Sirna Therapeutics, Inc.

Intellia Therapeutics, Inc.

Novartis Ag

Merck Sharp + Dohme Corp.

Seqirus UK Limited

David Morrissey

Chandra Vargeese

Peter W Mason

Walter R Strapps

Juerg Hunziker

Michael Franti

Heidi Trusheim

Amy Madison Rhoden Smith

Melissa Sackal

Leonid Beigelman

James A McSwiggen

Vasant R Jadhav

Nassim Usman

Weimin Wang

James D Thompson

James Mcswiggen

Shawn Patrick Zinnen

Peter Haeberli

Tongqian Chen

Pamela Pavco

Kathy L Fosnaugh

Bharat Chowrira

Narendra Vaish

Bjoern Keiner

Dennis G Macejak

Narendra K Vaish

Björn Keiner

Sharon F Jamison

Roberto Guerciolini

Francois Natt

François Jean-Charles Natt

Dennis Macejak

Victor Mokler

This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.

Modified guide RNAs

Methods for producing reassortant viruses are provided wherein the transcription and/or translation of the hemagglutinin and/or neuraminidase genes are suppressed.

Influenza virus reassortment method

Influenza virus reassortment

The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

RNA interference mediated inhibition of gene expression using chemically modified 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 hepatitis C virus (HCV) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in hepatitis C virus (HCV) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.

RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)

This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of VEGF and/or VEGFR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of VEGF and/or VEGFR genes. The application also relates to methods of treating diseases and conditions associated with VEGF and/or VEGFR gene expression, such as ocular diseases and conditions, including age related macular degeneration (AMD) and diabetic retinopathy, as well as providing dosing regimens and treatment protocols.

RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)

Growing community of inventors

Winchester, MA, United States of America

David Morrissey