Ionis Pharmaceuticals, Inc.

Isis Pharmaceuticals, Inc.

Regulus Therapeutics Inc.

Other

University of Utah Research Foundation

Biogen Ma Inc.

University of California

Genzyme Corporation

Cold Spring Harbor Laboratory

The Hospital for Sick Children

The Johns Hopkins University

Astrazeneca Ab

University of British Columbia

Baylor College of Medicine

Ludwig Institute for Cancer Research Limited

Susan M Freier

C Frank Bennett

Eric E Swayze

Brett P Monia

Sanjay Bhanot

Richard Holmes Griffey

Mark J Graham

Rosanne M Crooke

Huynh-Hoa Bui

Kenneth W Dobie

Punit P Seth

Gene Hung

Robert A MacLeod

Jacqueline R Wyatt

Holly Kordasiewicz

Youngsoo Kim

Michael L McCaleb

Andrew T Watt

Don W Cleveland

David J Ecker

Seng H Cheng

Frank Rigo

Thazha P Prakash

Robert McKay

Andrew M Siwkowski

Lamya S Shihabuddin

James G Karras

Lisa Stanek

Lex M Cowsert

Brenda F Baker

Eric G Marcusson

Ravi Jain

Diane Tribble

Timothy A Vickers

Shuling Guo

Hong Zhang

Jeffrey R Crosby

Kristina M Lemonidis

Tamar R Grossman

Balkrishen Bhat

Stanley T Crooke

Marc Lim

Andrew Dibble

Bridget Lollo

Chenguang Zhao

Tianyuan Zhou

Phillip Dan Cook

Yogesh S Sanghvi

Donna T Ward

William A Gaarde

Thomas P Condon

Susan F Murray

Sarah Greenlee

Priyam Singh

Stefan M Pulst

Berge Minassian

Christine Esau

Edward Wancewicz

Madeline M Butler

Xing-Xian Yu

Erich Koller

Daniel R Scoles

Sanjay K Pandey

Janet M Leeds

Mausumee Guha

David L Spector

Adam Mullick

Lynnetta Watts

Susan Gregory

Muthiah Manoharan

Steven A Hofstadler

Nicholas M Dean

Rangarajan Sampath

John McNeil

Thomas W Bruice

Michael R Hayden

Kevin P Anderson

Walter F Lima

Ronnie C Hanecak

Michael G Rosenfeld

Peter W Davis

Huda Y Zoghbi

Richard Alan Smith

Niall O'Donnell

Vickie L Brown-Driver

Jeffrey D Rothstein

Ian Popoff

William R Shanahan

J Gordon Foulkes

Hien Thuy Zhao

Bruce M Wentworth

Ssucheng Jeff Hsu

Charles A Thornton

Christopher K Glass

Alexey Revenko

Kristin Sannes Lowery

Alexander H Borchers

Henri M Sasmor

Andrew Leger

Jeffrey Carroll

Methods, compounds, and compositions useful for inhibiting HSD17B13 expression are provided. Such compounds, compositions, and methods are useful for treating, preventing, or ameliorating a disease associated with HSD 17B 13.

Modulators of HSD17B13 expression

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include SCA3.

Compounds and methods for reducing ATXN3 expression

Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.

Compositions and methods for modulating MECP2 expression

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

Modulation of huntingtin expression

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.

Compounds and methods for reducing prion expression

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.

Compounds and methods for reducing LRRK2 expression

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include gait and limb ataxia, cognitive impairments, difficulty with speaking and swallowing, atrophy of the cerebellum and brainstem in magnetic resonance imaging (MRI), neurochemical abnormalities in the cerebellum and brainstem detected via magnetic resonance spectroscopy (MRS), and death within 10-15 years of symptom onset. Such neurodegenerative diseases include Spinocerebellar ataxia type 1.

Compounds and methods for modulating ATXN1

The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.

Modulators of APOL1 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

Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of methyl CpG binding protein 2 (MECP2) RNA in a cell or animal, and in certain instances reducing the amount of MECP2 protein in a cell or animal Such oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodevelopmental disease or disorder. Such neurodevelopmental diseases or disorders include MECP2 duplication syndrome. Such symptoms or hallmarks include autism, intellectual disability, motor dysfunction, hypotonia, global developmental delays, gastrointestinal symptoms, anxiety, epilepsy, recurrent respiratory tract infections, epileptic encephalopathy, and early death.

Compounds and methods for reducing MECP2 expression

Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).

Selective reduction of allelic variants

Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

Compositions for modulating tau expression

Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression. Also disclosed herein are methods for treating, preventing, and ameliorating neurodegenerative diseases in an individual in need thereof.

Modulation of alpha synuclein expression

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

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.

Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression

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San Diego, CA, United States of America

Susan M Freier