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EPLONTERSEN
0GRZ0F5XJ6
US Approved Rx (2023)
First approved in 2023
General
Record Details
Names
12
Classification
1
Identifiers
5
Subunits
1
Sugars
2
Linkages
2
Relationships
1
Active Moiety
1
Modifications
3
EPLONTERSEN
0GRZ0F5XJ6
US Approved Rx (2023)
First approved in 2023
General
Approval Year
2023
455
Record Details
Substance Class
Nucleic Acid
Nucleic Acid Type
OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type
COMPLETE
Record UNII
0GRZ0F5XJ6
Record Status
Validated (UNII)
Record Version
21
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Names
12
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Name
Type
Language
Name
Type
Language
EPLONTERSEN
Official Name
English
ION-TTR-LRX
Common Name
English
DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))RG-G-SP-T-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-G-SP-A-SP-A-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))M5RU-
Common Name
English
ISIS-682884 FREE ACID
Code
English
ALL-P-AMBO-5-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTID
Common Name
English
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Classification
1
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Classification Tree
Code System
Code
Classification Tree
Code System
Code
Designated
ORPHAN DRUG
Designated
Treatment of transthyretin-mediated amyloidosis
FDA ORPHAN DRUG
827521
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Identifiers
5
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Code System
Code
Type
Description
Code System
Code
Type
Description
CAS
1637600-16-8
PRIMARY
NCI_THESAURUS
C175062
PRIMARY
INN
11482
PRIMARY
FDA UNII
0GRZ0F5XJ6
PRIMARY
USAN
GH-148
PRIMARY
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Subunits
1
Subunit 0
>SUBUNIT_0
Sugars
2
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Sugar
Site Range
Site Count
Sugar
Site Range
Site Count
2'-O-Methoxyethyl ribose
1_1-1_5;1_16-1_20
10 / 20
Deoxyribose (DNA)
1_6-1_15
10 / 20
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Linkages
2
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Linkage
Site Range
Site Count
Linkage
Site Range
Site Count
Phosphate
1_3-1_6;1_17-1_18
6 / 19
Phosporothioate
1_2;1_7-1_16;1_19-1_20
13 / 19
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Relationships
1
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Related Record
Type
Details
Related Record
Type
Details
WSP2DHR2BD
EPLONTERSEN SODIUM
SALT/SOLVATE -> PARENT
Amount:
Showing 1 to 1 of 1 entries
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Active Moiety
1
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Related Record
Type
Details
Related Record
Type
Details
0GRZ0F5XJ6
EPLONTERSEN
ACTIVE MOIETY
Amount:
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Modifications
3
Structural Modifications
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entries
Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
NUCLEOSIDE_SUBSTITUTION
[1_2] [1_10] [1_18] [1_19] [1_20]
SITE_SPECIFIC
2'-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE
AOQ54IZZ6J
NUCLEOSIDE_SUBSTITUTION
[1_3] [1_4] [1_17]
SITE_SPECIFIC
2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE
9Q36I4D9VB
NUCLEOSIDE_SUBSTITUTION
[1_1]
N-TERMINUS
UNSPECIFIED SUBSTANCE
69c9da7b
(not in database)
Showing 1 to 3 of 3 entries
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