TOFERSEN SODIUM

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National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Tue Apr 01 22:12:14 GMT 2025` Edited by `admin` on `Tue Apr 01 22:12:14 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	5YL205692C
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

QALSODY

Preferred Name

English

TOFERSEN SODIUM

Official Name

English

Tofersen sodium [WHO-DD]

Common Name

English

TOFERSEN SODIUM [USAN]

Common Name

English

ALL-P-AMBO-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)ADENYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)-P-THIOGUANYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)GUANYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3'->5')-P-THIOTHYMIDYLYL-

Systematic Name

English

DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R

Systematic Name

English

BIIB-067 SODIUM

Code

English

Code System Code Type Description

FDA UNII

5YL205692C

Created by admin on Tue Apr 01 22:12:14 GMT 2025 , Edited by admin on Tue Apr 01 22:12:14 GMT 2025

PRIMARY

USAN

EF-128

Created by admin on Tue Apr 01 22:12:14 GMT 2025 , Edited by admin on Tue Apr 01 22:12:14 GMT 2025

PRIMARY

CAS

1898254-60-8

Created by admin on Tue Apr 01 22:12:14 GMT 2025 , Edited by admin on Tue Apr 01 22:12:14 GMT 2025

PRIMARY

SMS_ID

100000183579

Created by admin on Tue Apr 01 22:12:14 GMT 2025 , Edited by admin on Tue Apr 01 22:12:14 GMT 2025

PRIMARY

NCI_THESAURUS

C166873

Created by admin on Tue Apr 01 22:12:14 GMT 2025 , Edited by admin on Tue Apr 01 22:12:14 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methoxyethyl ribose	1_1-1_5;1_16-1_19	9 / 20
Deoxyribose (DNA)	1_5-1_15	11 / 20

Linkage	Site Range	Site Count
Sodium Phosporothioate	1_2;1_4;1_6-1_16;1_18;1_20	15 / 19
	1_3;1_5;1_17;1_19	4 / 19

Related Record Type Details


					2NU6F9601K

TOFERSEN

PARENT -> SALT/SOLVATE

Amount:

Related Record Type Details


					2NU6F9601K

TOFERSEN

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_8] [1_13]	SITE_SPECIFIC			5-METHYLDEOXYCYTIDINE	B200GV71QM
NUCLEOSIDE_SUBSTITUTION	[1_20]	SITE_SPECIFIC			2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE	9Q36I4D9VB
NUCLEOSIDE_SUBSTITUTION	[1_1] [1_16] [1_19]	SITE_SPECIFIC	C		2'-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE	AOQ54IZZ6J