ZILEBESIRAN SODIUM

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National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 12:08:02 GMT 2025` Edited by `admin` on `Wed Apr 02 12:08:02 GMT 2025`
Nucleic Acid Type	RNAI
Sequence Type	COMPLETE
Record UNII	A8P3J9MPQ3
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ZILEBESIRAN SODIUM

Official Name

English

ALN-AGT01 SODIUM

Preferred Name

English

RNA, (Gm-sp-Um-sp-Cm-Am-Um-Cm-(2'-deoxy-2'-fluoro)C-Am-(2'-deoxy-2'-fluoro)C- (2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)A-Um-Gm-Am-Gm-Am-Gm-Um-Am-Cm-Am), 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-?-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3- [[5-[[2-(ac

Common Name

English

[(2S,4R)-1-{1-[(2-acetamido-2-deoxy-?-D-galactopyranosyl)oxy]-16,16-bis({3-[(3-{5-[(2- acetamido-2-deoxy-?-D-galactopyranosyl)oxy]pentanamido}propyl)amino]-3- oxopropoxy}methyl)-5,11,18-trioxo-14-oxa-6,10,17- triazanonacosan-29-oyl}-4- hydroxypyrrolidin-

Common Name

English

ZILEBESIRAN SODIUM [USAN]

Common Name

English

Code System Code Type Description

NCI_THESAURUS

C188750

Created by admin on Wed Apr 02 12:08:02 GMT 2025 , Edited by admin on Wed Apr 02 12:08:02 GMT 2025

PRIMARY

SMS_ID

300000045532

Created by admin on Wed Apr 02 12:08:02 GMT 2025 , Edited by admin on Wed Apr 02 12:08:02 GMT 2025

PRIMARY

USAN

KL-81

Created by admin on Wed Apr 02 12:08:02 GMT 2025 , Edited by admin on Wed Apr 02 12:08:02 GMT 2025

PRIMARY

FDA UNII

A8P3J9MPQ3

Created by admin on Wed Apr 02 12:08:02 GMT 2025 , Edited by admin on Wed Apr 02 12:08:02 GMT 2025

PRIMARY

CAS

2380166-34-5

Created by admin on Wed Apr 02 12:08:02 GMT 2025 , Edited by admin on Wed Apr 02 12:08:02 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-Fluoro-Ribose	1_2;1_14;1_16;2_7;2_9-2_11	7 / 44
2'-O-Methyl-Ribose	1_1;1_3-1_13;1_15;1_17-1_23;2_1-2_6;2_8;2_12-2_21	37 / 44

Linkage	Site Range	Site Count
Sodium Phosporothioate	1_2-1_3;1_22-1_23;2_2-2_3	6 / 42
	1_4-1_21;2_4-2_21	36 / 42

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Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[2_21]				ZILEBESIRAN 3'-ADENOSINE MODIFICATION SODIUM	DJ674LG53M
NUCLEOTIDE SUBSTITUTION	[1_6]				UNSPECIFIED SUBSTANCE	a44a79de (not in database)