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 14:42:07 GMT 2023
Edited
by admin
on Sat Dec 16 14:42:07 GMT 2023
Source Materials Class ORGANISM
Source Materials Type VIRUS
Source Materials State LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family Parvoviridae
Organism Genus Dependovirus
Organism Species adeno-associated virus
Infraspecific Type SUBSTRAIN
Infraspecific Name RAAV REP2-CAP9-CK8-H-.MU.D5
Part WHOLE
Record UNII
ASM9NQ89TK
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ZILDISTROGENE VAROPARVOVEC
INN  
Official Name English
A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/9 (RAAV REP2-CAP9) VECTOR, ENCODING HUMAN MICRODYSTROPHIN 5 (H-.MU.D5) UNDER CONTROL OF CK8 MUSCLE CREATINE KINASE PROMOTER AND ENHANCER ELEMENTS
Common Name English
zildistrogene varoparvovec [INN]
Common Name English
Code System Code Type Description
CAS
2220231-04-7
Created by admin on Sat Dec 16 14:42:07 GMT 2023 , Edited by admin on Sat Dec 16 14:42:07 GMT 2023
PRIMARY
NCI_THESAURUS
C171806
Created by admin on Sat Dec 16 14:42:07 GMT 2023 , Edited by admin on Sat Dec 16 14:42:07 GMT 2023
PRIMARY
CLINICAL_TRIALS.GOV
NCT03368742
Created by admin on Sat Dec 16 14:42:07 GMT 2023 , Edited by admin on Sat Dec 16 14:42:07 GMT 2023
PRIMARY
FDA UNII
ASM9NQ89TK
Created by admin on Sat Dec 16 14:42:07 GMT 2023 , Edited by admin on Sat Dec 16 14:42:07 GMT 2023
PRIMARY
INN
11088
Created by admin on Sat Dec 16 14:42:07 GMT 2023 , Edited by admin on Sat Dec 16 14:42:07 GMT 2023
PRIMARY
Related Record Type Details
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
INACTIVATION CHEMICAL

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR ZILDISTROGENE VAROPARVOVEC RAAV9-CK8-H-MU D5 CONSTRUCT 7MZW6BJA64