EPLONTERSEN

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 14:38:59 GMT 2023` Edited by `admin` on `Sat Dec 16 14:38:59 GMT 2023`
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type	COMPLETE
Record UNII	0GRZ0F5XJ6
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

EPLONTERSEN

Official Name

English

ION-TTR-LRX

Common Name

English

DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))RG-G-SP-T-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-G-SP-A-SP-A-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))M5RU-

Common Name

English

ISIS-682884 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)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTID

Common Name

English

AKCEA-TTR-LRX

Common Name

English

EPLONTERSEN [USAN]

Common Name

English

IONIS-TTR-LRX

Common Name

English

AKCEA TTR LRX

Common Name

English

Eplontersen [WHO-DD]

Common Name

English

eplontersen [INN]

Common Name

English

ION-682884 FREE ACID

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

827521

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

Code System Code Type Description

CAS

1637600-16-8

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

PRIMARY

NCI_THESAURUS

C175062

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

PRIMARY

INN

11482

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

PRIMARY

FDA UNII

0GRZ0F5XJ6

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

PRIMARY

USAN

GH-148

Created by admin on Sat Dec 16 14:38:59 GMT 2023 , Edited by admin on Sat Dec 16 14:38:59 GMT 2023

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methoxyethyl ribose	1_1-1_5;1_16-1_20	10 / 20
Deoxyribose (DNA)	1_6-1_15	10 / 20

Linkage	Site Range	Site Count
Phosphate	1_3-1_6;1_17-1_18	6 / 19
Phosporothioate	1_2;1_7-1_16;1_19-1_20	13 / 19

Related Record Type Details


					WSP2DHR2BD

EPLONTERSEN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					0GRZ0F5XJ6

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_2] [1_10] [1_18] [1_19] [1_20]	SITE_SPECIFIC			2'-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE	AOQ54IZZ6J
NUCLEOSIDE_SUBSTITUTION	[1_3] [1_4] [1_17]	SITE_SPECIFIC			2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE	9Q36I4D9VB
NUCLEOSIDE_SUBSTITUTION	[1_1]	N-TERMINUS			UNSPECIFIED SUBSTANCE	69c9da7b (not in database)