Ionis Pharmaceuticals, Inc.

Isis Pharmaceuticals, Inc.

Lonis Pharmaceuticals, Inc.

University of Rochester

Robert A MacLeod

Susan M Freier

Youngsoo Kim

Eric E Swayze

Brett P Monia

Tianyuan Zhou

Alexey Revenko

Huynh-Hoa Bui

Gourab Bhattacharjee

C Frank Bennett

Punit P Seth

Seng H Cheng

Eric G Marcusson

Jeffrey R Crosby

Sanjay K Pandey

Bruce M Wentworth

Ssucheng Jeff Hsu

Charles A Thornton

Andrew Leger

Thurman Wheeler

Xiaolin Luo

Minji Jo

Sanjey K Pandey

The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.

Modulators of IRF4 expression

The present embodiments provide methods, compounds, and compositions useful for inhibiting FOXP3 expression, which may be useful for treating, preventing, or ameliorating cancer.

Modulators of FOXP3 expression

The present embodiments provide methods, compounds, and compositions useful for inhibiting EZH2 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with EZH2.

Modulators of EZH2 expression

The present embodiments provide methods, compounds, and compositions useful for inhibiting MALAT1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with MALAT1.

Modulators of MALAT1 expression

The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1.

Modulators of YAP1 expression

Certain embodiments are directed to compounds and compositions targeted to human androgen receptor (AR) for inhibiting androgen receptor levels in a cell, which can be useful for methods of treating cancer and inhibiting cancer cell growth or proliferation.

Modulation of androgen receptor expression

Disclosed herein are antisense compounds and methods for decreasing STAT3 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate hyperproliferative diseases.

Modulation of signal transducer and activator of transcription 3 (STAT3) expression

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

Modulation of dystrophia myotonica-protein kinase (DMPK) expression

The present embodiments provide compounds and methods for reducing expression of Metastasis-Associated-in-Lung-Adenocarcinoma-Transcript-1 (MALAT-1) RNA and/or protein in an animal. Such methods are useful for treating cancer, such as colon cancer, intestinal cancer, lung cancer (e.g. non-small cell lung cancer), liver cancer, and/or prostate cancer. In various aspects, the cancer is a primary cancer.

Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1 (MALAT-1) expression

Disclosed herein are methods for decreasing kallikrein and treating or preventing thromboembolic conditions in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to kallikrein include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Methods for inhibiting kallikrein can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

Methods for modulating kallikrein (KLKB1) expression

Disclosed herein are methods for decreasing kallikrein and treating or preventing inflammatory conditions in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to kallikrein include hereditary angioedema (HAE). Methods for inhibiting kallikrein can also be used as a prophylactic treatment to prevent individuals at risk for developing an inflammatory condition, such as, hereditary angioedema.

Disclosed herein are methods for decreasing Factor 12 and treating or preventing inflammatory conditions in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 12 include hereditary angioedema (HAE). Methods for inhibiting Factor 12 can also be used as a prophylactic treatment to prevent individuals at risk for developing an inflammatory condition, such as, hereditary angioedema.

Growing community of inventors

Carlsbad, CA, United States of America

Robert A MacLeod