Publications

Our scientific publications underscore our best-in-class, proprietary chemistry and innovations in drug discovery and development.

• April 2023

  Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference

  View Publication
• April 2022

  Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD

  View Publication
• April 2022

  Improvements to Hybridization-Ligation Enzyme-Linked Immunosorbent Assay Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics

  View Publication
• April 2022

  Development of a Sensitive Trial-ready Poly(GP) CSF Biomarker Assay for C9orf72-associated Frontotemporal Dementia and Amyotrophic Lateral Sclerosis

  View Publication
• March 2022

  Predicting Exon Criticality from Protein Sequence

  View Publication
• March 2022

  Endogenous ADAR-mediated RNA Editing in Non-human Primates using Stereopure Chemically Modified Oligonucleotides

  View Publication
• February 2022

  Impact of Guanidine-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS

  View Publication
• January 2022

  Control of Backbone Chemistry and Chirality Boost Oligonucleotide Splice Switching Activity

  View Publication
• February 2021

  Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

  View Publication
• January 2021

  Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species

  View Publication
• December 2020

  Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease

  View Publication
• September 2020

  Investigational Assay for Haplotype Phasing of the Huntingtin Gene

  View Publication
• June 2020

  Genotyping Single Nucleotide Polymorphisms for Allele-Selective Therapy in Huntington’s Disease

  View Publication
• September 2017

  Control of Phosphorothioate Stereochemistry Substantially Increases the Efficacy of Antisense Oligonucleotides

  View Publication