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

Approval Year

Substance Class Nucleic Acid
Created
by admin
on Wed Apr 02 06:38:49 GMT 2025
Edited
by admin
on Wed Apr 02 06:38:49 GMT 2025
Nucleic Acid Type OLIGONUCLEOTIDE
Sequence Origin SYNTHETIC
Sequence Type COMPLETE
Record UNII
ABG25J5B9G
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
CHIMERIC LOCKED NUCLEIC ACID AND RIBONUCLEIC-DEOXYRIBONUCLEIC ANTISENSE OLIGONUCLEOTIDE SPECIFIC FOR THE HUMAN UBE3A-ANTISENSE TRANSCRIPT
Preferred Name English
Apazunersen
INN  
Official Name English
apazunersen [INN]
Common Name English
all-P-ambo-2'-O,4'-C-methylene-P-thioadenylyl-(3'?5')-2'-O,4'-C-methylene-P-thioguanylyl-(3'?5')-2'-O,4'-C-methylene-P-thioadenylyl-(3'?5')-2'-deoxy-P-thioadenylyl-(3'?5')-P-thiothymidylyl-(3'?5')-2'-deoxy-P-thioguanylyl-(3'?5')-2'-deoxy-P-thioguanylyl-(
Common Name English
DNA, d(P-thio)((2?-O,4?-C-methylene)rA-(2?-O,4?-C-methylene)rG-(2?-O,4?-C-methylene)rA-A-T-G-G-C-A-C-A-T-C-T-(2?-O,4?-C-methylene)m5rC-(2?-O,4?-C-methylene)m5rU-(2?-O,4?-C-methylene)m5rU-(2?-O,4?-C-methylene)rG)
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 702619
Created by admin on Wed Apr 02 06:38:50 GMT 2025 , Edited by admin on Wed Apr 02 06:38:50 GMT 2025
Code System Code Type Description
INN
13469
Created by admin on Wed Apr 02 06:38:49 GMT 2025 , Edited by admin on Wed Apr 02 06:38:49 GMT 2025
PRIMARY
FDA UNII
ABG25J5B9G
Created by admin on Wed Apr 02 06:38:50 GMT 2025 , Edited by admin on Wed Apr 02 06:38:50 GMT 2025
PRIMARY
CAS
2919324-01-7
Created by admin on Wed Apr 02 06:38:50 GMT 2025 , Edited by admin on Wed Apr 02 06:38:50 GMT 2025
PRIMARY
Sugar Site Range Site Count
2,'4'-locked-Ribose (alpha-L-LNA) 1_1-1_3;1_15-1_18 7 / 18
Deoxyribose (DNA) 1_4-1_14 11 / 18
Linkage Site Range Site Count
Phosporothioate 1_2-1_18 17 / 17
Related Record Type Details
TARGET->INHIBITOR OF EXPRESSION
Related Record Type Details
ACTIVE MOIETY