Ionis Pharmaceuticals, Inc.

Isis Pharmaceuticals, Inc.

Other

Lonis Pharmaceuticals, Inc.

University of California

The University of Texas System

Astrazeneca Ab

Alnylam Pharmaceuticals, Inc.

Punit P Seth

Eric E Swayze

Thazha P Prakash

Michael Oestergaard

Andrew M Siwkowski

Susan M Freier

Brett P Monia

Michael T Migawa

Mark J Graham

Sanjay Bhanot

Balkrishen Bhat

Robert McKay

Richard S Geary

Edward Wancewicz

Charles Allerson

Michael L McCaleb

Xue-hai Liang

Elizabeth Anne Jefferson

W Brad Wan

Stanley T Crooke

Rosanne M Crooke

Huynh-Hoa Bui

Garth A Kinberger

Susan F Murray

Wen Shen

Guillermo Vasquez

Priyam Singh

Richard Holmes Griffey

Frank Rigo

Stephen Hanessian

Robert A MacLeod

Youngsoo Kim

Bruce S Ross

Shuling Guo

Quanlai Song

Mingming Han

Tamar R Grossman

Jinghua Yu

Tianyuan Zhou

Chrissa A Dwyer

Benjamin R Schroeder

Muthiah Manoharan

Kallanthottathil G Rajeev

Richard Theodore Lee

Yun He

Andrew T Watt

Holly Kordasiewicz

Ivan Zlatev

Jie Xia

Nitin Nitin

Adam Mullick

Joseph M Tuscano

Noriko Satake

Maria Astrid Ölwegård-Halvarsson

Sayten P Patel

Jacqueline T Hecht

William John Drury, Iii

Eric Valeur

Bradley L Merner

Daniel Laurent Knerr

Eva Carina Ämmälä

Shalini Andersson

Satyen P Patel

Robert D Giacometti

Dale E Robinson, Jr

Curt Mazur

Brooke A Anderson

Thazah P Prakash

Karen LaShea Posey

Robert A Macleod

Michael L Mccaleb

Robert Mckay

Bhat Balkrishen

Eric E Swayz

Muthiah Manorharan

COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION

COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

Compositions and methods for modulating apolipoprotein (a) expression

The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.

Conjugated antisense compounds and their use

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

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, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

Selective antisense compounds and uses thereof

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

GLP-1 receptor ligand moiety conjugated oligonucleotides and uses thereof

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.

Allele specific modulators of P23H rhodopsin

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.

Compositions and methods for modulating complement factor B 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.

The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linking group.

Linkage modified oligomeric compounds and uses thereof

The present disclosure 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.

Antisense compounds and uses thereof

Growing community of inventors

Carlsbad, CA, United States of America

Punit P Seth