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 21:17:21 GMT 2025
Edited
by admin
on Wed Apr 02 21:17:21 GMT 2025
Nucleic Acid Type dsRNA
Sequence Origin SYNTHETIC
Sequence Type COMPLETE
Record UNII
K4A9V4HK29
Record Status FAILED
Record Version
  • Download
Name Type Language
Vortosiran
INN  
Official Name English
(2RS)-3-(1,23-bis[(2-acetamido-2-deoxy-?-Dgalactopyranosyl) oxy]-14-{5-[(2-acetamido-2-deoxy-?-Dgalactopyranosyl) oxy]pentanoyl}-5,19-dioxo-6,10,14,18-tetraazatricosan-10-yl)-2-hydroxy-3-oxopropyl hydrogen all-Pambo-2'-O-methyl-P-thioguanylyl-(3'?5')-2'-
Preferred Name English
RNA, (Cm-sp-(2?-deoxy-2?-fluoro)A-sp-Gm-Am-Am-(2?-deoxy-2?-fluoro)U-Cm-Cm-Am-Gm-Um-Cm-Cm-(2?-deoxy-2?-fluoro)A-Cm-(2?-deoxy-2?-fluoro)G-Um-Am-Cm-sp-Um-sp-Um), complex with RNA (Gm-sp-Um-sp-Am-Cm-Gm-Um-(2?-deoxy-2?-fluoro)G-(2?-deoxy-2?-fluoro)G-(2?-deoxy
Common Name English
vortosiran [INN]
Common Name English
Code System Code Type Description
CAS
3061406-99-0
Created by admin on Wed Apr 02 21:17:21 GMT 2025 , Edited by admin on Wed Apr 02 21:17:21 GMT 2025
PRIMARY
INN
13491
Created by admin on Wed Apr 02 21:17:21 GMT 2025 , Edited by admin on Wed Apr 02 21:17:21 GMT 2025
PRIMARY
FDA UNII
K4A9V4HK29
Created by admin on Wed Apr 02 21:17:21 GMT 2025 , Edited by admin on Wed Apr 02 21:17:21 GMT 2025
PRIMARY
Sugar Site Range Site Count
2'-Fluoro-Ribose 1_2;1_6;1_14;1_16;2_7-2_9 7 / 40
2'-O-Methyl-Ribose 1_1;1_3-1_5;1_7-1_13;1_15;1_17-1_21;2_1-2_6;2_10-2_19 33 / 40
Linkage Site Range Site Count
Phosporothioate 1_2-1_3;1_19-1_21;2_1-2_3 8 / 38
Phosphate 1_4-1_18;2_4-2_19 31 / 38
Related Record Type Details
TARGET->INHIBITOR OF EXPRESSION
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOTIDE SUBSTITUTION 3'-TERMINUS G Vortosiran 3'-guanosine modification Z2NK85Z4VJ