CEMDISIRAN

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

National Institutes of Health Divider Arrow

NCATS

Investigational

Investigational

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Mon Mar 31 23:13:57 GMT 2025` Edited by `admin` on `Mon Mar 31 23:13:57 GMT 2025`
Nucleic Acid Type	siRNA
Sequence Type	COMPLETE
Record UNII	S66Z65E10T
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

cemdisiran [INN]

Preferred Name

English

CEMDISIRAN

Official Name

English

AD-62643 FREE ACID

Code

English

ALN-62643 FREE ACID

Code

English

CEMDISIRAN [USAN]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

760920

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

FDA ORPHAN DRUG

861721

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

Code System Code Type Description

CAS

1639264-46-2

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

INN

10269

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

NCI_THESAURUS

C169836

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

ALTERNATIVE

SMS_ID

300000025197

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

FDA UNII

S66Z65E10T

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

NCI_THESAURUS

C120319

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

USAN

HI-211

Created by admin on Mon Mar 31 23:13:57 GMT 2025 , Edited by admin on Mon Mar 31 23:13:57 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_24-1_25;2_22	3 / 47
2'-O-Methyl-Ribose	1_1;1_4;1_6-1_7;1_9;1_11-1_13;1_15;1_17;1_19-1_23;2_1-2_2;2_4;2_6;2_8;2_12;2_14-2_15;2_17;2_19-2_21	27 / 47
2'-Fluoro-Ribose	1_2-1_3;1_5;1_8;1_10;1_14;1_16;1_18;2_3;2_5;2_7;2_9-2_11;2_13;2_16;2_18	17 / 47

Linkage	Site Range	Site Count
Phosphate	1_4-1_25;2_4-2_22	41 / 45
Phosporothioate	1_2-1_3;2_2-2_3	4 / 45

Related Record Type Details


					7A6G4V3R84

TARGET -> INHIBITOR

Amount:

Related Record Type Details


					S66Z65E10T

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOTIDE SUBSTITUTION	[2_22]	SITE_SPECIFIC	A		CEMDISIRAN, TERMINAL SUGAR MODIFICATION-	CQQ59545BV

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
MOL_WEIGHT:SEQUNENCE(CALCULATED)	CHEMICAL