Nivudirsen

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 16:22:14 GMT 2025` Edited by `admin` on `Wed Apr 02 16:22:14 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	34LVJ6J2YV
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Nivudirsen

Official Name

English

RNA, (P-thio)((2?-O,4?-C-methylene)G-(2?-O,4?-C-methylene)G-Um-Am-Am-(2?-O,4?-C-methylene)G-Um-Um-m5Cm-Um-Gm-Um-m5Cm-m5Cm-Am-Am-Gm-(2?-O,4?-C-methylene)m5C), 5?-[2-[2-(2-hydroxyethoxy)ethoxy]ethyl hydrogen phosphate]

Preferred Name

English

nivudirsen [INN]

Common Name

English

all-P-ambo-5'-O-(1,10-dihydroxy-1-oxo-2,5,8-trioxa-1?5-phosphadecan-1-yl)-2'-O,4'-C-methylene-P-thioguanylyl-(3'?5')-2'-O,4'-C-methylene-P-thioguanylyl-(3'?5')-2'-O-methyl-Pthiouridylyl-( 3'?5')-2'-O-methyl-P-thioadenylyl-(3'?5')-2'-Omethyl-P-thioadenyly

Common Name

English

Code System Code Type Description

FDA UNII

34LVJ6J2YV

Created by admin on Wed Apr 02 16:22:14 GMT 2025 , Edited by admin on Wed Apr 02 16:22:14 GMT 2025

PRIMARY

CAS

3053113-45-1

Created by admin on Wed Apr 02 16:22:14 GMT 2025 , Edited by admin on Wed Apr 02 16:22:14 GMT 2025

PRIMARY

INN

13425

Created by admin on Wed Apr 02 16:22:14 GMT 2025 , Edited by admin on Wed Apr 02 16:22:14 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
	1_1-1_2;1_6;1_18	4 / 18
2'-O-Methyl-Ribose	1_3-1_5;1_7-1_17	14 / 18

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

Related Record Type Details


					60RX8439K2

DYSTROPHIN

TARGET->CORRECTOR

Comments:

BMN 351-induced exon 51 skips allow for restoration of the mutated open-reading frame and result in significant levels of near full-length dystrophin

Amount:

Related Record Type Details


					34LVJ6J2YV

Nivudirsen

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[1_1]	5'-TERMINUS			UNSPECIFIED SUBSTANCE	610daa94 (not in database)
NUCLEOSIDE BASE SUBSTITUTION	[1_9] [1_13] [1_14] [1_18]				5-METHYLCYTOSINE	6R795CQT4H