CASIMERSEN

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 `Mon Mar 31 22:38:58 GMT 2025` Edited by `admin` on `Mon Mar 31 22:38:58 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	X8UHF7SX0R
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

EXON-45: NG-12-0064

Preferred Name

English

CASIMERSEN

Official Name

English

CASIMERSEN [USAN]

Common Name

English

casimersen [INN]

Common Name

English

Casimersen [WHO-DD]

Common Name

English

RNA, (P-DEOXY-P-(DIMETHYLAMINO)) (2',3'-DIDEOXY-2',3'-IMINO-2',3'-SECO) (2'A-5')(C-A-A-M5U-G-C-C-A-M5U-C-C-M5U-G-G-A-G-M5U-M5U-C-C-M5U-G), 5'-(P-(4-((2-(2-(2-HYDROXYETHOXY)ETHOXY)ETHOXY)CARBONYL)-1-PIPERAZINYL)-N,N-DIMETHYLPHOSPHONAMIDATE)

Common Name

English

AMONDYS 45

Brand Name

English

CASIMERSEN [ORANGE BOOK]

Common Name

English

SRP-4045

Common Name

English

ALL-P-AMBO-(2',3'-AZANEDIYL-P-(DIMETHYLAMINO)-P,2',3'-TRIDEOXY-2',3'-SECO)(2'-N->5')(C-A-A-T-G-C-C-A-T-C-C-T-GG-A-G-T-T-C-C-T-G) 5'-(P-(4-((2-(2-(2-HYDROXYETHOXY)ETHOXY)ETHOXY)CARBONYL)PIPERAZIN-1-YL)-N,NDIMETHYLPHOSPHONAMIDATE)

Systematic Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

690019

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

Code System Code Type Description

CAS

1422958-19-7

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

RXCUI

2480096

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

FDA UNII

X8UHF7SX0R

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

SMS_ID

100000174623

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

WIKIPEDIA

Casimersen

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

NCI_THESAURUS

C169830

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

USAN

FG-220

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

DRUG BANK

DB14984

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

DAILYMED

X8UHF7SX0R

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

INN

10354

Created by admin on Mon Mar 31 22:38:58 GMT 2025 , Edited by admin on Mon Mar 31 22:38:58 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
morpholino	1_1-1_22	22 / 22

Linkage	Site Range	Site Count
Phosphoryldimethylamine	1_2-1_22	21 / 21

Related Record Type Details


					60RX8439K2

TARGET -> INDUCER

Comments:

Exon 45 skipping

Related Record Type Details


					X8UHF7SX0R

ACTIVE MOIETY

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_4] [1_9] [1_12] [1_17] [1_18] [1_21]				MORPHOLINO THYMIDINE	YML1Y6H5HD
NUCLEOSIDE_SUBSTITUTION	[1_2] [1_3] [1_8] [1_15]				MORPHOLINO ADENOSINE	3ZYB3DY5HB
NUCLEOSIDE_SUBSTITUTION	[1_5] [1_13] [1_14] [1_16] [1_22]				MORPHOLINO GUANOSINE	XHA1WD2RB2
NUCLEOSIDE_SUBSTITUTION	[1_6] [1_7] [1_10] [1_11] [1_19] [1_20]				MORPHOLINO CYTIDINE	KFE1UKU3TI
NUCLEOTIDE SUBSTITUTION	[1_1]	5'-TERMINUS			CASIMERSEN 5'-CYTOSINE MODIFICATION	8CQO07490I

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