REMLARSEN

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National Institutes of Health Divider Arrow

NCATS

Investigational

Investigational

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Tue Apr 01 21:08:25 GMT 2025` Edited by `admin` on `Tue Apr 01 21:08:25 GMT 2025`
Nucleic Acid Type	RNAI
Sequence Type	COMPLETE
Record UNII	X2MD0LH19A
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

REMLARSEN

Official Name

English

remlarsen [INN]

Preferred Name

English

RNA, (AM-AM-CM-A-CM-UM-G-UM-UM-UM-A-CM-A-A-A-UM-G-G-UM-CM-CM-UM-A), 3'-(3-((6-((3.BETA.)-CHOLEST-5-EN-3-YLOXY)HEXYL)OXY)-2-HYDROXYPROPYL HYDROGEN PHOSPHATE), COMPLEX WITH RNA ((2'-DEOXY-2'-FLUORO)U-A-G-(2'-DEOXY-2'-FLUORO)C-A-(2'-DEOXY-2'-FLUORO)C-(2'-DE

Systematic Name

English

MRG-201

Code

English

Code System Code Type Description

FDA UNII

X2MD0LH19A

Created by admin on Tue Apr 01 21:08:25 GMT 2025 , Edited by admin on Tue Apr 01 21:08:25 GMT 2025

PRIMARY

CAS

1848266-71-6

Created by admin on Tue Apr 01 21:08:25 GMT 2025 , Edited by admin on Tue Apr 01 21:08:25 GMT 2025

PRIMARY

SMS_ID

300000011765

Created by admin on Tue Apr 01 21:08:25 GMT 2025 , Edited by admin on Tue Apr 01 21:08:25 GMT 2025

PRIMARY

NCI_THESAURUS

C170367

Created by admin on Tue Apr 01 21:08:25 GMT 2025 , Edited by admin on Tue Apr 01 21:08:25 GMT 2025

PRIMARY

INN

10560

Created by admin on Tue Apr 01 21:08:25 GMT 2025 , Edited by admin on Tue Apr 01 21:08:25 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	2_1-2_3;2_5-2_6;2_8-2_10;2_12;2_16;2_19-2_22	14 / 48
2'-Fluoro-Ribose	1_1;1_4;1_6-1_7;1_9-1_11;1_16-1_17;1_20;1_22-1_23	12 / 48
D-Ribose (RNA)	1_2-1_3;1_5;1_8;1_12-1_15;1_18-1_19;1_21;1_24-1_25;2_4;2_7;2_11;2_13-2_15;2_17-2_18;2_23	22 / 48

Linkage	Site Range	Site Count
Phosphate	1_2-1_23;2_2-2_23	44 / 46
Phosporothioate	1_24-1_25	2 / 46

Related Record Type Details


					X2MD0LH19A

REMLARSEN

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[2_23]	SITE_SPECIFIC			REMLARSEN 3'-MODIFICATION	IU0AC7632G