VUTRISIRAN

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 `Sat Dec 16 13:54:40 UTC 2023` Edited by `admin` on `Sat Dec 16 13:54:40 UTC 2023`
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type	COMPLETE
Record UNII	GB4I2JI8UI
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

VUTRISIRAN

Official Name

English

{(2S,4R)-1-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)-17,17-bis-({3-[(3-{5- [(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-pentanamido}propyl)amino]- 3-oxopropoxy}methyl)-6,12,19-trioxo-1,15-dioxa-7,11,18-triazatriacontan-30- oyl]-4-hydroxypyrrolidin

Common Name

English

VOTRISIRAN

Official Name

English

ALN-TTRSC02 FREE ACID

Code

English

Vutrisiran [WHO-DD]

Common Name

English

ALN-65492

Code

English

vutrisiran [INN]

Common Name

English

VUTRISIRAN [USAN]

Common Name

English

RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-spGm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-de

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

637618

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

Code System Code Type Description

INN

10579

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

RXCUI

2604578

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

DAILYMED

GB4I2JI8UI

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

USAN

KL-54

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

NCI_THESAURUS

C152919

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

FDA UNII

GB4I2JI8UI

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

CAS

1867157-35-4

Created by admin on Sat Dec 16 13:54:40 UTC 2023 , Edited by admin on Sat Dec 16 13:54:40 UTC 2023

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_1-1_6;1_8;1_12-1_22;2_1-2_5;2_7-2_8;2_10-2_13;2_15;2_17-2_23	37 / 45
2'-Fluoro-Ribose	1_7;1_9-1_11;2_6;2_9;2_14;2_16	8 / 45

Linkage	Site Range	Site Count
Phosphate	1_4-1_22;2_4-2_21	37 / 43
Phosporothioate	1_2-1_3;2_2-2_3;2_22-2_23	6 / 43

Related Record Type Details


					2COU0B578R

TARGET->INHIBITOR OF EXPRESSION

Amount:


					28O0WP6Z1P

VUTRISIRAN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					GB4I2JI8UI

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[1_22]	SITE_SPECIFIC			CEMDISIRAN, TERMINAL SUGAR MODIFICATION-	CQQ59545BV

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
Molecular Formula	CHEMICAL