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 20:21:17 GMT 2025
Edited
by admin
on Wed Apr 02 20:21:17 GMT 2025
Nucleic Acid Type dsRNA
Sequence Origin SYNTHETIC
Sequence Type COMPLETE
Record UNII
K699R35T33
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Ozisiran
INN  
Official Name English
(2RS)-3-(1,23-bis[(2-acetamido-2-deoxy-?-D-galactopyranosyl) oxy]-14-{5-[(2-acetamido-2-deoxy-?-D-galactopyranosyl) oxy]pentanoyl}-5,19-dioxo-6,10,14,18-tetraazatricosan-10-yl)-2-hydroxy-3-oxopropyl hydrogen all-Pambo-2'-O-methyl-P-thiocytidylyl-(3'?5')-
Preferred Name English
RNA, (Cm-sp-Cm-sp-Um-Um-Gm-Am-(2?-deoxy-2?-fluoro)G-(2?-deoxy-2?-fluoro)G-(2?-deoxy-2?-fluoro)C-Am-Um-Am-Cm-Um-Um-Cm-Am-Am-Am), 3?-[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-?-D-galactopyranosyl]oxy]-1-oxopentyl][3-[[5-[[2-(acetylamino)-2-deoxy-?-D-galactopyra
Common Name English
ozisiran [INN]
Common Name English
Code System Code Type Description
INN
13490
Created by admin on Wed Apr 02 20:21:17 GMT 2025 , Edited by admin on Wed Apr 02 20:21:17 GMT 2025
PRIMARY
FDA UNII
K699R35T33
Created by admin on Wed Apr 02 20:21:17 GMT 2025 , Edited by admin on Wed Apr 02 20:21:17 GMT 2025
PRIMARY
CAS
3061406-96-7
Created by admin on Wed Apr 02 20:21:17 GMT 2025 , Edited by admin on Wed Apr 02 20:21:17 GMT 2025
PRIMARY
Sugar Site Range Site Count
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
2'-Fluoro-Ribose 1_2;1_6;1_14;1_16;2_7-2_9 7 / 40
Linkage Site Range Site Count
Phosporothioate 1_2-1_3;1_20-1_21;2_2-2_3 6 / 38
Phosphate 1_4-1_19;2_4-2_19 32 / 38
Related Record Type Details
ACTIVE MOIETY
hepatitis B virus (HBV) RNA transcript reducer, antiviral

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOTIDE SUBSTITUTION [1_1] SITE_SPECIFIC U Vinylphosphonate 2'-O-methyluridine 5V3K9SGM4U
NUCLEOTIDE SUBSTITUTION [2_19] SITE_SPECIFIC A Ozisiran 3'-adenosine modification FX4KH7KJ4V