Marpinersen

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 05:41:59 GMT 2025` Edited by `admin` on `Wed Apr 02 05:41:59 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	JZ0VQQ1YLD
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Marpinersen

Official Name

English

all-P-ambo-2'-O-(2-methoxyethyl)-5-methyl-Pthiouridylyl-( 3'?5')-2'-O-(2-methoxyethyl)guanylyl-(3'?5')-2'-O-(2-methoxyethyl)guanylyl-(3'?5')-2'-O-(2-methoxyethyl)adenylyl-(3'?5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'?5')-2'-O-(2-methoxyethyl)-5-meth

Preferred Name

English

marpinersen [INN]

Common Name

English

Code System Code Type Description

CAS

2757908-12-4

Created by admin on Wed Apr 02 05:41:59 GMT 2025 , Edited by admin on Wed Apr 02 05:41:59 GMT 2025

PRIMARY

INN

13366

Created by admin on Wed Apr 02 05:41:59 GMT 2025 , Edited by admin on Wed Apr 02 05:41:59 GMT 2025

PRIMARY

FDA UNII

JZ0VQQ1YLD

Created by admin on Wed Apr 02 05:41:59 GMT 2025 , Edited by admin on Wed Apr 02 05:41:59 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_7-1_16	10 / 20
2'-O-Methoxyethyl ribose	1_1-1_6;1_17-1_20	10 / 20

Linkage	Site Range	Site Count
Phosporothioate	1_2;1_8-1_17;1_19-1_20	13 / 19
Phosphate	1_3-1_7;1_18	6 / 19

Related Record Type Details


					2H28ZCL4VO

ATAXIN-2

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					JZ0VQQ1YLD

Marpinersen

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_7] [1_12] [1_17] [1_19]	SITE_SPECIFIC	C		5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE BASE SUBSTITUTION	[1_1] [1_5] [1_6] [1_18]	SITE_SPECIFIC	U		THYMINE	QR26YLT7LT