Therapeutic uses of inhibitors of rtp801
AssigneeQuark Pharmaceuticals, Inc.
InventorsElena Feinstein, Jorg Kaufmann, Klaus Giese
Life of this patent
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Abstract
The present invention provides novel molecules, compositions, methods and uses for treating microvascular disorders, eye diseases and respiratory conditions based upon inhibition of the RTP801 gene and/or protein.
Claims23
1-28. (canceled)
A double-stranded compound comprising a sense strand and an antisense strand wherein the sense strand comprises ribonucleotides, the sequence of which is set forth in SEQ ID NO:16 and the antisense strand comprises ribonucleotides, the sequence of which is set forth in SEQ ID NO:66;
- wherein each ribonucleotide in the sense strand and in the antisense strand is independently unmodified or modified;
- and
- wherein each ribonucleotide is bound to each adjacent ribonucleotide by a covalent bond.
A compound having the structure:
- 5′ (N)x-Z 3′ (antisense strand)
- 3′ Z′-(N′)y 5′ (sense strand),
- wherein each N and N′ is a ribonucleotide which may be modified or unmodified in its sugar residue and each of (N)x and (N′)y is an oligomer in which each consecutive N or N′ is joined to the next N or N′ by a covalent bond;
- wherein at least 19 consecutive ribonucleotides in (N)x are complementary to ribonucleotides in (N′);
- wherein each of x and y is an integer from 19 to 40;
- wherein each of Z and Z′ may be present or absent, but if present is dTdT and is covalently attached at the 3′ terminus of the strand in which it is present;
- and wherein the sequence of (N)x comprises a sequence selected from the group consisting of:
- (SEQ ID NO:
- 74)
- UUCUAGAUGGAAGACCCAG;
- (SEQ ID NO:
- 75)
- UUGAACAUCAAGUGUAUUC;
- (SEQ ID NO:
- 77)
- AAAAAUAUUGCAUAGGUCU;
- (SEQ ID NO:
- 79)
- UACUUGAACAUCAAGUGUA;
- and
- (SEQ ID NO:
- 91)
- AACUCAAUGAGCUUCCUGG.
A method of treating a subject suffering from an eye disease which comprises administering to the subject an RTP801 inhibitor and a VEGF inhibitor, so as to thereby treat the subject.
A method of treating a subject suffering from a disorder which is a respiratory disorder, a microvascular disorder or a spinal cord injury or disease, which comprises administering to the subject a therapeutically effective amount of a siRNA compound having the structure:
- wherein alternating ribonucleotides in the antisense strand and the sense strand comprise a 2′OCH3 modification in the sugar residue of such ribonucleotides;
- wherein the sugar residue of the ribonucleotides at the 5′ terminus and at the 3′ terminus of the antisense strand comprise the 2′OCH3 modification and the sugar residue of the ribonucleotides at the 5′ terminus and at the 3′ terminus of the sense strand are unmodified;
- wherein each ribonucleotide at each of the 5′ terminus and the 3′ terminus of the antisense strand and of the sense strand is independently phosphorylated or non-phosphorylated;
- wherein each vertical line represents base pairing;
- and wherein the compound is administered in an amount to treat the subject.
Dependencies are inferred from the claim text.