Cimdelirsen

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National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Sat Dec 16 15:07:46 GMT 2023` Edited by `admin` on `Sat Dec 16 15:07:46 GMT 2023`
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type	COMPLETE
Record UNII	F593G1C1YO
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Cimdelirsen

Official Name

English

DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC-sp-T-sp-T-sp-T-sp-G-sp-G-sp-G-sp-T-sp-G-sp-A-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)

Common Name

English

ISIS-766720

Code

English

CIMDELIRSEN [USAN]

Common Name

English

IONIS-GHR-LRX FREE ACID

Code

English

cimdelirsen [INN]

Common Name

English

all-P-ambo-5′-O-{[(6-{5-[(tris{3-[6-(2-acetamido-2-deoxy-β-Dgalactopyranosyloxy)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)-5-

Common Name

English

IONIS-GHR-LRX FREE ACID(ANTISENSE INHIBITOR OF THE GROWTH HORMONE RECEPTOR)

Common Name

English

Code System Code Type Description

INN

11675

Created by admin on Sat Dec 16 15:07:46 GMT 2023 , Edited by admin on Sat Dec 16 15:07:46 GMT 2023

PRIMARY

USAN

GH-83

Created by admin on Sat Dec 16 15:07:46 GMT 2023 , Edited by admin on Sat Dec 16 15:07:46 GMT 2023

PRIMARY

FDA UNII

F593G1C1YO

Created by admin on Sat Dec 16 15:07:46 GMT 2023 , Edited by admin on Sat Dec 16 15:07:46 GMT 2023

PRIMARY

NCI_THESAURUS

C162807

Created by admin on Sat Dec 16 15:07:46 GMT 2023 , Edited by admin on Sat Dec 16 15:07:46 GMT 2023

PRIMARY

CAS

2131025-82-4

Created by admin on Sat Dec 16 15:07:46 GMT 2023 , Edited by admin on Sat Dec 16 15:07:46 GMT 2023

PRIMARY

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

Linkage	Site Range	Site Count
Phosporothioate	1_1-1_2;1_5-1_15;1_18-1_19	15 / 19
Phosphate	1_3-1_4;1_16-1_17	4 / 19

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GROWTH HORMONE RECEPTOR

TARGET->INHIBITOR OF EXPRESSION

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Cimdelirsen Sodium

SALT/SOLVATE -> PARENT

Amount:

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CIMDELIRSEN SODIUM ACTIVE METABOLITE

METABOLITE ACTIVE -> PRODRUG

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ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_16]	SITE_SPECIFIC	U		THYMINE	QR26YLT7LT
NUCLEOTIDE SUBSTITUTION	[1_1]	C-TERMINUS			ISIS-757456 CYTOSINE PHOSPHATE 5'-MODIFICATION	49G0W4AL3R
NUCLEOSIDE BASE SUBSTITUTION	[1_2] [1_4] [1_5] [1_19]	SITE_SPECIFIC			5-METHYLCYTOSINE	6R795CQT4H