Isis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc.

Andrew T Watt

Susan M Freier

Mark J Graham

Sanjay Bhanot

Rosanne M Crooke

Diane Tribble

C Frank Bennett

Donna T Ward

Kristina M Lemonidis

Brett P Monia

William A Gaarde

Michael L McCaleb

Tamar R Grossman

Kristina L Lemonidis

Eric E Swayze

Punit P Seth

Thazha P Prakash

Hong Zhang

Mausumee Guha

Michael L Mccaleb

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

Modulators of Complement Factor B

Compositions and methods for modulating complement factor B expression

Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

Methods for treating hypercholesterolemia

Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating hypercholesterolemia, or alternatively for treating or preventing atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

Antisense compounds, compositions and methods are provided for modulating the expression of BCL2-associated X protein. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding BCL2-associated X protein. Methods of using these compounds for modulation of BCL2-associated X protein expression and for treatment of diseases associated with expression of BCL2-associated X protein are provided.

Antisense modulation of BCL2-associated X protein expression

Antisense compounds, compositions and methods are provided for modulating the expression of connective tissue growth factor. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding connective tissue growth factor. Methods of using these compounds for modulation of connective tissue growth factor expression and for treatment of diseases associated with expression of connective tissue growth factor are provided.

Antisense modulation of connective tissue growth factor expression

Compounds, compositions and methods are provided for modulating the RNAi pathway. The compositions comprise oligonucleotides, targeted to nucleic acid molecules encoding EIF2C2. Methods of using these compounds for modulation of RNA interference as well as EIF2C2 expression and for diagnosis and treatment of disease associated with expression of EIF2C2 are provided. Also provided are compounds, compositions and methods for modulating the expression of DDX36. The compositions comprise oligonucleotides, targeted to nucleic acid encoding DDX36. Methods of using these compounds for modulation of DDX36 expression and for diagnosis and treatment of diseases and conditions associated with expression of DDX36 are provided.

Modulation of the RNA interference pathway

Antisense compounds, compositions and methods are provided for modulating the expression of helicase-moi. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding helicase-moi. Methods of using these compounds for modulation of helicase-moi expression and for treatment of diseases associated with expression of helicase-moi are provided.

Antisense modulation of helicase-moi expression

Antisense compounds, compositions and methods are provided for modulating the expression of Interferon gamma receptor 2. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding Interferon gamma receptor 2. Methods of using these compounds for modulation of Interferon gamma receptor 2 expression and for treatment of diseases associated with expression of Interferon gamma receptor 2 are provided.

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Vista, CA, United States of America

Andrew T Watt