Sirna Therapeutics, Inc.

Merck Sharp + Dohme Corp.

Microsoft Technology Licensing, LLC

Steven Bartz

Duncan F Brown

Michael Robinson

Peter S Linsley

Julja Burchard

Aimee L Jackson

Guy L Cavet

Lee Weng

Hongyue Dai

Janell M Schelter

Ge Wei

Wei Ge

Yong Ma

The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of HBV gene expression and/or activity, and/or modulate a HBV gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.

RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (SINA)

RNA interference mediated inhibition of hepatitis B Virus (HBV) gene expression using short interfering nucleic acid (siNA)

RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (siNA)

The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of IDH1 and mutant IDH1 gene expression and/or activity, and/or modulate an IDH1 or mutant IDH1 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules, including small nucleic acid molecules such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, that are capable of mediating or that mediate RNA interference (RNAi) against IDH1 or mutant IDH1 gene expression.

RNA interference mediated inhibition of isocitrate dehydrogenase (IDH1) gene expression

The invention provides methods and compositions for gene silencing by RNA interference. In particular, the invention provides methods for gene silencing or RNA knockdown using small interfering RNAs (siRNAs) having partial sequence homology to its target gene. The invention also provides methods for identifying common and/or differential responses to a plurality of different siRNAs targeting a gene. The invention also provides methods for evaluating the relative activity of the two strands of an siRNA. The invention further provides methods of designing siRNAs for gene silencing. The invention further provides methods of using siRNAs as therapeutics for treatment of diseases.

Methods and compositions for RNA interference

The present invention provides a method for identifying siRNA target motifs in a transcript using a position-specific score matrix approach. The invention also provides a method for identifying off-target genes of an siRNA using a position-specific score matrix approach. The invention further provides a method for designing siRNAs with higher silencing efficacy and specificity. The invention also provides a library of siRNAs comprising siRNAs with high silencing efficacy and specificity.

Method for selecting SIRNAs from a plurality of SIRNAs for gene silencing

The present invention provides methods and computer programs for detecting variations in measurements of a biological variable, e.g., variations in cell viability, under different conditions, e.g., with or without treatment of a drug, under the treatments of different drugs, or under different environmental conditions. The methods and programs of the invention determine a metric of difference between measurements under different condition, and make use of predetermined errors of the measurements, e.g., errors determined from an error model, to estimate the errors in the metric of difference. Such an approach provides a more accurate estimate of the errors of measurements, which, in turn, provides a more accurate estimate of the error of the difference metric.

Methods and computer systems for analyzing high-throughput assays

Method of designing siRNAs for gene silencing

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Seattle, WA, United States of America

Steven Bartz