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US 2022/0025376 A1Published application
Antisense oligomers targeting pcsk9
AssigneeRoche Innovation Center Copenhagen A/S
InventorsNanna ALBÆK, Maj HEDTJÄRN, Marie Wickstrom LINDHOLM, Niels Fisker NIELSEN, Andreas PETRI, Jacob RAVN
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Abstract
The present invention relates to oligomeric compounds and conjugates thereof that target Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) PCSK9 mRNA in a cell, leading to reduced expression of PCSK9. Reduction of PCSK9 expression is beneficial for a range of medical disorders, such as hypercholesterolemia and related disorders.
Claims22
1-26. (canceled)
An antisense oligonucleotide (ASO) comprising 10 to 16 nucleotides from the sequence aatgctacaaaaccca (SEQ ID NO:26) wherein:
- (i) the ASO is a gapmer 10 to 25 contiguous nucleotides in length;
- (ii) the ASO comprises at least one nucleotide analogue;
- (iii) the ASO targets mRNA encoding PCSK9;
- and,
- (iv) the ASO shows less kidney toxicity than an ASO of SEQ ID NO:1 as determined by a KIM-1 expression assay.
2 more elements
Dependencies are inferred from the claim text.