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 Wed Apr 02 20:08:45 GMT 2025
Edited
by admin
on Wed Apr 02 20:08:45 GMT 2025
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 AAV-SMN1-GeneBeam v2020-03-11
Part whole
Record UNII
2Q4YZ4G35L
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Pasemnogene Gleparvovec
INN  
Official Name English
Capsomere, recombinant adeno-associated virus 9 viral capsid protein VP1 [422-tryptophan,446-phenylalanine] complex with viral capsid protein VP2 [285-tryptophan,309-phenylalanine] complex with viral capsid protein VP3 [220-tryptophan,244-phenylalanine]
Preferred Name English
recombinant non-replicating adeno-associated virus serotype 9 (AAV9) vector, encoding a codon-optimised human survival of motor neuron 1 (SMN1) gene, under control of the cytomegalovirus (CMV) enhancer/promoter and terminated by a human growth hormone po
Common Name English
pasemnogene gleparvovec [INN]
Common Name English
Code System Code Type Description
FDA UNII
2Q4YZ4G35L
Created by admin on Wed Apr 02 20:08:45 GMT 2025 , Edited by admin on Wed Apr 02 20:08:45 GMT 2025
PRIMARY
CAS
2913126-89-1
Created by admin on Wed Apr 02 20:08:45 GMT 2025 , Edited by admin on Wed Apr 02 20:08:45 GMT 2025
PRIMARY
NCI_THESAURUS
C211721
Created by admin on Wed Apr 02 20:08:45 GMT 2025 , Edited by admin on Wed Apr 02 20:08:45 GMT 2025
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 Pasemnogene gleparvovec transgene insert encoding codon-optimised human survival of motor neuron 1 (SMN1) gene JZW2TY9A6W
VIRAL CAPSID Pasemnogene gleparvovec capsid F9M4Q9QC7M