Senvesiran

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 19:46:37 GMT 2025` Edited by `admin` on `Wed Apr 02 19:46:37 GMT 2025`
Nucleic Acid Type	RNAI
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	C7FGP2Y3GM
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Senvesiran

Official Name

English

RNA, (Am-sp-dC-sp-Gm-Am-dA-Um-dC-Um-Cm-Am-Gm-dT-Gm-(2?-deoxy-2?-fluoro)C-Um-Um-Cm-Cm-Um-Gm-Cm-sp-Am-sp-Cm), complex with RNA (Gm-sp-Cm-sp-Am-Gm-Gm-Am-Am-Gm-(2?-deoxy-2?-fluoro)C-(2?-deoxy-

Preferred 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-oxopropyl}methyl)-5,11,18-trioxo-14-oxa-6,10,17-triazanonacosan-29-oyl)-4-hydroxypyrrolidin-2-

Common Name

English

senvesiran [INN]

Common Name

English

Code System Code Type Description

INN

13106

Created by admin on Wed Apr 02 19:46:37 GMT 2025 , Edited by admin on Wed Apr 02 19:46:37 GMT 2025

PRIMARY

CAS

2894810-55-8

Created by admin on Wed Apr 02 19:46:37 GMT 2025 , Edited by admin on Wed Apr 02 19:46:37 GMT 2025

PRIMARY

FDA UNII

C7FGP2Y3GM

Created by admin on Wed Apr 02 19:46:37 GMT 2025 , Edited by admin on Wed Apr 02 19:46:37 GMT 2025

PRIMARY

NCI_THESAURUS

C213394

Created by admin on Wed Apr 02 19:46:37 GMT 2025 , Edited by admin on Wed Apr 02 19:46:37 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_1-1_6;1_8-1_11;1_13;1_15-1_23;2_1-2_8;2_12-2_21	38 / 44
Deoxyribose (DNA)	1_7;1_12	2 / 44
2'-Fluoro-Ribose	1_14;2_9-2_11	4 / 44

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

Related Record Type Details


					C7FGP2Y3GM

Senvesiran

ACTIVE MOIETY

Comments:

ALN-KHK is specifically designed to reduce the hepatic synthesis of ketohexokinase (KHK) protein. By reducing expression of KHK, it is postulated that ALN-KHK will reduce fatty acid synthesis, metabolic dysfunction-associated steatotic liver disease, and thus reduce insulin resistance and improve overall glycemic control in obese individuals with T2DM

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[2_21]	3'-TERMINUS	U		UNSPECIFIED SUBSTANCE	c6b500e6 (not in database)