Rapirosiran

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 11:39:48 GMT 2025` Edited by `admin` on `Wed Apr 02 11:39:48 GMT 2025`
Nucleic Acid Type	RNAI
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	F3NU7CDG3E
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

Rapirosiran

Official Name

English

AD-288996

Preferred Name

English

RNA, (Am-sp-(2?-deoxy-2?-fluoro)G-sp-Am-Um-Am-Gm-(3??3?)-[1-de-?-D-ribofuranosyl-1-[(2S)-2,3-dihydroxypropyl]]m5U-(2??5?)-Cm-Cm-Am-Um-Gm-Cm-(2?-deoxy-2?-fluoro)A-Am-(2?-deoxy-2?-fluoro)A-Am-Gm-Cm-Am-Um-sp-Um-sp-Cm), complex with RNA (Am-sp-Um-sp-Gm-Cm-Um

Common Name

English

RAPIROSIRAN [USAN]

Common Name

English

rapirosiran [INN]

Common Name

English

[(2S,4R)-1-(1-[(2-acetamido-2-deoxy-?- D -galactopyranosyl)oxy]-16,16-bis({3-[(3-{5-[(2-acetamido-2-deoxy-?-D -galactopyranosyl)oxy]pentanamido}propyl)amino]-3-oxopropyl}methyl)-5,11,18-trioxo-14-oxa-6,10,17-triazanonacosan-29-oyl)-4-hydroxypyrrolidin-2-

Common Name

English

ALN-288996

Code

English

ALN-HSD

Code

English

Code System Code Type Description

CAS

2755196-97-3

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

INN

12805

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

FDA UNII

F3NU7CDG3E

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

NCI_THESAURUS

C199099

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

SMS_ID

300000023358

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

USAN

KL-191

Created by admin on Wed Apr 02 11:39:48 GMT 2025 , Edited by admin on Wed Apr 02 11:39:48 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_1;1_3-1_13;1_15;1_17-1_23;2_1-2_6;2_8;2_12-2_21	37 / 44
2'-Fluoro-Ribose	1_2;1_14;1_16;2_7;2_9-2_11	7 / 44

Linkage	Site Range	Site Count
Phosphate	1_4-1_21;2_4-2_21	36 / 42
Phosporothioate	1_2-1_3;1_22-1_23;2_2-2_3	6 / 42

Related Record Type Details


					NZ52LD879F

Rapirosiran Sodium

SALT/SOLVATE -> PARENT

Amount:


					T29BM4XEQ6

ASIALOGLYCOPROTEIN RECEPTOR 2

TARGET->LIGAND

Interaction Type:

IN-VIVO

Amount:

Related Record Type Details


					F3NU7CDG3E

ACTIVE MOIETY

Comments:

ALN-HSD utilizes the ASGPR by binding to it through the GalNAc ligand, allowing specific delivery of ALN-HSD to the liver after SC injection, resulting in liverspecific silencing of HSD17B13 expression

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE DELETIONS	[2_21]	3'-TERMINUS			UNSPECIFIED SUBSTANCE	c6b500e6 (not in database)
NUCLEOTIDE SUBSTITUTION	[1_7]	SITE_SPECIFIC			1-((2S)-1-(2,3-DIHYDROXY)PROPYL)THYMINE-PHOSPHATE	3BH2CF5QM5