Sirna Therapeutics, Inc.

Ribozyme Pharmaceuticals, Incorporated

Alios Biopharma, Inc.

Merck Sharp + Dohme Corp.

Aligos Therapeutics, Inc.

Janssen Biopharma, Inc.

Intermune, Inc.

Other

Janssen Pharmaceutica Nv

Janssen Biotech, Inc.

Katholieke Universiteit Leuven

Vertex Pharmaceuticals, Inc.

Janssen Pharmaceuticals, Inc.

Quark Pharmaceuticals, Inc.

Array Biopharma Inc.

Leonid Beigelman

James A McSwiggen

David Bernard Smith

Guangyi Wang

Jasenka Matulic-Adamic

Nassim Usman

Jerome Deval

Alexander Karpeisky

Vivek Kumar Rajwanshi

Alex Karpeisky

Lawrence M Blatt

Vladimir Serebryany

David Sweedler

Antitsa Dimitrova Stoycheva

James Mcswiggen

Scott D Seiwert

Chandra Vargeese

Francine E Wincott

Natalia Dyatkina

Marija Prhavc

Jin Hong

Koen Vandyck

Karl Kossen

Alex B Burgin

Sergei M Gryaznov

Robert Than Hendricks

Peter Haeberli

Lech W Dudycz

Julian Alexander Symons

Rajendra K Pandey

Pierre Jean-Marie Bernard Raboisson

Anthony DiRenzo

Santhosh Kumar Thatikonda

Minghong Zhong

Amber Beaudry

James Patrick Edwards

Peter J Connolly

David Craig McGowan

Stuart Emanuel

Ramachandran Radhakrishnan

Dorothée Alice Marie-Eve Bardiot

Gilles C Bignan

Jyanwei Liu

Anh P Truong

Shawn Patrick Zinnen

Tao Hu

Susan Grimm

Danuta Tracz

Arnaud Didier Marie Marchand

Donald Ruhrmund

Mark Richter

Luca Francesco Mario Raveglia

Stefania Vallese

Steven Wade Andrews

Haiying He

Thale Jarvis

Markus Hossbach

Daniele Andreotti

Laurent Bellon

Carmela Napolitano

Anh Truong

Elena Feinstein

Brad Owen Buckman

Timothy S Kercher

Luigi Anzalone

Yi Jin

James D Thompson

Young Chun Jung

Sandrine Vendeville

April L Kennedy

Pamela Pavco

Karin Ann Stein

Yutong Jiang

Julia Haas

Tianbao Lu

Tod M Woolf

Igor Mett

Kevin Ronald Condroski

Bin Wang

Weimin Wang

Hagar Kalinski

Sharon Avkin-Nachum

Williamson Ziegler Bradford

Andras Horvath

Michael Lyon

Luca Francesco Raveglia

Rami Skaliter

Fritz Eckstein

Ronald R Breaker

Peter Jamison Rose

Tongqian Chen

Kathy L Fosnaugh

David Morrissey

Bharat Chowrira

Min Jiang

Yannick Debing

Robert J Kaus

Hua Tan

Described are short interfering nucleic acid (siNA) molecules comprising modified nucleotides, compositions, and methods and uses thereof.

Modified short interfering nucleic acid (siNA) molecules and uses thereof

The present disclosure relates to compounds and compositions containing 5'-phosphoramidite nucleoside monomers, and methods of making and use.

Modified nucleoside phosphoramidites

Provided herein are compounds of Formula (I), or pharmaceutically acceptable salts thereof, pharmaceutical compositions that include a compound described herein (including pharmaceutically acceptable salts of a compound described herein) and methods of synthesizing the same. Also provided herein are methods of treating diseases and/or conditions with a compound of Formula (I), or a pharmaceutically acceptable salt thereof.

Anti-viral compounds

Disclosed herein are nucleotide analogs, methods of synthesizing nucleotide analogs and methods of treating diseases and/or conditions such as a HCV infection with one or more nucleotide analogs.

Substituted nucleosides, nucleotides and analogs thereof

Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2'-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap; and (b) a short interfering nucleic acid (siNA).

Modified oligonucleotides comprising modifications at the 2' and/or 3′ position(s) along with methods of making and use, e.g., against HBV are disclosed.

Modified oligonucleotides and methods of use

A method of making a compound represented by Formula (I): includes a reducing step, a protecting step, and a phosphitylating step, where X is NH; Y is OCF, —O(CR)CR, —O(CR)OCR, or —O(CR)—CR═CR; Z is H; B is adenine (A), guanine (G), thymine (T), cytosine (C), uracil (U), 5-methylcytosine (5-me-C), or a protected version of A, G, T, C, U, or 5-me-C; each Ris independently Calkyl or cycloalkyl; Ris CHCHCN or Calkyl; or Rand Rtogether form an optionally substituted Ccycloalkyl; Ris H or PG; PG is a protecting group; Ris independently in each instance H or F; a is 1 or 2; and b is 1, 2, or 3.

The present disclosure relates to small interfering RNA (siRNA) molecules directed to mRNA transcripts of CD274 to cause downregulation of programmed death-ligand 1 (PD-L1) expression in humans. The siRNA can be constructed of unmodified nucleotides or modified nucleotides that exhibit modified sugars, nucleobases, linkages, or covalently bound targeting moieties. Also disclosed herein are pharmaceutical compositions of siRNAs and uses of or methods of using the siRNAs for the treatment of PD-L1 related diseases including but not limited to liver diseases, cancer, hepatocellular carcinoma, viral diseases, or hepatitis B.

Methods and compositions for targeting PD-L1

Disclosed herein are compounds of Formula I: or a stereoisomer or a tautomer thereof, or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising such compounds, and methods of treating disease by administering or contacting a subject with one or more of the above compounds.

Bicyclic pyridazinones and methods of use thereof

Disclosed herein are compounds of Formula I': or a stereoisomer or a tautomer thereof, or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising such compounds, and methods of treating disease by administering or contacting a subject with one or more of the above compounds.

Oxindoles and methods of use thereof

Disclosed herein are compounds of Formula I&#x2033;: or a stereoisomer or a tautomer thereof, or a pharmaceutically acceptable salt thereof, pharmaceutical compositions comprising such compounds, and methods of treating disease by administering or contacting a subject with one or more of the above compounds.

2-pyridones and methods of use thereof

Provided herein are compounds of Formula (I), Formula (II) and/or Formula (III), or pharmaceutically acceptable salts of any of the foregoing, pharmaceutical compositions that include a compound described herein (including pharmaceutically acceptable salts of a compound described herein) and methods of synthesizing the same. Also provided herein are methods of treating diseases and/or conditions with a compound of Formula (I), Formula (II) and/or Formula (III), or a pharmaceutically acceptable salt of any of the foregoing.

Modified cyclic dinucleoside compounds as sting modulators

Growing community of inventors

San Mateo, CA, United States of America

Leonid Beigelman