Isis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc.

Other

Ciba-geigy Corporation

Astrazeneca Ab

The University of British Columbia

Novartis Corporation

Lonis Pharmaceuticals, Inc.

National University of Singapore

Brett P Monia

Lex M Cowsert

Susan M Freier

Robert McKay

Sanjay Bhanot

Andrew M Siwkowski

Edward Wancewicz

Punit P Seth

William A Gaarde

Eric E Swayze

Jacqueline R Wyatt

C Frank Bennett

Kenneth W Dobie

Richard S Geary

Phillip Dan Cook

Madeline M Butler

David J Ecker

Nicholas M Dean

Shuling Guo

Xing-Xian Yu

Pamela S Nero

Hong Zhang

Brenda F Baker

Jeffrey R Crosby

Muthiah Manoharan

Mark J Graham

Karl-Heinz Altmann

Robert A MacLeod

Chenguang Zhao

Pierre Martin

Youngsoo Kim

Russell T Boggs

Tianyuan Zhou

Brian J Nickoloff

Yogesh S Sanghvi

Andrew T Watt

James G Karras

Donna T Ward

Michael L McCaleb

Mausumee Guha

Loren J Miraglia

Lynnetta Watts

QingQing Zhang

Thazha P Prakash

Martin E Gleave

Richard Theodore Lee

Vincent P Stanton, Jr

Ravi Jain

Clarence Frank Bennett

Walter F Lima

Paul Rennie

Hideaki Miyake

Colleen Nelson

Doriano Fabbro

Garth A Kinberger

Susan F Murray

Ian Popoff

James P Basilion

Vivian Qingqing Zhang

Thomas Geiger

John G Geisler

Thomas A Leedom

Trisha Lockhart

Maria Astrid Ölwegård-Halvarsson

Eric Valeur

Daniel Laurent Knerr

Eva Carina Ämmälä

Shalini Andersson

William John Drury, Iii

Marcel Muller

Xiaoxing S Xu

Richard A Roth

Stanley T Cooke

Madelline M Butler

Xaoxing S Xu

Namir Sioufi

Robert A Macleod

Robert Mckay

The present embodiments provide compounds and methods for targeting cells expressing GLP-1 receptor.

GLP-1 receptor ligand moiety conjugated oligonucleotides and uses thereof

Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.

Modulation of transthyretin expression

Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

Modulation of factor 11 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

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

Modulation of DYRK1B expression

Disclosed herein are methods for decreasing A1AT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis. Disclosed herein are methods for decreasing A1AT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of liver disease, such as, A1ATD associated liver disease, and pulmonary disease, such as, A1ATD associated pulmonary disease in an individual in need thereof. Methods for inhibiting A1AT mRNA and protein expression can also be used as a prophylactic treatment to prevent individuals at risk for developing a liver disease, such as, A1ATD associated liver disease and pulmonary disease, such as, A1ATD associated pulmonary disease.

Methods and compositions for modulating alpha-1 antitrypsin expression

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, the present invention provides compounds comprising oligonucleotides. In certain embodiments, such oligonucleotides comprise a region having a gapmer sugar motif. In certain embodiments, oligonucleotides comprise one or more type of modified sugar moieties and/or naturally occurring sugar moieties arranged along an oligonucleotide or region thereof in a defined pattern or sugar modification motif.

Oligomeric compounds comprising bicyclic nucleosides and uses thereof

The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.

Compounds and methods for modulating gene expression

Disclosed herein are methods for decreasing AIAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis. Disclosed herein are methods for decreasing AIAT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of liver disease, such as, AIATD associated liver disease, and pulmonary disease, such as, AIATD associated pulmonary disease in an individual in need thereof. Methods for inhibiting AIAT mRNA and protein expression can also be used as a prophylactic treatment to prevent individuals at risk for developing a liver disease, such as, AIATD associated liver disease and pulmonary disease, such as, AIATD associated pulmonary disease.

Methods and compositions for modulating alpha-1-antitrypsin expression

Disclosed herein are methods for decreasing Factor 12 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 Factor 12 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, stroke, and mesenteric thrombosis. Methods for inhibiting Factor 12 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

Methods for modulating factor 12 expression

Short antisense compounds with gapmer configuration

Growing community of inventors

Encinitas, CA, United States of America

Brett P Monia