R338L) OF HUMAN COAGULATION FACTOR IX (F9, FACTOR IX, FIX), UNDER THE CONTROL OF THE LIVER-SPECIFIC APOLIPOPROTEIN E (APO E) ENHANCER/ALPHA1-ANTITRYPSIN (HAAT) PROMO
Common Name English
SPARK 101
Common Name English
DNA (SYNTHETIC ADENO-ASSOCIATED VIRUS VECTOR AAV-SPARK100-HFIX39-PADUA)
Systematic Name English
RAAV-SPARK100-HFIX-PADUA
Common Name English
AAV8 HFIX19
Common Name English
Fidanacogene elaparvovec [WHO-DD]
Common Name English
Classification Tree Code System Code
EU-Orphan Drug EU/3/18/2090
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
Code System Code Type Description
INN
10798
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
PRIMARY
FDA UNII
413EU9081Y
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
PRIMARY
NCI_THESAURUS
C171800
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
PRIMARY
CAS
1954659-47-2
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
PRIMARY
USAN
EF-63
Created by admin on Sat Dec 16 14:47:05 GMT 2023 , Edited by admin on Sat Dec 16 14:47:05 GMT 2023
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
GENE_EXPRESSION VECTOR UNSPECIFIED SUBSTANCE
b9f1e28a
(not in database)
VECTOR EXPRESSED PROTEIN COAGULATION FACTOR IX HUMAN, (R338L)- MK6N2W9HQD