U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Unknown
149124
Substance Class Structurally Diverse
Created
by admin
on Wed Apr 02 10:53:22 GMT 2025
Edited
by admin
on Wed Apr 02 10:53:22 GMT 2025
Source Materials Class ORGANISM
Source Materials Type RECOMBINANT VIRUS
Organism Family Parvoviridae
Organism Genus Dependovirus
Organism Species adeno-associated virus
Author NONE
Infraspecific Type SUBSTRAIN
Infraspecific Name AAV8-RPGR
Part whole
Record UNII
V1R4T1S1M0
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
A RECOMBINANT REPLICATION-DEFECTIVE ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 (RAAV8) ENCODING CODON-OPTIMISED HUMAN RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR), UNDER THE CONTROL OF HUMAN RHODOPSIN KINASE (RK) PROMOTER AND BOVINE GROWTH HORMONE POLY(A)
Preferred Name English
COTORETIGENE TOLIPARVOVEC
INN   USAN  
Official Name English
RECOMBINANT SEROTYPE 8 ADENO-ASSOCIATED VIRAL VECTOR (AAV8) ENCODING A CODON OPTIMISED (CO) COPY OF THE CDNA ENCODING HUMAN RETINITIS PIGMENTOSA GTPASE REGULATOR (CORPGR)
Common Name English
AAV8-RPGR
Common Name English
cotoretigene toliparvovec [INN]
Common Name English
COTORETIGENE TOLIPARVOVEC [USAN]
Common Name English
Code System Code Type Description
INN
11358
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
SMS_ID
100000181881
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
FDA UNII
V1R4T1S1M0
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
CAS
2246424-95-1
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
NCI_THESAURUS
C177095
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
USAN
GH-110
Created by admin on Wed Apr 02 10:53:22 GMT 2025 , Edited by admin on Wed Apr 02 10:53:22 GMT 2025
PRIMARY
Related Record Type Details
COTORETIGENE TOLIPARVOVEC
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
VECTOR EXPRESSED PROTEIN X-LINKED RETINITIS PIGMENTOSA GTPASE REGULATOR ORF15 L0031JC5I7
INSERTED GENE EXPRESSION VECTOR COTORETIGENE TOLIPARVOVEC TRANSGENE INSERT VEC94OC288
NIH
NCATS
PRIVACY NOTICE
ACCESSIBILITY
DISCLAIMER
HHS VULNERABILITY DISCLOSURE
For language access assistance, contact the NCATS Public Information Officer
National Center for Advancing Translational Sciences (NCATS),
6701 Democracy Boulevard, Bethesda MD 20892-4874 • 301-594-8966