Double-stranded rna compounds to casp2 and uses thereof
AssigneeQuark Pharmaceuticals, Inc.
InventorsShai Erlich, James D. Thompson, Rabia Ozden
Life of this patent
Estimating the term…
Abstract
The present disclosure relates to methods of treating a patient suffering from or at risk of developing an ocular disease, disorder or injury, and includes treatment regimens using a double-stranded RNA compound that down-regulates CASP2 expression, or a pharmaceutically acceptable salt thereof.
Claims50
A double-stranded RNA compound having the structure:
- (sense strand;
- SEQ ID NO:
- 1)
- 5′ iB-GCCAGAAUGUGGAACUCCU 3′
- (antisense strand;
- SEQ ID NO:
- 2)
- 3′ CGGUCUUACACCUUGAGGA 5′
- wherein each A, C, U and G is a nucleotide and each consecutive nucleotide is joined to the next nucleotide by a phosphodiester bond;
- wherein each nucleotide is independently an unmodified ribonucleotide, a 2′-O-Methyl sugar modified ribonucleotide or a L-DNA nucleotide;
- wherein the sense strand comprises, counting from the 5′ terminus, an unmodified ribonucleotide at each of positions 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 19 and a L-deoxycytidine at position 18, and an inverted abasic deoxyribose cap (iB) covalently bound to the 5′ terminus;
- wherein the antisense strand comprises, counting from the 5′ terminus, a 2′-O-Methyl sugar modified ribonucleotide at each of positions 2, 4, 6, 8, 11, 13, 15, 17 and 19 and an unmodified ribonucleotide at each of positions 1, 3, 5, 7, 9, 10, 12, 14, 16 and 18;
- or a pharmaceutically acceptable salt thereof;
- for use in the treatment of a patient suffering from or at risk of developing an ocular disease, an ocular disorder or an ocular injury;
- wherein the compound is to be administered to the patient's eye at a dose of about 0.05 mg to about 10 mg per eye.
A method of treating a patient suffering from or at risk for developing an ocular disease, an ocular disorder or an ocular injury, comprising administering to the patient's eye a double-stranded RNA compound that down-regulates CASP2 expression, or a pharmaceutically acceptable salt thereof, the double-stranded RNA compound having the structure:
- (sense strand;
- SEQ ID NO:
- 1)
- 5′ iB-GCCAGAAUGUGGAACUCCU 3′
- (antisense strand;
- SEQ ID NO:
- 2)
- 3′ CGGUCUUACACCUUGAGGA 5′
- wherein each A, C, U and G is a nucleotide and each consecutive nucleotide is joined to the next nucleotide by a phosphodiester bond;
- wherein each nucleotide is independently an unmodified ribonucleotide, a 2′-O-Methyl sugar modified ribonucleotide or a L-DNA nucleotide;
- wherein the sense strand comprises, counting from the 5′ terminus, an unmodified ribonucleotide at each of positions 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 19 and a L-deoxycytidine at position 18, and an inverted abasic deoxyribose cap (iB) covalently bound to the 5′ terminus;
- wherein the antisense strand comprises, counting from the 5′ terminus, a 2′-O-Methyl sugar modified ribonucleotide at each of positions 2, 4, 6, 8, 11, 13, 15, 17 and 19 and an unmodified ribonucleotide at each of positions 1, 3, 5, 7, 9, 10, 12, 14, 16 and 18;
- wherein the compound is administered to the patient's eye at a dose of about 0.05 mg to about 10.0 mg per eye.
Dependencies are inferred from the claim text.