Tonlamarsen

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National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Sat Dec 16 19:01:05 GMT 2023` Edited by `admin` on `Sat Dec 16 19:01:05 GMT 2023`
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype	ANTI-SENSE
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	W6YJ85G6YG
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Tonlamarsen

Official Name

English

ION-904 FREE ACID

Code

English

ION904

Code

English

ION904 FREE ACID

Code

English

ALL-P-AMBO-5'-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXYL))PHOSPHO)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)GUANYLYL-

Common Name

English

tonlamarsen [INN]

Common Name

English

DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RG-(3'->4')-(2',5'-ANHYDRO-6'-DEOXY-4'-C-(HYDROXYMETHYL)-.ALPHA.-L-MANNOFURANO)M5C-T-SP-G-SP-A-SP-T-SP-T-SP-T-SP-G-SP-T-SP-M5C-SP-M5C-SP-(3'->4')-(2',5'-ANHYDRO-6'-DEOXY-4'-C-(HYDROXYMETHYL)-.A

Common Name

English

TONLAMARSEN [USAN]

Common Name

English

ION-904

Code

English

Code System Code Type Description

FDA UNII

W6YJ85G6YG

Created by admin on Sat Dec 16 19:01:05 GMT 2023 , Edited by admin on Sat Dec 16 19:01:05 GMT 2023

PRIMARY

NCI_THESAURUS

C188648

Created by admin on Sat Dec 16 19:01:05 GMT 2023 , Edited by admin on Sat Dec 16 19:01:05 GMT 2023

PRIMARY

INN

12351

Created by admin on Sat Dec 16 19:01:05 GMT 2023 , Edited by admin on Sat Dec 16 19:01:05 GMT 2023

PRIMARY

USAN

JK-207

Created by admin on Sat Dec 16 19:01:05 GMT 2023 , Edited by admin on Sat Dec 16 19:01:05 GMT 2023

PRIMARY

CAS

2589926-20-3

Created by admin on Sat Dec 16 19:01:05 GMT 2023 , Edited by admin on Sat Dec 16 19:01:05 GMT 2023

PRIMARY

Sugar	Site Range	Site Count
D-Ribose (RNA)	1_4-1_13	10 / 16
	1_3;1_14-1_15	3 / 16
2'-O-Methoxyethyl ribose	1_1-1_2;1_16	3 / 16

Linkage	Site Range	Site Count
Phosporothioate	1_2;1_5-1_14;1_16	12 / 15
Phosphate	1_3-1_4;1_15	3 / 15

Related Record Type Details


					W6YJ85G6YG

ACTIVE MOIETY

Comments:

The LICA technology incorporated in ION904 a triantennary N-acetyl galactosamine (GalNAc) moiety which targets the asialoglycoprotein receptors (ASGPR) expressed abundantly on the hepatocyte cell surface, resulting in antisense mediated inhibition of angiotensinogen (AGT) expression within the hepatocyte

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_3] [1_12] [1_13]				5-METHYLCYTOSINE	6R795CQT4H
NUCLEOTIDE SUBSTITUTION	[1_1]	5'-TERMINUS			ISIS-757456 CYTOSINE PHOSPHATE 5'-MODIFICATION	49G0W4AL3R