PELACARSEN

U.S. Department of Health & Human Services Divider Arrow

National Institutes of Health Divider Arrow

NCATS

Investigational

Investigational

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Tue Apr 01 21:10:16 GMT 2025` Edited by `admin` on `Tue Apr 01 21:10:16 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	LSO9H7UZ90
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

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

Preferred Name

English

PELACARSEN

Official Name

English

ISIS-681257 (GALNAC3--CONJUGATED ASO TARGETING HUMAN LIPOPROTEIN (A)) FREE ACID

Common Name

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)GUANYLYL-(3-O-

Common Name

English

AKCEA-APO(A)-LRX

Common Name

English

Pelacarsen [WHO-DD]

Common Name

English

PELACARSEN [USAN]

Common Name

English

IONIS-APO(A)-LRX

Common Name

English

pelacarsen [INN]

Common Name

English

ISIS-681257 FREE ACID

Code

English

Code System Code Type Description

FDA UNII

LSO9H7UZ90

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

CAS

1637637-70-7

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

NCI_THESAURUS

C174676

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

INN

11187

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

SMS_ID

100000184297

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

USAN

FG-172

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

PRIMARY

CAS

1637639-99-6

Created by admin on Tue Apr 01 21:10:16 GMT 2025 , Edited by admin on Tue Apr 01 21:10:16 GMT 2025

ALTERNATIVE

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_7-1_15	9 / 20
2'-O-Methoxyethyl ribose	1_1-1_6;1_16-1_20	11 / 20

Linkage	Site Range	Site Count
Phosporothioate	1_2;1_7-1_16;1_20	12 / 19
Phosphate	1_3-1_6;1_17-1_19	7 / 19

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ACTIVE MOIETY

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_3] [1_5] [1_6] [1_14] [1_20]	SITE_SPECIFIC			5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE_SUBSTITUTION	[1_1]	5'-TERMINUS			UNSPECIFIED SUBSTANCE	69c9da7b (not in database)