Antisense Oligonucleotides Targeting ATXN3
AssigneeHoffmann-La Roche Inc.
InventorsHeidi Rye Hudlebusch, Lykke Pedersen, Erik Funder, Lukasz Kielpinski, Christoffer Sondergaard, Alexander Herbert Stephan
Life of this patent
Estimating the term…
Abstract
The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.
Claims33
An antisense oligonucleotide selected from the group consisting of Compound ID Nos. 1122_107, 1122_156, 1122_91, 1122_154, 1122_155, 1122_157, 1122_158, 1122_167, 1122_172, 1122_175, 1122_294, 1122_296, 1816_13, 1816_15, 1816_28, 1816_41, 1816_42, 1816_43, 1816_60, 1816_61, 1816_64, 1816_65, and 1816_68, or a pharmaceutically acceptable salt thereof.
An in vivo or in vitro method for modulating ATXN3 expression in a target cell which is expressing ATXN3, said method comprising administering an oligonucleotide selected from the group consisting of Compound ID Nos. 1122_91, 1122_107, 1122_154, 1122_155, 1122_156, 1122_157, 1122_158, 1122_167, 1122_172, 1122_175, 1122_294, 1122_296, 1816_13, 1816_15, 1816_28, 1816_41, 1816_42, 1816_43, 1816_60, 1816_61, 1816_64, 1816_65, and 1816_68, a conjugate, a salt, or a pharmaceutical composition thereof in an effective amount to said cell.
A method for treating or preventing a disease comprising administering a therapeutically or prophylactically effective amount of an oligonucleotide selected from the group consisting of Compound ID Nos. 1122_91, 1122_107, 1122_154, 1122_155, 1122_156, 1122_157, 1122_158, 1122_167, 1122_172, 1122_175, 1122_294, 1122_296, 1816_13, 1816_15, 1816_28, 1816_41, 1816_42, 1816_43, 1816_60,1816_61,1816_64,1816_65, and 1816_68, a conjugate, a salt, or a pharmaceutical composition thereof to a subject suffering from or susceptible to the disease.
Dependencies are inferred from the claim text.