COFIRASERSEN

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 07:28:19 GMT 2025` Edited by `admin` on `Wed Apr 02 07:28:19 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	XV92IM2BG0
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ION-827359

Preferred Name

English

COFIRASERSEN

Official Name

English

IONIS-ENACRX

Code

English

DNA, D(P-THIO)((2'-O,4'-C-((S)-ETHYLIDENE))M5RC-(2'-O,4'-C-((S)-ETHYLIDENE))M5RC-(2'-O,4'-C-((S)-ETHYLIDENE))M5RC-G-A-T-A-G-M5C-T-G-G-T-(2'-O,4'-C-((S)-ETHYLIDENE))M5RU-(2'-O,4'-C-((S)-ETHYLIDENE))RG-(2'-O,4'-C-((S)-ETHYLIDENE))M5RU)

Common Name

English

DNA, D(P-THIO)((2',5'-ANHYDRO-6'-DEOXY-4'-C-(HYDROXYMETHYL)-.ALPHA.-L-MANNOFURANO)M5C-(3'->4')-(2',5'-ANHYDRO-6'-DEOXY-4'-C-(HYDROXYMETHYL)-.ALPHA.-L-MANNOFURANO)M5C-(3'->4')-(2',5'-ANHYDRO-6'-DEOXY-4'-C-(HYDROXYMETHYL)-.ALPHA.-L-MANNOFURANO)M5C-G-A-T-A-

Common Name

English

COFIRASERSEN [USAN]

Common Name

English

cofirasersen [INN]

Common Name

English

Code System Code Type Description

INN

11517

Created by admin on Wed Apr 02 07:28:19 GMT 2025 , Edited by admin on Wed Apr 02 07:28:19 GMT 2025

PRIMARY

CAS

2247669-96-9

Created by admin on Wed Apr 02 07:28:19 GMT 2025 , Edited by admin on Wed Apr 02 07:28:19 GMT 2025

PRIMARY

NCI_THESAURUS

C179078

Created by admin on Wed Apr 02 07:28:19 GMT 2025 , Edited by admin on Wed Apr 02 07:28:19 GMT 2025

PRIMARY

SMS_ID

100000181405

Created by admin on Wed Apr 02 07:28:19 GMT 2025 , Edited by admin on Wed Apr 02 07:28:19 GMT 2025

PRIMARY

FDA UNII

XV92IM2BG0

Created by admin on Wed Apr 02 07:28:19 GMT 2025 , Edited by admin on Wed Apr 02 07:28:19 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_4-1_13	10 / 16
2'-O,4'-ethylene bridged Ribose	1_1-1_3;1_14-1_16	6 / 16

Linkage	Site Range	Site Count
Phosporothioate	1_2-1_16	15 / 15

Related Record Type Details


					OWX7ZO8DD9

COFIRASERSEN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					XV92IM2BG0

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_14] [1_16]				THYMINE	QR26YLT7LT
NUCLEOSIDE BASE SUBSTITUTION	[1_1] [1_2] [1_3] [1_9]				5-METHYLCYTOSINE	6R795CQT4H