Events & Publications
Investor Events
Sep 20, 2022
8:30 AM EDT
8:30 AM EDT
View Presentation 2.3 MB
Aug 11, 2022
8:30 AM EDT
8:30 AM EDT
View Presentation 1.7 MB
Jun 8, 2022
3:00 PM EDT
3:00 PM EDT
May 24, 2022
3:30 PM
3:30 PM
Scientific Presentations
Application of ADAR-mediated RNA editing to modulate gene expression
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
VIEW POSTER 2.2 MB
PN modification of stereopure GalNAc-siRNAs enhances durability of human HSD17B13 silencing in transgenic mouse model
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
VIEW POSTER 3.5 MB
Phosphoryl guanidine (PN)-containing oligonucleotides support exon skipping in skeletal muscle in mice and boys with DMD
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
View Presentation 1.4 MB
RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
View Presentation 3 MB
Application of ADAR-mediated RNA editing to modulate protein-protein interactions
Source
European Society of Gene & Cell Therapy 29th Congress (October 2022)
VIEW POSTER 1.3 MB
RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
VIEW PRESENTATION 2.5 MB
Phosphoryl-guanidine backbone chemistry: understanding its impact on stereopure oligonucleotides
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
VIEW PRESENTATION 4.5 MB
Synthesis of stereopure chimeric oligonucleotides containing PN and PS backbone: A systematic evaluation of chiral auxiliaries
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
VIEW POSTER 1.4 MB
Stereopure oligonucleotides incorporating phosphoryl guanidine backbone increase durability of gene silencing by RNAi
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
VIEW POSTER 982.6 KB
Effect of stereochemistry and backbone chemistry on AIMer RNA editing efficiency
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
VIEW POSTER 1.4 MB
Synthesis and application of stereopure guanidine containing backbone to multiple oligonucleotide modalities in preclinical studies
Source
24th International Roundtable on Nucleosides, Nucleotides and Nucleic Acids (August 2022)
VIEW PRESENTATION 1.9 MB
Targeting pathological transcriptional variants in C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD): Initial results from the ongoing FOCUS-C9 clinical trial
Source
ENCALS Meeting (June 2022)
VIEW PRESENTATION 942.6 KB
Stereopure AIMers for the treatment of Alpha-1 antitrypsin deficiency (AATD)
Source
TIDES USA: Oligonucleotides and Peptide Therapeutics (May 2022)
VIEW PRESENTATION 4.6 MB
Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing
Source
7TH ANNUAL OLIGONUCLEOTIDE & PRECISION THERAPEUTICS CONGRESS (March 2022)
VIEW PRESENTATION 2.5 MB
Innovations that Led to SELECT-HD, a Phase 1b/2a Clinical Trial of an Allele-selective Therapy for Huntington’s Disease
Source
CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
VIEW PRESENTATION 1.8 MB
A Novel Quantitative Wild-type Huntingtin (wtHTT) Protein Biomarker Method for Human Cerebrospinal Fluid
Source
CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
VIEW POSTER 868.5 KB
Towards the Development of a Therapeutic RNA Editing Platform
Source
3RD INTERNATIONAL CONFERENCE ON BASE EDITING – ENZYMES AND APPLICATIONS (DEAMINET 2022) (January 2022)
VIEW PRESENTATION 858.1 KB
Chemically-optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing of SERPINA1 Transcripts, Yielding Functional Alpha-1 Antitrypsin Protein in a Mouse Model for Alpha-1 Antitrypsin Deficiency
Source
AASLD: THE LIVER MEETING (November 2021)
VIEW POSTER 1.3 MB
SELECT-HD: An Adaptive First-in-human Clinical Trial to Evaluate WVE-003, an Investigational Allele Selective mHTT-lowering Oligonucleotide, in Early Manifest Huntington's Disease
Source
28TH ANNUAL MEETING OF THE HUNTINGTON STUDY GROUP (November 2021)
VIEW PRESENTATION 1.2 MB
Exploring New Oligonucleotide Backbone Chemistries and Their Deployment to Improve the Properties of Stereopure Oligonucleotides
Source
TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
VIEW PRESENTATION 1.6 MB
Enhancing the Pharmacologic Profiles of CNS Targeting Therapeutic Oligonucleotides
Source
TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
VIEW PRESENTATION 892.7 KB
Impact of Nitrogen-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
Source
TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
VIEW POSTER 960.2 KB
Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing of SERPINA1 Transcripts, Yielding Functional Alpha-1 Antitrypsin Protein in a Mouse Model for Alpha-1 Antitrypsin Deficiency
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW PRESENTATION 930.4 KB
A Versatile Platform for ADAR-mediated RNA Editing In Vivo in Preclinical Models
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 901.7 KB
Predicting Exon Criticality from Primary Protein Structure
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 407.3 KB
In Vitro Models for the Assessment of Antisense Oligonucleotide Induced Hepatotoxicity
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 903.9 KB
Impact of Nitrogen-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 839 KB
Allele-selective Reduction of Rho P23H-mutant Rhodopsin Rescues Phenotype Associated with Retinitis Pigmentosa in Preclinical Models
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 1.5 MB
Variant-Selective Stereopure Oligonucleotides Protect Against Pathologies Associated with C9orf72-repeat Expansion in Preclinical Models
Source
KEYSTONE eSYMPOSIA: NEURODEGENERATIVE DISEASES: GENES, MECHANISMS AND THERAPEUTICS (June 2021)
VIEW POSTER 2.2 MB
A versatile platform for ADAR-mediated RNA editing in vivo in preclinical models
Source
AMERICAN SOCIETY OF GENE + CELL THERAPY 24TH ANNUAL MEETING (May 2021)
VIEW PRESENTATION 936.9 KB
Design of an Adaptive Phase 1b/2a Randomized Controlled Trial of WVE-004 in Patients with C9orf72-ALS/FTD
Source
EUROPEAN NETWORK TO CURE ALS (ENCALS) VIRTUAL MEETING (May 2021)
VIEW POSTER 1.1 MB
A Ph1b/2a study of WVE-003, an investigational allele-selective, mHTT–lowering oligonucleotide for the treatment of early manifest Huntington’s disease, and review of PRECISION-HD results
Source
CHDI FOUNDATION 16TH ANNUAL HD THERAPEUTICS CONFERENCE (April 2021)
VIEW PRESENTATION 1.8 MB
A Versatile Platform for ADAR-mediated RNA Editing in vivo in Preclinical Models
Source
KEYSTONE eSYMPOSIA: PRECISION ENGINEERING OF THE GENOME, EPIGENOME AND TRANSCRIPTOME (March 2021)
VIEW POSTER 783.8 KB
Stereopure Oligonucleotides in Development for the Treatment of Genetically Defined Diseases
Source
TIDES: OLIGONUCLEOTIDES AND PEPTIDE THERAPEUTICS 2020 MEETING (September 2020)
VIEW PRESENTATION 1.4 MB
Stereopure oligonucleotides produce potent and durable activity in the eye supporting their development for inherited retinal diseases
Source
TIDES: OLIGONUCLEOTIDES AND PEPTIDE THERAPEUTICS 2020 MEETING (September 2020)
VIEW PRESENTATION 2.8 MB
RNA Editing via Endogenous ADARs Using Stereopure Oligonucleotides
Source
1ST INTERNATIONAL CONFERENCE ON BASE EDITING - ENZYMES AND APPLICATIONS (DEAMINET 2020) (January 2020)
VIEW POSTER 443.1 KB
Publications
Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference
Source
NUCLEIC ACIDS RESEARCH (April 2023)
Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD
Source
MOLECULAR THERAPY NUCLEIC ACIDS (April 2022)
Improvements to Hybridization-Ligation Enzyme-Linked Immunosorbent Assay Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics
Source
NUCLEIC ACID THERAPEUTICS (April 2022)
Development of a Sensitive Trial-ready Poly(GP) CSF Biomarker Assay for C9orf72-associated Frontotemporal Dementia and Amyotrophic Lateral Sclerosis
Source
JOURNAL OF NEUROLOGY, NEUROSURGERY & PSYCHIATRY (April 2022)
Predicting Exon Criticality from Protein Sequence
Source
NUCLEIC ACIDS RESEARCH (March 2022)
Endogenous ADAR-mediated RNA Editing in Non-human Primates using Stereopure Chemically Modified Oligonucleotides
Source
NATURE BIOTECHNOLOGY (March 2022)
Impact of Guanidine-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
Source
NUCLEIC ACIDS RESEARCH (February 2022)
Control of Backbone Chemistry and Chirality Boost Oligonucleotide Splice Switching Activity
Source
NUCLEIC ACIDS RESEARCH (January 2022)
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
Source
NATURE COMMUNICATIONS (February 2021)
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species
Source
TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (January 2021)
Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease
Source
CLINICAL THERAPEUTICS (December 2020)
Investigational Assay for Haplotype Phasing of the Huntingtin Gene
Source
MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT (September 2020)
Genotyping Single Nucleotide Polymorphisms for Allele-Selective Therapy in Huntington’s Disease
Source
NEUROLOGY GENETICS (June 2020)
Control of Phosphorothioate Stereochemistry Substantially Increases the Efficacy of Antisense Oligonucleotides
Source
NATURE BIOTECHNOLOGY (September 2017)