Nucresiran

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NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 19:35:01 GMT 2025` Edited by `admin` on `Wed Apr 02 19:35:01 GMT 2025`
Nucleic Acid Type	siRNA
Sequence Type	COMPLETE
Record UNII	MCT9H26JQY
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

RNA, (Am-sp-(2?-deoxy-2?-fluoro)G-sp-Um-Am-Am-(2?-deoxy-2?-fluoro)A-Am-Am-Um-Gm-Gm-Am-Am-(2?-deoxy-2?-fluoro)U-Am-(2?-deoxy-2?-fluoro)C-Um-Cm-Um-Um-Gm-sp-Gm-sp-Um), complex with RNA (Cm-sp-Am-sp-Am-Gm-Am-Gm-(2?-deoxy-2?-fluoro)U-Am-(2?-deoxy-2?-fluoro)U-

Preferred Name

English

Nucresiran

Common Name

English

nucresiran [INN]

Common Name

English

all-P-ambo-2'-O-methyl-P-thiocytidylyl-(3'?5')-2'-O-methyl-Pthioadenylyl-( 3'?5')-2'-O-methyladenylyl-(3'?5')-2'-O-methylguanylyl-(3'?5')-2'-O-methyladenylyl-(3'?5')-2'-O-methylguanylyl-(3'?5')-2'-deoxy-2'-fluorouridylyl-(3'?5')-2'-O-methyladenylyl-(3'?5

Common Name

English

Code System Code Type Description

INN

12909

Created by admin on Wed Apr 02 19:35:01 GMT 2025 , Edited by admin on Wed Apr 02 19:35:01 GMT 2025

PRIMARY

NCI_THESAURUS

C206949

Created by admin on Wed Apr 02 19:35:01 GMT 2025 , Edited by admin on Wed Apr 02 19:35:01 GMT 2025

PRIMARY

CAS

2847913-45-3

Created by admin on Wed Apr 02 19:35:01 GMT 2025 , Edited by admin on Wed Apr 02 19:35:01 GMT 2025

PRIMARY

SMS_ID

300000046953

Created by admin on Wed Apr 02 19:35:01 GMT 2025 , Edited by admin on Wed Apr 02 19:35:01 GMT 2025

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FDA UNII

MCT9H26JQY

Created by admin on Wed Apr 02 19:35:01 GMT 2025 , Edited by admin on Wed Apr 02 19:35:01 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
D-Ribose (RNA)	2_22	1 / 45
2'-Fluoro-Ribose	1_2;1_6;1_14;1_16;2_7;2_9-2_11	8 / 45
2'-O-Methyl-Ribose	1_1;1_3-1_5;1_7-1_13;1_15;1_17-1_23;2_1-2_6;2_8;2_12-2_21	36 / 45

Linkage	Site Range	Site Count
Phosphate	1_4-1_21;2_4-2_22	37 / 43
Phosporothioate	1_2-1_3;1_22-1_23;2_2-2_3	6 / 43

Related Record Type Details


					2COU0B578R

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					MCT9H26JQY

ACTIVE MOIETY

Comments:

transthyretin synthesis reducer

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[2_22]	3'-TERMINUS	Abasic		CEMDISIRAN, TERMINAL SUGAR MODIFICATION-	CQQ59545BV