Gatuzosiran

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 15:02:07 GMT 2025` Edited by `admin` on `Wed Apr 02 15:02:07 GMT 2025`
Nucleic Acid Type	siRNA
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	K5P487RKV3
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Gatuzosiran

Official Name

English

RNA, ([1?-de(6-amino-9H-purin-9-yl)]dA-(5??5?)-sp-Cm-Gm-Um-Am-Am-Gm-Am-Am-(2?-deoxy-2?-fluoro)G-Um-(2?-deoxy-2?-fluoro)C-Um-(2?-deoxy-2?-fluoro)G-Am-Um-Am-Gm-Am-Um-Gm-Am-(3??3?)-sp-[1?-de(6-amino-9H-purin-9-yl)]dA), 3?-[O-[cis-4-[(3S,8S)-17-[[2-(acetylam

Preferred Name

English

gatuzosiran [INN]

Common Name

English

all-P-ambo-[(2R,3S)-2-(hydroxymethyl)oxolan-3-yl] hydrogen 5'-O-({[(2R,3S)-3-({[(cis-4-{(3S,8S)-17-[(2-acetamido-2-deoxy-?-Dgalactopyranosyl) oxy]-3,8-bis[(2-{2-[(2-acetamido-2-deoxy-?-Dgalactopyranosyl) oxy]ethoxy}ethyl)carbamoyl]-6,11-dioxo-15-oxa-2,7,

Common Name

English

Code System Code Type Description

INN

13156

Created by admin on Wed Apr 02 15:02:07 GMT 2025 , Edited by admin on Wed Apr 02 15:02:07 GMT 2025

PRIMARY

FDA UNII

K5P487RKV3

Created by admin on Wed Apr 02 15:02:07 GMT 2025 , Edited by admin on Wed Apr 02 15:02:07 GMT 2025

PRIMARY

CAS

2409783-34-0

Created by admin on Wed Apr 02 15:02:07 GMT 2025 , Edited by admin on Wed Apr 02 15:02:07 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-Fluoro-Ribose	1_10;1_12;1_14;2_2;2_4;2_6;2_12;2_14;2_16;2_18;2_20	11 / 44
	1_1;1_23	2 / 44
2'-O-Methyl-Ribose	1_2-1_9;1_11;1_13;1_15-1_22;2_1;2_3;2_5;2_7-2_11;2_13;2_15;2_17;2_19;2_21	31 / 44

Linkage	Site Range	Site Count
Phosporothioate	1_2;1_23;2_2-2_4;2_21	6 / 42
Phosphate	1_3-1_22;2_5-2_20	36 / 42

Related Record Type Details


					TQ3E83UKE3

17-BETA-HYDROXYSTEROID DEHYDROGENASE 13

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					K5P487RKV3

Gatuzosiran

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[1_23]	3'-TERMINUS	Abasic	Amount:	1,2-Dideoxy-D-ribofuranose 3-phosphorothioate	EKE4PS2PRF
NUCLEOTIDE INSERTIONS	[1_1]	5'-TERMINUS	Abasic	Amount:	UNSPECIFIED SUBSTANCE	cfd6533c (not in database)