Diranersen

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 12:37:12 GMT 2025` Edited by `admin` on `Wed Apr 02 12:37:12 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	96J3L33PWD
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

BIIB-080

Preferred Name

English

Diranersen

Official Name

English

all-P-ambo-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'?5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'?5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'?5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'?5')-2'-O-(2-methoxyethyl)-5-methyl-Pthiouridy

Common Name

English

BIIB080

Code

English

RNA, ([2?-O-(2-methoxyethyl)]m5C-sp-[2?-O-(2-methoxyethyl)]m5C-[2?-O-(2-methoxyethyl)]G-sp-[2?-O-(2-methoxyethyl)]m5U-sp-[2?-O-(2-methoxyethyl)]m5U-sp-dT-sp-dT-sp-m5dC-sp-dT-sp-dT-sp-dA-sp-m5dC-sp-m5dC-sp-[2?-O-(2-methoxyethyl)]A-sp-[2?-O-(2-methoxyethyl

Common Name

English

diranersen [INN]

Common Name

English

Code System Code Type Description

FDA UNII

96J3L33PWD

Created by admin on Wed Apr 02 12:37:12 GMT 2025 , Edited by admin on Wed Apr 02 12:37:12 GMT 2025

PRIMARY

CAS

2304711-06-4

Created by admin on Wed Apr 02 12:37:12 GMT 2025 , Edited by admin on Wed Apr 02 12:37:12 GMT 2025

PRIMARY

INN

13317

Created by admin on Wed Apr 02 12:37:12 GMT 2025 , Edited by admin on Wed Apr 02 12:37:12 GMT 2025

PRIMARY

SMS_ID

300000013142

Created by admin on Wed Apr 02 12:37:12 GMT 2025 , Edited by admin on Wed Apr 02 12:37:12 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_6-1_13	8 / 18
2'-O-Methoxyethyl ribose	1_1-1_5;1_14-1_18	10 / 18

Linkage	Site Range	Site Count
Phosporothioate	1_2;1_4-1_15;1_17-1_18	15 / 17
Phosphate	1_3;1_16	2 / 17

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MICROTUBULE-ASSOCIATED PROTEIN TAU (PAIRED HELICAL FILAMENTS (PHF))

TARGET->INHIBITOR OF EXPRESSION

Interaction Type:

PRECLINICAL

Amount:

Related Record Type Details


					96J3L33PWD

Diranersen

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_1] [1_2] [1_8] [1_12] [1_13] [1_15] [1_16] [1_17]	SITE_SPECIFIC	C		5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE BASE SUBSTITUTION	[1_4] [1_5] [1_18]	SITE_SPECIFIC	U		THYMINE	QR26YLT7LT