Isis Pharmaceuticals, Inc.

Ibis Biosciences, Inc.

Ionis Pharmaceuticals, Inc.

Other

Cold Spring Harbor Laboratory

Lonis Pharmaceuticals, Inc.

Stanley T Crooke

David J Ecker

Richard Holmes Griffey

Steven A Hofstadler

Rangarajan Sampath

John McNeil

Christopher K Mirabelli

Thomas A Hall

Lawrence B Blyn

Eric E Swayze

Walter F Lima

James C Hannis

Lex M Cowsert

C Frank Bennett

Susan M Freier

Kenneth G Draper

Mark W Eshoo

Jared James Drader

Michael T Migawa

Muthiah Manoharan

Punit P Seth

Thazha P Prakash

Balkrishen Bhat

Kenneth W Dobie

Brenda F Baker

Timothy A Vickers

Michael Oestergaard

Anne Bettina Eldrup

Jacqueline R Wyatt

Roland Stoughton

Clarence Frank Bennett

Kevin P Anderson

Venkatraman Mohan

Ronnie C Hanecak

Garth A Kinberger

Xue-hai Liang

Wen Shen

Hongjiang Wu

Yun Jiang

Vivek Samant

Neill K White

Mason Yamashita

Raymond Ranken

Ray Ranken

Vickie L Brown-Driver

David L Spector

Tadeusz Krzysztof Wyrzykiewicz

Kristin Sannes Lowery

Ranga Sampath

Christian Massire

Steve Hofstadler

Walt F Lima

Louis Galvan

Stephen A Hofstadler

Muthiah Monoharan

Neil White

John P Penhune

David W Robbins

Dennis P Murray

Jonathan R Bar-on

Brons M Larson

Clifford T Lewis

Cecil L Basham, Jr

Duane J Knize

Karl H Rudnick

Dino J Sofianos

Joshua Nichols

Heather Murray

The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

Modified compounds and uses thereof

In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.

Compounds and methods for the modulation of proteins

In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of STAT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-cell lymphoma or hepatocellular carcinoma.

Cancer treatment

Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2'-OCHsubstituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.

2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations

In certain embodiments, the present disclosure provides compounds and methods for increasing the antisense activity of an antisense compound in a cell. In certain embodiments, the present disclosure provides methods for identifying antisense compounds having high activity. In certain embodiments, the present disclosure provides methods for identifying antisense compounds that bind to enhancer or repressor proteins.

Compounds and methods for increasing antisense activity

In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of ST AT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-cell lymphoma or hepatocellular carcinoma. The STAT (signal transducers and activators of transcription) family of proteins are DNA-binding proteins that play a dual role in signal transduction and activation of transcription.

Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.

Compositions and methods for modulation of LMNA expression

The present invention provides compounds and methods for modulating target nucleic acids found in organelles or sub-organelles of cells. The invention includes, but is not limited to compounds and methods that modulate target nucleic acids in a sub-nuclear organelle, such as the nucleolus and/or a cajal body. In certain embodiments, the cell is in an animal.

Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals

Growing community of inventors

Carlsbad, CA, United States of America

Stanley T Crooke