VUTRISIRAN

U.S. Department of Health & Human Services Divider Arrow

National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid
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

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Classification Tree

Code System

Code

Designated/Approved

FDA ORPHAN DRUG

637618

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Code System

Code

Type

Description

INN

10579

PRIMARY

RXCUI

2604578

PRIMARY

DAILYMED

GB4I2JI8UI

PRIMARY

USAN

KL-54

PRIMARY

NCI_THESAURUS

C152919

PRIMARY

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Subunit 0

Subunit 1

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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

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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

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Related Record

Type

Details


					2COU0B578R

TARGET->INHIBITOR OF EXPRESSION

Amount:


					28O0WP6Z1P

VUTRISIRAN SODIUM

SALT/SOLVATE -> PARENT

Amount:

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Related Record

Type

Details


					GB4I2JI8UI

ACTIVE MOIETY

Amount:

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Structural Modifications

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

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Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
Molecular Formula	CHEMICAL	EXACT C530H715F9N171O323P43S6