Roche Innovation Center Copenhagen A/s

Santaris Pharma A/s

Roche Innovation Center Copenhagen

Ellen Marie Straarup

Henrik Frydenlund Hansen

Henrik Orum

Niels Fisker Nielsen

Marie Wickstrom Lindholm

Jens Bo Rode Hansen

Gregroy Hardee

Christoph Rosenbohm

Maj Hedtjärn

Marie Lindholm

Jens Bo Hansen

Yu Bagger

Joacim Elmèn

Maj HEDTJÄRN

The invention provides for LNA oligomers, for the treatment of a metabolic or liver disorder, wherein the LNA oligomer is administered orally in a unit dose of less than 50 mgs/kg, wherein the LNA oligomer is administered in the presence of a penetration (permeation) enhancer.

Oral delivery of therapeutically effective LNA oligonucleotides

The present invention relates to oligomer compounds (oligomers), which target APO-B100 mRNA in a cell, leading to reduced expression of APO-B100. Reduction of APO-B100 expression is beneficial for the treatment of certain medical disorders, such as diseases associated with apolipoproteinB activity, such as in non-limiting example, different types of HDL/LDL cholesterol imbalance; dyslipidemias, e.g., familial combined dyslipidemia (FCHL), acquired hyperlipidemia, hypercholesterolemia, statin-resistant hypercholesterolemia; coronary artery disease (CAD), coronary heart disease (CHD), atherosclerosis.

Potent anti APOB antisense compounds

The present invention relates to the use of LNA antisense apoB oligonucleotides for the treatment of acute coronary syndrome.

Use of LNA ApoB antisense oligomers for the treatment of acute coronary syndromes

The invention provides LNA gapmer oligomers of between 10-20 nucleobases in length, which have a total of 1-3 phosphodiester internucleoside linkages. Such oligomers have been found to have superior bioavailability and have also been found to selectively accumulate in kidney cells.

Short oligomer antagonist compounds for the modulation of target mRNA

The present invention relates to oligomer compounds (oligomers), which target PCSK9 mRNA in a cell, leading to reduced expression of PCSK9. Reduction of PCSK9 expression is beneficial for the treatment of certain medical disorders, such as hypercholesterolemia and related disorders.

Antisense oligomers targeting PCSK9

Short oligonucleotides directed against the Apo-B100 gene are provided for modulating the expression of Apo-B100. Methods of using these compounds for modulation of Apo-B100 expression and for the treatment of diseases associated with Apo-B100 expression are provided. The oligonucleotides comprise deoxyribonucleosides and locked nucleic acids.

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Birkerød, Denmark

Ellen Marie Straarup