Oligonucleotide compositions and methods of use thereof
AssigneeWave Life Sciences Ltd.
InventorsYuanjing Liu, Naoki Iwamoto, Chandra Vargeese, Zhong Zhong, Amy Jada Andreucci, Susovan Mohapatra
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Abstract
Among other things, the present disclosure provides C9orf72 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides methods for treating C9orf72-associated conditions, disorders or diseases, such as amyotrophic lateral sclerosis and frontotemporal dementia.
Claims8
An oligonucleotide, wherein the oligonucleotide is mA Sm5Ceon001RTeom5Ceon001RmA * SC * SC * SC * RA * SC * ST * Sm5C * SG * Rm5C SC * SmA * SmC * Sm5Ceo * SmGn001RmC (SEQ ID NO:
- 493) or a salt thereof, wherein:
- m represents a 2′-OMe modified nucleoside;
- eo represents a 2′-MOE modified nucleoside;
- m5C represents 5-methylcytosine;
- * S represents a phosphorothioate in the Sp configuration;
- * R represents a phosphorothioate in the Rp configuration;
- and
- n001R is
- wherein the phosphorus is of the Rp configuration.
An oligonucleotide composition comprising an oligonucleotide, wherein the oligonucleotide is mA * Sm5Ceon001RTeom5Ceon001RmA * SC * SC * SC * RA * SC * ST * Sm5C * SG * Rm5C * SC * SmA * SmC * Sm5Ceo SmGn001RmC (SEQ ID NO:
- 493) or a salt thereof, wherein:
- m represents a 2′-OMe modified nucleoside;
- eo represents a 2′-MOE modified nucleoside;
- m5C represents 5-methylcytosine;
- S represents a phosphorothioate in the Sp configuration;
- * R represents a phosphorothioate in the Rp configuration;
- and
- n001R is
- wherein the phosphorus is of the Rp configuration;
- and
- wherein the composition is chirally controlled in that it is enriched, relative to a substantially racemic preparation of the oligonucleotide, for the oligonucleotide.
Dependencies are inferred from the claim text.