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 Sat Dec 16 14:18:12 GMT 2023
Edited
by admin
on Sat Dec 16 14:18:12 GMT 2023
Nucleic Acid Type OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type COMPLETE
Record UNII
T87K47QVF3
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Sefaxersen
USAN  
Official Name English
DNA,d(P-thio)([2’-O-(2-methoxyethyl)]rA-[2’-O-(2-methoxyethyl)]m5rU-[2’-O-( 2-methoxyethyl)]m5rC-[2’-O-(2-methoxyethyl)]m5rC-[2’-O-(2-methoxyethyl) ]m5rC-A-m5C-G-m5C-m5C-m5C-m5C-T-G-T-[2’-O-(2-methoxyethyl)]m5rC-[2’-O-( 2-methoxyethyl)]m5rC
Systematic Name English
SEFAXERSEN [USAN]
Common Name English
IONIS-FB-LRX FREE ACID
Code English
all-P-ambo-5′-O-{[(6-{5-[(tris{3-[6-(2-acetamido-2-deoxy-β-D-galactopyranosyloxy)hexylamino]-3-oxopropoxymethyl})methyl]amino-5-oxopentanamido}hexyl)]phospho}-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3′-O→5′-O)-2′-O-(2-methoxyethyl)-5-methyl-P-
Systematic Name English
Code System Code Type Description
CAS
2272975-74-1
Created by admin on Sat Dec 16 14:18:12 GMT 2023 , Edited by admin on Sat Dec 16 14:18:12 GMT 2023
PRIMARY
USAN
KL-233
Created by admin on Sat Dec 16 14:18:13 GMT 2023 , Edited by admin on Sat Dec 16 14:18:13 GMT 2023
PRIMARY
FDA UNII
T87K47QVF3
Created by admin on Sat Dec 16 14:18:12 GMT 2023 , Edited by admin on Sat Dec 16 14:18:12 GMT 2023
PRIMARY
INN
12845
Created by admin on Sat Dec 16 14:18:13 GMT 2023 , Edited by admin on Sat Dec 16 14:18:13 GMT 2023
PRIMARY
Sugar Site Range Site Count
Deoxyribose (DNA) 1_6-1_15 10 / 20
2'-O-Methoxyethyl ribose 1_1-1_5;1_16-1_20 10 / 20
Linkage Site Range Site Count
Phosporothioate 1_1-1_19 19 / 19
Related Record Type Details
TARGET->INHIBITOR OF EXPRESSION
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION [1_3] [1_4] [1_5] [1_9] [1_10] [1_11] [1_12] [1_16] [1_17] [1_20] SITE_SPECIFIC 5-METHYLCYTOSINE 6R795CQT4H
NUCLEOSIDE_SUBSTITUTION [1_1] 5'-TERMINUS UNSPECIFIED SUBSTANCE
6174f862
(not in database)
NUCLEOSIDE BASE SUBSTITUTION [1_2] SITE_SPECIFIC THYMINE QR26YLT7LT
Name Property Type Amount Referenced Substance Defining Parameters References
MOL_WEIGHT:NUMBER(CALCULATED) CHEMICAL