BALIFORSEN

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 `Tue Apr 01 20:59:10 GMT 2025` Edited by `admin` on `Tue Apr 01 20:59:10 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	HUMAN
Sequence Type	COMPLETE
Record UNII	DR9CF3915M
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

baliforsen [INN]

Preferred Name

English

BALIFORSEN

Official Name

English

IONIS-598769 FREE ACID

Code

English

ISIS 598769 FREE ACID

Code

English

ISIS-598769 FREE ACID

Code

English

BALIFORSEN [USAN]

Common Name

English

Baliforsen [WHO-DD]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

457914

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

Code System Code Type Description

INN

10433

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

SMS_ID

300000011807

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

FDA UNII

DR9CF3915M

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

USAN

CD-183

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

PUBCHEM

121494124

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

CAS

1698048-23-5

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

NCI_THESAURUS

C169801

Created by admin on Tue Apr 01 20:59:10 GMT 2025 , Edited by admin on Tue Apr 01 20:59:10 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methoxyethyl ribose	1_1-1_2;1_15-1_16	4 / 16
	1_3-1_4;1_13-1_14	4 / 16
Deoxyribose (DNA)	1_5-1_12	8 / 16

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

Related Record Type Details


					X9E1Y3I7P0

MYOTONIN-PROTEIN KINASE

TARGET->INHIBITOR OF EXPRESSION

Amount:


					JAC5IJI520

BALIFORSEN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					DR9CF3915M

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_2] [1_15]	SITE_SPECIFIC			2'-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE	AOQ54IZZ6J
NUCLEOSIDE_SUBSTITUTION	[1_1]	SITE_SPECIFIC			2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE	9Q36I4D9VB
NUCLEOSIDE_SUBSTITUTION	[1_3] [1_4] [1_11] [1_12]	SITE_SPECIFIC			2'-O,4'-C-((1S)-ETHANE-1,1-DIYL).BETA.-D-RIBOSE 5-METHYL CYTIDINE	7XRE49320R