Dharmacon, Inc.

University of Massachusetts

Thermo Fisher Scientific Gmbh

Rxi Pharmaceuticals Corporation

Phio Pharmaceuticals Corp.

Ge Healthcare Dharmacon, Inc.

Beth Israel Deaconess Medical Center, Inc.

Thermo Fisher Scientific Biosciences Inc.

Merck + Co., Inc.

University Technology Corporation

Elitech Holding B.v.

Elitechgroup B.v.

Rxi Parmaceuticals Corporation

Merck Sharp + Dohme Corp.

Anastasia Khvorova

Devin Leake

William Marshall

Angela Reynolds

Stephen Scaringe

Steven Read

Joanne Kamens

Tod M Woolf

William Salomon

Dmitry Samarsky

James Cardia

Neil Aronin

Julia Alterman

Pamela A Pavco

Matthew Hassler

Anton A Turanov

Lyn Libertine

Bruno Miguel Da Cruz Godinho

Barbara Robertson

Ken Yamada

Karen G Bulock

Jon Michael Karpilow

Christina Yamada

Annaleen Vermeulen

Rob Kaiser

Ananth Karumanchi

Annabelle Biscans

Sarah Davis

Reka Haraszti

Melissa Moore

Melissa Kelley

Amanda Birmingham

Kevin Sullivan

Emily Anderson

Reka Agnes Haraszti

Kathryn Robinson

Mehran Nikan

Nicolaas M J Vermeulen

Walt Mahoney

Edith L Pfister

Loïc Maurice René Jean Roux

Faith Conroy

Zachary Kennedy

Marie Cecile Didiot

Peter S Linsley

Michael J Yarus

Jonathan Watts

Anja Smith

William S Marshall

Maire Osborn

Andrew Coles

Michelle Miller

Chantal Ferguson

Vignesh Narayan Hariharan

Nicholas McHugh

Jacquelyn Sousa

Jillian Caiazzi

Pranathi Meda Krishnamurthy

Gitali Devi

Dimas Echeverria Moreno

Yann Thillier

Daniel O′Reilly

Scott Baskerville

Yuriy Fedorov

Michael O Delaney

James W Gilbert

Kathryn Monopoli

Qi Tang

Samuel Hildebrand

Socheata Ly

Minwook Shin

Kimberly Nichols

Jon Karpilow

Karen Gunn Bulock

Nicolaas Vermeulen

Barbara Roberson

This disclosure relates to an siRNA-lipid conjugate of formula Y-L-(H). Y is an siRNA molecule, L is a linker covalently bonded to Y and H, each H is independently a hydrophobic chain comprising 5 to 50 carbon atoms, n is 1, 2, or 3, and linker L is bonded to the 3' end of the sense strand of the siRNA.

Branched lipid conjugates of siRNA for specific tissue delivery

This disclosure relates to novel C9ORF72 targeting sequences. Novel sense and antisense dual-targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

Dual-acting siRNA based modulation of C9orf72

Novel oligonucleotides that are fully chemically stabilized are provided. Methods of using oligonucleotides that are fully chemically stabilized are also provided.

O-methyl rich fully stabilized oligonucleotides

This disclosure relates to novel PRNP targeting sequences. Novel PRNP targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

Oligonucleotides for PRNP modulation

Therapeutic oligonucleotides comprising pharmacokinetic (PK)-modifying anchors are provided. Methods for treating diseases or disorders comprising administering to a subject a therapeutic oligonucleotide comprising one or more PK-modifying anchors are provided.

Dynamic pharmacokinetic-modifying anchors

Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.

Fully stabilized asymmetric siRNA

This disclosure relates to the synthesis of novel modified oligonucleotides. The synthesis of novel phosphoramidites are also provided.

Synthesis of modified oligonucleotides with increased stability

Bioactive conjugates for oligonucleotide delivery

This disclosure relates to novel SARS-CoV-2 targeting sequences. Novel SARS-CoV-2 targeting oligonucleotides for the treatment of SARS-CoV-2 infection are also provided.

Oligonucleotides for SARS-CoV-2 modulation

Provided are compositions and methods comprising two-tailed siRNAs (tt-siRNAs) that exhibit unprecedented cellular uptake and silencing. Also provided are methods of treating neurological and other diseases with the two-tailed siRNAs of the invention.

Two-tailed self-delivering siRNA

This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

Oligonucleotides for tissue specific gene expression modulation

Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.

Artificial exosome composition and related methods

Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

Modified oligonucleotides targeting SNPs

Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.

Branched oligonucleotides

Growing community of inventors

Boulder, CO, United States of America

Anastasia Khvorova