Renadirsen

U.S. Department of Health & Human Services Divider Arrow

National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 19:17:22 GMT 2025` Edited by `admin` on `Wed Apr 02 19:17:22 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	4RW6WRD7SW
Record Status	`FAILED`
Record Version

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Name

Type

Language

renadirsen [INN]

Preferred Name

English

Renadirsen

Official Name

English

RNA, (P-thio)([2?,6?-anhydro-5?-deoxy-4?-C-(hydroxymethyl)-?-L-lyxo-hexofurano]m5C-Gm-(3??4?)-[2?,6?-anhydro-5?-deoxy-4?-C-(hydroxymethyl)-?-L-lyxo-hexofurano]m5C-(3??4?)-[2?,6?-anhydro-5?-deoxy-4?-C-(hydroxymethyl)-?-L-lyxo-hexofurano]m5U-Gm-Cm-(3??4?)-

Systematic Name

English

RENAPERSEN

Common Name

English

Classification Tree Code System Code

NCI_THESAURUS

C78281

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

Code System Code Type Description

FDA UNII

4RW6WRD7SW

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

PRIMARY

SMS_ID

300000005921

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

PRIMARY

NCI_THESAURUS

C152190

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

PRIMARY

PUBCHEM

131801099

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

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CAS

1782108-31-9

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

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INN

10371

Created by admin on Wed Apr 02 19:17:22 GMT 2025 , Edited by admin on Wed Apr 02 19:17:22 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O,4'-ethylene bridged Ribose	1_1;1_3-1_4;1_7-1_8;1_11;1_13-1_14;1_17-1_18	10 / 18
2'-O-Methyl-Ribose	1_2;1_5-1_6;1_9-1_10;1_12;1_15-1_16	8 / 18

Linkage	Site Range	Site Count
Phosporothioate	1_2-1_18	17 / 17

Related Record Type Details


					H5MKY4U4D5

Renadirsen sodium

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					4RW6WRD7SW

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[1_18]	3'-TERMINUS			2'-O,4'-C-(ethane-1,2-diyl)-5-methyl-3'-(O-(2-hydroxyethyl)-P-thio)-3'-cytidylate	KY4EJ5CG5L
NUCLEOSIDE BASE SUBSTITUTION	[1_1] [1_3] [1_7] [1_8] [1_13] [1_14] [1_17]	SITE_SPECIFIC			5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE BASE SUBSTITUTION	[1_4] [1_11]	SITE_SPECIFIC			THYMINE	QR26YLT7LT