Lepodisiran

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NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 13:50:38 GMT 2025` Edited by `admin` on `Wed Apr 02 13:50:38 GMT 2025`
Nucleic Acid Type	siRNA
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	X4BE7S8PN9
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

LY-3819469 FREE ACID

Preferred Name

English

Lepodisiran

Official Name

English

duplex of all-P-ambo-4?-de(hydroxymethyl)-4?-[(hydroxymethylphosphinoyl)methoxy]-2?-O-methylthiouridylyl-2?-deoxy-2?-fluorothioadenylyl-(3'?5')-2?-deoxy-2?-fluorothioguanylyl-(3'?5')-2?-deoxy-2?-fluoroadenylyl-(3'?5')-2?-deoxy-2?-fluorouridylyl-(3'?5')-2

Common Name

English

RNA, ([4?-de(hydroxymethyl)-4?-[(hydroxymethoxyphosphinyl)methoxy]]Um-sp-(2?-deoxy-2?-fluoro)A-sp-(2?-deoxy-2?-fluoro)G-sp-(2?-deoxy-2?-fluoro)A-(2?-deoxy-2?-fluoro)U-Gm-(2?-deoxy-2?-fluoro)A-Cm-Cm-(2?-deoxy-2?-fluoro)A-Am-Gm-Cm-(2?-deoxy-2?-fluoro)U-Um-

Common Name

English

Lepodisiran [WHO-DD]

Common Name

English

LY3819469 FREE ACID

Code

English

lepodisiran [INN]

Common Name

English

LEPODISIRAN [USAN]

Common Name

English

Code System Code Type Description

INN

13068

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

NCI_THESAURUS

C203143

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

SMS_ID

300000046254

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

USAN

LM-86

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

CAS

2808361-18-2

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

FDA UNII

X4BE7S8PN9

Created by admin on Wed Apr 02 13:50:38 GMT 2025 , Edited by admin on Wed Apr 02 13:50:38 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_1-1_7;1_12-1_36;2_1;2_6;2_8-2_9;2_11-2_13;2_15-2_22	47 / 58
2'-Fluoro-Ribose	1_8-1_11;2_2-2_5;2_7;2_10;2_14	11 / 58

Linkage	Site Range	Site Count
Phosporothioate	1_2;2_2-2_4;2_21-2_22	6 / 56
Phosphate	1_3-1_36;2_5-2_20	50 / 56

Related Record Type Details


					X4BE7S8PN9

ACTIVE MOIETY

Comments:

LY3819469 is a novel Dicer-substrate small interfering RNA (siRNA) oligonucleotide designed to reduce the levels of LPA mRNA and apolipoprotein(a) expression.

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_28] [1_29] [1_30]	SITE_SPECIFIC			UNSPECIFIED SUBSTANCE	3a625511 (not in database)
NUCLEOTIDE SUBSTITUTION	[2_1]	3'-TERMINUS			UNSPECIFIED SUBSTANCE	2f426b3b (not in database)