Sefaxersen

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Sat Dec 16 14:18:12 GMT 2023` Edited by `admin` on `Sat Dec 16 14:18:12 GMT 2023`
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type	COMPLETE
Record UNII	T87K47QVF3
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Sefaxersen

Official Name

English

DNA,d(P-thio)([2’-O-(2-methoxyethyl)]rA-[2’-O-(2-methoxyethyl)]m5rU-[2’-O-( 2-methoxyethyl)]m5rC-[2’-O-(2-methoxyethyl)]m5rC-[2’-O-(2-methoxyethyl) ]m5rC-A-m5C-G-m5C-m5C-m5C-m5C-T-G-T-[2’-O-(2-methoxyethyl)]m5rC-[2’-O-( 2-methoxyethyl)]m5rC

Systematic Name

English

SEFAXERSEN [USAN]

Common Name

English

IONIS-FB-LRX FREE ACID

Code

English

all-P-ambo-5′-O-{[(6-{5-[(tris{3-[6-(2-acetamido-2-deoxy-β-D-galactopyranosyloxy)hexylamino]-3-oxopropoxymethyl})methyl]amino-5-oxopentanamido}hexyl)]phospho}-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3′-O→5′-O)-2′-O-(2-methoxyethyl)-5-methyl-P-

Systematic Name

English

Code System Code Type Description

CAS

2272975-74-1

Created by admin on Sat Dec 16 14:18:12 GMT 2023 , Edited by admin on Sat Dec 16 14:18:12 GMT 2023

PRIMARY

USAN

KL-233

Created by admin on Sat Dec 16 14:18:13 GMT 2023 , Edited by admin on Sat Dec 16 14:18:13 GMT 2023

PRIMARY

FDA UNII

T87K47QVF3

Created by admin on Sat Dec 16 14:18:12 GMT 2023 , Edited by admin on Sat Dec 16 14:18:12 GMT 2023

PRIMARY

INN

12845

Created by admin on Sat Dec 16 14:18:13 GMT 2023 , Edited by admin on Sat Dec 16 14:18:13 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_19	19 / 19

Related Record Type Details


					3635PCK4HN

COMPLEMENT FACTOR B

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					T87K47QVF3

Sefaxersen

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_3] [1_4] [1_5] [1_9] [1_10] [1_11] [1_12] [1_16] [1_17] [1_20]	SITE_SPECIFIC	5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE_SUBSTITUTION	[1_1]	5'-TERMINUS	UNSPECIFIED SUBSTANCE	6174f862 (not in database)
NUCLEOSIDE BASE SUBSTITUTION	[1_2]	SITE_SPECIFIC	THYMINE	QR26YLT7LT

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
MOL_WEIGHT:NUMBER(CALCULATED)	CHEMICAL