Ionis Pharmaceuticals, Inc.

The Hospital for Sick Children

Tamar R Grossman

Susan M Freier

Michael L McCaleb

Eric E Swayze

Punit P Seth

Thazha P Prakash

Andrew T Watt

Berge Minassian

Saija Ahonen

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

Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.

Modulation of GYS1 expression

Provided herein are methods, compounds, and compositions for modulating expression of at least one member of the Notch signaling pathway in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a respiratory disorder associated with excessive mucus production in an individual.

Modulation of the notch signaling pathway for treatment of respiratory disorders

Growing community of inventors

La Jolla, CA, United States of America

Tamar R Grossman