The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies

Reversal of siRNA-mediated gene silencing in vivo

(2018) Ivan Zlatev et al.   NATURE BIOTECHNOLOGY

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Chemical Modification Study of Antisense Gapmers

(2018) Robert Stanton et al.   Nucleic Acid Therapeutics

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

(2017) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of Oligonucleotide Structure, Chemistry, and Delivery Method on In Vitro Cytotoxicity

(2017) Maja M. Janas et al.   Nucleic Acid Therapeutics

Assessment of the Effects of 2′-Methoxyethyl Antisense Oligonucleotides on Platelet Count in Cynomolgus Nonhuman Primates

(2017) Scott P. Henry et al.   Nucleic Acid Therapeutics

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials

(2017) Stanley T. Crooke et al.   Nucleic Acid Therapeutics

Assessing single-stranded oligonucleotide drug-induced effects in vitro reveals key risk factors for thrombocytopenia

(2017) Sabine Sewing et al.   PLoS One

Rationally designed siRNAs without miRNA-like off-target repression

(2016) Heeyoung Seok et al.   BMB Reports

Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

(2016) Victor Kotelianski et al.   International Journal of Nanomedicine

An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria

(2016) Abigail Liebow et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

OSWG Recommendations for Genotoxicity Testing of Novel Oligonucleotide-Based Therapeutics

(2016) Cindy L. Berman et al.   Nucleic Acid Therapeutics

Exposure to siRNA-GalNAc Conjugates in Systems of the Standard Test Battery for Genotoxicity

(2016) Maja M. Janas et al.   Nucleic Acid Therapeutics

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

(2015) Sebastien A. Burel et al.   NUCLEIC ACIDS RESEARCH

Scientific and Regulatory Policy Committee Points-to-consider Paper*

(2015) Jeffery A. Engelhardt et al.   TOXICOLOGIC PATHOLOGY

Abasic pivot substitution harnesses target specificity of RNA interference

(2015) Hye-Sook Lee et al.   Nature Communications

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

(2014) Andrew D. Burdick et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotide Therapies

(2014) Kendall S. Frazier   TOXICOLOGIC PATHOLOGY

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Species-specific Inflammatory Responses as a Primary Component for the Development of Glomerular Lesions in Mice and Monkeys Following Chronic Administration of a Second-generation Antisense Oligonucleotide

(2013) Kendall S. Frazier et al.   TOXICOLOGIC PATHOLOGY

Comparison of Hepatic Transcription Profiles of Locked Ribonucleic Acid Antisense Oligonucleotides: Evidence of Distinct Pathways Contributing to Non-target Mediated Toxicity in Mice

(2013) Satoko Kakiuchi-Kiyota et al.   TOXICOLOGICAL SCIENCES

Hepatotoxic Potential of Therapeutic Oligonucleotides Can Be Predicted from Their Sequence and Modification Pattern

(2013) Peter H. Hagedorn et al.   Nucleic Acid Therapeutics

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

(2012) Glen F. Deleavey et al.   CHEMISTRY & BIOLOGY

miR-122 regulates hepatic lipid metabolism and tumor suppression

(2012) Jessica Wen et al.   JOURNAL OF CLINICAL INVESTIGATION

Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver

(2012) Shu-hao Hsu et al.   JOURNAL OF CLINICAL INVESTIGATION

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

Preclinical toxicity and toxicokinetics of GTI-2040, a phosphorothioate oligonucleotide targeting ribonucleotide reductase R2

(2010) Eckhardt S. Ferdinandi et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs

(2010) Narendra Vaish et al.   NUCLEIC ACIDS RESEARCH

A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects

(2010) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

Diagnostic Performance of Traditional Hepatobiliary Biomarkers of Drug-Induced Liver Injury in the Rat

(2010) Daniela Ennulat et al.   TOXICOLOGICAL SCIENCES