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 19:13:34 GMT 2023
Edited
by admin
on Sat Dec 16 19:13:34 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 AAVhu68.CB7.CI.hGALCco.rBG
Part WHOLE
Record UNII
AFM8985FXH
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Crosigalcogene Omlixparvovec
INN  
Official Name English
DNA (recombinant replication-defective adeno-associated virus serotype hu68 vector PBKR03 cytomegalovirus enhancer fusion with chicken β-actin promoter plus chicken β-actin intron plus human galactosylceramidase codon-optimized gene GALC plus rabbit β
Common Name English
Crosigalcogene omlixparvovec (recombinant, non-replicating adeno-associated virus serotype hu68 (AAVhu68) vector encoding codon-optimised human galactosylceramidase (GALC), under control of a cytomegalovirus immediate early enhancer/chicken β-actin prom
Common Name English
crosigalcogene omlixparvovec [INN]
Common Name English
Code System Code Type Description
NCI_THESAURUS
C190372
Created by admin on Sat Dec 16 19:13:34 GMT 2023 , Edited by admin on Sat Dec 16 19:13:34 GMT 2023
PRIMARY
INN
12227
Created by admin on Sat Dec 16 19:13:34 GMT 2023 , Edited by admin on Sat Dec 16 19:13:34 GMT 2023
PRIMARY
FDA UNII
AFM8985FXH
Created by admin on Sat Dec 16 19:13:34 GMT 2023 , Edited by admin on Sat Dec 16 19:13:34 GMT 2023
PRIMARY
CAS
2607030-09-9
Created by admin on Sat Dec 16 19:13:34 GMT 2023 , Edited by admin on Sat Dec 16 19:13:34 GMT 2023
PRIMARY
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 Crosigalcogene omlixparvovec transgene insert DKX9K34DCJ