Tau antisense oligomers and uses thereof
AssigneeF. Hoffmann-La Roche AG
InventorsRichard E. Olson, Angela M. Cacace, Peter Hagedorn, Anja Mølhart Høg, Marianne Lerbech Jensen, Niels Fisker Nielsen, Dong Li, Jeffrey M. Brown, Stephen E. Mercer
Life of this patent
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Abstract
The present invention relates to oligomer compounds (oligomers), which target Tau mRNA in a cell, leading to reduced expression of Tau protein. Reduction of Tau protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder.
Claims13
An oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside.
A method of inhibiting or reducing Tau protein expression in a cell, the method comprising:
- administering a pharmaceutical composition comprising at least one of:
- an oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside;
- or
- a conjugate comprising an oligomer having a nucleic acid sequence comprising ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside, wherein the oligomer is covalently attached to at least one non-nucleotide or non-polynucleotide moiety;
- to the cell expressing Tau protein, wherein the Tau protein expression in the cell is inhibited or reduced after the administration.
A method for treating a tauopathy, the method comprising:
- administering an effective amount of a pharmaceutical composition comprising at least one of:
- an oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside;
- or
- a conjugate comprising an oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside, wherein the oligomer is covalently attached to at least one non-nucleotide or non-polynucleotide moiety.
A method for treating a disorder associated with over expression or expression of a mutated version of Tau protein, the method comprising:
- administering an effective amount of a pharmaceutical composition comprising at least one of:
- an oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside;
- or
- a conjugate comprising an oligomer consisting of the nucleic acid sequence ATTtCcaaattcacTtTtAC (SEQ ID NO:487) or ATtTCcaaattcactTTtAC (SEQ ID NO:472), wherein each upper case letter is a beta-D-oxy-LNA nucleoside, and wherein each lower case letter is a DNA nucleoside, wherein the oligomer is covalently attached to at least one non-nucleotide
- or non-polynucleotide moiety.
Dependencies are inferred from the claim text.