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 16:26:30 GMT 2023
Edited
by admin
on Sat Dec 16 16:26:30 GMT 2023
Source Materials Class ORGANISM
Source Materials Type 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 AAV9.CB7.HIDS
Part whole
Record UNII
K5LW2WBP48
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Clemidsogene Lanparvovec
INN  
Official Name English
Recombinant, non-replicating adeno-associated virus serotype 9 (rAAV9) vector encoding human iduronate 2-sulfatase (IDS, α-L-iduronate sulfate sulfatase) under control of a human cytomegalovirus (CMV) immediate-early enhancer/chicken β-actin (CBA) prom
Common Name English
clemidsogene lanparvovec [INN]
Common Name English
DNA (recombinant replication-defective adeno-associated virus 9 vector RGX-121 cytomegalovirus immediate-early promoter-enhancer plus chicken β-actin promoter plus chicken β-actin intron plus human iduronate-2-sulfatase gene plus rabbit β-globin polya
Common Name English
Code System Code Type Description
CAS
2766142-50-9
Created by admin on Sat Dec 16 16:26:30 GMT 2023 , Edited by admin on Sat Dec 16 16:26:30 GMT 2023
PRIMARY
FDA UNII
K5LW2WBP48
Created by admin on Sat Dec 16 16:26:30 GMT 2023 , Edited by admin on Sat Dec 16 16:26:30 GMT 2023
PRIMARY
INN
12682
Created by admin on Sat Dec 16 16:26:30 GMT 2023 , Edited by admin on Sat Dec 16 16:26:30 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 Clemidsogene lanparvovec transgene insert XAV5AZB9QQ