Events & Publications
Investor Events
May 24, 2022
3:30 PM
3:30 PM
May 17, 2022
2:05 PM
2:05 PM
May 12, 2022
8:30 AM EDT
8:30 AM EDT
View Presentation 1.6 MB
Apr 26, 2022
2:30 PM EDT
2:30 PM EDT
Scientific Presentations
Stereopure AIMers for the treatment of Alpha-1 antitrypsin deficiency (AATD)
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TIDES USA: Oligonucleotides and Peptide Therapeutics (May 2022)
VIEW PRESENTATION 4.6 MB
Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing
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7TH ANNUAL OLIGONUCLEOTIDE & PRECISION THERAPEUTICS CONGRESS (March 2022)
VIEW PRESENTATION 2.5 MB
A Novel Quantitative Wild-type Huntingtin (wtHTT) Protein Biomarker Method for Human Cerebrospinal Fluid
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CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
VIEW POSTER 868.5 KB
Innovations that Led to SELECT-HD, a Phase 1b/2a Clinical Trial of an Allele-selective Therapy for Huntington’s Disease
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CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
VIEW PRESENTATION 1.8 MB
Towards the Development of a Therapeutic RNA Editing Platform
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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
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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
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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
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TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
VIEW PRESENTATION 1.6 MB
Enhancing the Pharmacologic Profiles of CNS Targeting Therapeutic Oligonucleotides
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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
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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
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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
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17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
VIEW POSTER 901.7 KB
Predicting Exon Criticality from Primary Protein Structure
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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TIDES: OLIGONUCLEOTIDES AND PEPTIDE THERAPEUTICS 2020 MEETING (September 2020)
VIEW PRESENTATION 2.8 MB
RNA Editing via Endogenous ADARs Using Stereopure Oligonucleotides
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1ST INTERNATIONAL CONFERENCE ON BASE EDITING - ENZYMES AND APPLICATIONS (DEAMINET 2020) (January 2020)
VIEW POSTER 443.1 KB
Publications
Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD
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MOLECULAR THERAPY NUCLEIC ACIDS (April 2022)
Improvements to Hybridization-Ligation Enzyme-Linked Immunosorbent Assay Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics
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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
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JOURNAL OF NEUROLOGY, NEUROSURGERY & PSYCHIATRY (April 2022)
Predicting Exon Criticality from Protein Sequence
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NUCLEIC ACIDS RESEARCH (March 2022)
Endogenous ADAR-mediated RNA Editing in Non-human Primates using Stereopure Chemically Modified Oligonucleotides
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NATURE BIOTECHNOLOGY (March 2022)
Impact of Guanidine-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
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NUCLEIC ACIDS RESEARCH (February 2022)
Control of Backbone Chemistry and Chirality Boost Oligonucleotide Splice Switching Activity
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NUCLEIC ACIDS RESEARCH (January 2022)
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
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NATURE COMMUNICATIONS (February 2021)
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species
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TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (January 2021)
Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease
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CLINICAL THERAPEUTICS (December 2020)
Investigational Assay for Haplotype Phasing of the Huntingtin Gene
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MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT (September 2020)
Genotyping Single Nucleotide Polymorphisms for Allele-Selective Therapy in Huntington’s Disease
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NEUROLOGY GENETICS (June 2020)
Control of Phosphorothioate Stereochemistry Substantially Increases the Efficacy of Antisense Oligonucleotides
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NATURE BIOTECHNOLOGY (September 2017)