Mivelsiran

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 `Wed Apr 02 13:55:51 GMT 2025` Edited by `admin` on `Wed Apr 02 13:55:51 GMT 2025`
Nucleic Acid Type	siRNA
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	39CFL75KXH
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

Mivelsiran

Official Name

English

RNA, ((5?,6?-dideoxy-6?-phosphono-?-D-ribo-hex-5-enofurano)Um-sp-(2?-deoxy-2?-fluoro)U-sp-Am-Gm-Gm-Um-(3??3?)-[1-de-?-D-ribofuranosyl-1-[(2S)-2,3-dihydroxypropyl]]m5U-(2??5?)-Gm-Gm-Am-Um-dT-Um-(2?-deoxy-2?-fluoro)U-Cm-dG-Um-Am-Gm-Cm-Cm-sp-Gm-sp-Um), comp

Preferred Name

English

MIVELSIRAN [USAN]

Common Name

English

ALN-APP

Code

English

all-P-ambo-2'-O-methyl-P-thioguanylyl-(3'?5')-2'-O-methyl-Pthioguanylyl-(3'?5')-2'-O-methylcytidylyl-(3'?5')-2'-O-methyluridylyl-(3'?5')-2'-O-methyladenylyl-(3'?5')-2'-O-hexadecylcytidylyl-(3'?5')-2'-O-methylguanylyl-(3'?5')-2'-O-methyladenylyl-(3'?5')-2

Systematic Name

English

AD-961583

Code

English

mivelsiran [INN]

Common Name

English

ALN-961583

Code

English

Code System Code Type Description

NCI_THESAURUS

C203135

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

FDA UNII

39CFL75KXH

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

INN

12750

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

CAS

2572991-83-2

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

SMS_ID

300000055680

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

USAN

MN-204

Created by admin on Wed Apr 02 13:55:51 GMT 2025 , Edited by admin on Wed Apr 02 13:55:51 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_12;1_16;2_9;2_11	4 / 44
2'-O-Methyl-Ribose	1_1;1_3-1_11;1_13;1_15;1_17-1_23;2_1-2_8;2_10;2_12-2_21	38 / 44
2'-Fluoro-Ribose	1_2;1_14	2 / 44

Linkage	Site Range	Site Count
Phosporothioate	1_2-1_3;1_22-1_23;2_2-2_3;2_20-2_21	8 / 42
Phosphate	1_4-1_21;2_4-2_19	34 / 42

Related Record Type Details


					P4Z43RA9BK

Amyloid-beta precursor protein

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					39CFL75KXH

ACTIVE MOIETY

Comments:

ALN-APP is a small interfering RNA (siRNA) that targets amyloid precursor protein (APP) messenger RNA (mRNA) resulting in APP synthesis inhibition

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[2_6]	SITE_SPECIFIC			UNSPECIFIED SUBSTANCE	4dcb2604 (not in database)
NUCLEOTIDE SUBSTITUTION	[1_7]	SITE_SPECIFIC			1-((2S)-1-(2,3-DIHYDROXY)PROPYL)THYMINE-PHOSPHATE	3BH2CF5QM5
NUCLEOTIDE SUBSTITUTION	[1_1]	SITE_SPECIFIC			Vinylphosphonate 2'-O-methyluridine	5V3K9SGM4U