Antagonist of pcsk9
AssigneeArgonaute Rna Limited
InventorsDaniel Mitchell, Michael Khan
Life of this patent
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Abstract
This disclosure relates to a nucleic acid comprising a double stranded RNA molecule comprising sense and antisense strands and further comprising a single stranded DNA molecule covalently linked to the 3′ end of either the sense or antisense RNA part of the molecule wherein the double stranded inhibitory RNA targets proprotein convertase subtilisin kexin type 9 (PCSK9); pharmaceutical compositions comprising said nucleic acid molecule and methods for the treatment of diseases associated with increased levels of PCSK9, for example hypercholesterolemia and cardiovascular disease.
Claims29
A nucleic acid molecule comprising:
- a first part that comprises a double stranded inhibitory ribonucleic acid (RNA) molecule comprising a sense strand and an antisense strand of at least part of the human PCSK9 nucleotide sequence or polymorphic sequence variant thereof;
- and
- a second part that comprises a single stranded deoxyribonucleic acid (DNA) molecule, wherein the 5′ end of said single stranded DNA molecule is covalently linked to the 3′ end of the sense strand of the double stranded inhibitory RNA molecule or wherein the 5′ end of the single stranded DNA molecule is covalently linked to the 3′ of the antisense strand of the double stranded inhibitory RNA molecule, wherein said single stranded DNA molecule comprises a nucleotide sequence that is adapted over at least part of its length to anneal by complementary base pairing to a part of said single stranded DNA to form a double stranded DNA structure comprising a double stranded stem domain and a single stranded loop domain.
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Dependencies are inferred from the claim text.