LUMASIRAN

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 `Tue Apr 01 21:06:09 GMT 2025` Edited by `admin` on `Tue Apr 01 21:06:09 GMT 2025`
Nucleic Acid Type	RNAI
Sequence Origin	HUMAN
Sequence Type	COMPLETE
Record UNII	RZT8C352O1
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

RNA, (GM-SP-AM-SP-CM-UM-UM-UM-(2'-DEOXY-2'-FLUORO)C-AM-(2'-DEOXY-2'-FLUORO)U-(2'-DEOXY-2'-FLUORO)C-(2'-DEOXY-2'-FLUORO)C-UM-GM-GM-AM-AM-AM-UM-AM-UM-AM), 3'-(((2S,4R)-1-(29-((2-(ACETYLAMINO)-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-14,14-BIS((3-((3-((5-((2-

Preferred Name

English

LUMASIRAN

Official Name

English

LUMASIRAN [USAN]

Common Name

English

ALN-65585

Code

English

((2S,4R)-1-(1-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-16,16-BIS((3-((3-(5-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)PENTANAMIDO)PROPYL)AMINO)-3-OXOPROPOXY)METHYL)-5,11,18-TRIOXO-14-OXA-6,10,17-TRIAZANONACOSAN-29-OYL)-4-HYDROXYPYRRO

Systematic Name

English

Lumasiran [WHO-DD]

Common Name

English

ALN-G01

Code

English

lumasiran [INN]

Common Name

English

AD-65585

Code

English

ALN-GO1

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

505315

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

Code System Code Type Description

USAN

GH-49

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

CAS

1834610-13-7

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

INN

10684

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

FDA UNII

RZT8C352O1

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

DAILYMED

RZT8C352O1

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

RXCUI

2467140

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

NCI_THESAURUS

C170137

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

SMS_ID

100000178319

Created by admin on Tue Apr 01 21:06:09 GMT 2025 , Edited by admin on Tue Apr 01 21:06:09 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-Fluoro-Ribose	1_7;1_9-1_11;1_22;2_2;2_6;2_8-2_9;2_14;2_16	11 / 45
2'-O-Methyl-Ribose	1_1-1_6;1_8;1_12-1_21;2_1;2_3-2_5;2_7;2_10-2_13;2_15;2_17-2_23	34 / 45

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

Related Record Type Details


					IF1T9G3DOK

HYDROXYACID OXIDASE 1

TARGET->INHIBITOR OF EXPRESSION


					67P6XH37HD

LUMASIRAN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					RZT8C352O1

LUMASIRAN

ACTIVE MOIETY

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