Ziclumeran

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NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 14:31:32 GMT 2025` Edited by `admin` on `Wed Apr 02 14:31:32 GMT 2025`
Nucleic Acid Type	MRNA
Sequence Type	COMPLETE
Record UNII	JJ5AS2HL3G
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

NTLA-2001 component mRNA000042

Preferred Name

English

Ziclumeran

Official Name

English

messenger RNA (mRNA), 5' capped, encoding codon-optimised clustered regularly interspaced short palindromic repeats (CRISPR) associated protein 9 (Cas9), flanked by untranslated regions (UTR) with significant sequence homology to the Xenopus beta globin

Common Name

English

ZICLUMERAN [USAN]

Common Name

English

RNA (recombinant 5?-(m7Gm-(5??5?)-ppp-G)-capped all uridine?N1-methylpseudouridine-substituted synthetic CRISPR-associated endodeoxyribonuclease CAS9 low codon A-optimized-specifying plus Xenopus laevis ?-globin 5?-and 3?-untranslated flanking region-con

Common Name

English

ziclumeran [INN]

Common Name

English

cas9 mRNA (mRNA000042)

Common Name

English

cas9 mRNA

Common Name

English

Code System Code Type Description

USAN

MN-129

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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FDA UNII

JJ5AS2HL3G

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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CAS

2305431-29-0

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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SMS_ID

300000023313

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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NCI_THESAURUS

C203205

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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INN

12328

Created by admin on Wed Apr 02 14:31:32 GMT 2025 , Edited by admin on Wed Apr 02 14:31:32 GMT 2025

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Sugar	Site Range	Site Count
D-Ribose (RNA)	1_1-1_4423	4423 / 4423

Linkage	Site Range	Site Count
Phosphate	1_2-1_4423	4422 / 4422

Related Record Type Details


					HB8LE6H4UK

Ziclumeran expressed CRISPR-associated protein 9

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					JJ5AS2HL3G

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION		RESIDUE_SPECIFIC	U		N1-METHYLPSEUDOURIDINE	09RAD4M6WF
NUCLEOTIDE SUBSTITUTION	[1_1]	5'-TERMINUS			7-methyl(3'-O-methyl)GpppGP cap	427EK6X4GY