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 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
  • Download
Name Type Language
Tonlamarsen
INN   USAN  
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
ACTIVE MOIETY
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

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