Sirnas targeting slc10a1 transcripts, compositions and uses thereof
AssigneeHemoShear Therapeutics, Inc.
InventorsRyan Feaver, Brian Johns, Jennifer Marks, Emile Velthuisen, Maria Sol Collado, Robert Alan Figler
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Abstract
The present invention relates to siRNA molecules targeting a SLC10A1 mRNA transcript and methods of using the siRNA compositions for treatment of a NTCP associated disorder.
Claims36
A small interfering RNA (siRNA) molecule targeting a SLC10A1 mRNA transcript, comprising a double-stranded region of 17- to 30-base pairs in length that is formed between the complementary base pairs of a sense strand sequence and an antisense strand sequence, wherein the siRNA comprises a sense strand sequence selected from the group consisting of SEQ ID Nos. 1-15, 31-45 and 481-611, and/or an antisense strand sequence selected from the group consisting of SEQ ID Nos. 16-30, 46-60 and 612-742.
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A method of treating a cholestatic disorder in a patient in need thereof comprising administering to the patient a siRNA molecule targeting a human SLC10A1 mRNA transcript, comprising a pair of a sense strand sequence and an antisense strand sequence, wherein the siRNA comprises a sense strand sequence selected from the group consisting of SEQ ID Nos. 1-15, 31-45, 61-270, 481-611, and 743-873, and/or an antisense strand sequence selected from the group consisting of SEQ ID Nos. 16-30, 46-60 271-480, 612-742, and 874-1004.
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A method of treating a viral hepatitis B and/or hepatitis D disease in a subject using a siRNA targeting a human SLC10A1 mRNA transcript, comprising a pair of a sense strand sequence and an antisense strand sequence as a monotherapy or in a regimen combination with one or more antiviral agents, wherein the siRNA comprises a sense strand sequence selected from the group consisting of SEQ ID Nos. 1-15, 31-45, 61-270, 481-611, and 743-873, and/or an antisense strand sequence selected from the group consisting of SEQ ID Nos. 16-30, 46-60 271-480, 612-742, and 874-1004.
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Dependencies are inferred from the claim text.