Dicerna Pharmaceuticals, Inc.

Genta Incorporated

Promega Biosciences, Inc.

Gen-probe, Inc.

Oasis Biosciences Incorporated

Bob Dale Brown

Henryk T Dudek

David Aaron Schwartz

Brian Patrick Dwyer

Cheng Lai

William J Daily

Kumar Srinivasan

Utsav Saxena

Lyle J Arnold, Jr

Weimin Wang

Natalie Wayne Pursell

Sujit Basu

Naim Nazef

Timothy A Riley

Lyle J Arnold

Marc Abrams

Wen Han

Boyoung Kim

Rachel Storr

Alexandre V Lehedev

Bo Ying

Anton Turanov

Mark A Reynolds

Richard I Hogrefe

Timothy Andrew Riley

Morteza M Vaghefi

Allister Fraser

Sujit Kumar Basu

Oligonucleotides are provided herein that inhibit apolipoprotein(a) (LPA) expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with LPA expression.

Compositions and methods for inhibiting LPA expression

Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing apolipoprotein C-III (APOC3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with APOC3 expression.

Compositions and methods for modulating APOC3 expression

Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with PNPLA3 expression.

Compositions and methods for modulating PNPLA3 expression

The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

Extended dicer substrate agents and methods for the specific inhibition of gene expression

This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA

Provided herein are double-stranded nucleic acid inhibitor molecules having a sense strand with a stem loop structure and an antisense strand, where the stem portion of the stem loop structure contains one or more T-increasing nucleotides. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

Double-stranded nucleic acid inhibitor molecules modified with Tm-increasing nucleotides

This invention relates to compounds, compositions, and methods useful for reducing Glycolate Oxidase (HAO1) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA

This invention relates to compounds, compositions, and methods useful for reducing C5 target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Methods and compositions for the specific inhibition of Complement Component 5(C5) by double-stranded RNA

This invention relates to compounds, compositions, and methods useful for reducing transthyretin (TTR) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA

This invention relates to compounds, compositions, and methods useful for reducing AT3 target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA

This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Methods and compositions for inhibiting expression of LDHA

This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.

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Millington, NJ, United States of America

Bob Dale Brown