Defining a new era of oligonucleotides

Defining a new era of oligonucleotides

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Events & Publications

Events & Publications

Investor Events Scientific Presentations Publications

Investor Events

    • Scientific Presentations
    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)
    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)
    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)
    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)
    Enhancing the Pharmacologic Profiles of CNS Targeting Therapeutic Oligonucleotides
    Source
    TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
    Impact of Nitrogen-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
    Source
    TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
    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)
    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)
    Predicting Exon Criticality from Primary Protein Structure
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    17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
    In Vitro Models for the Assessment of Antisense Oligonucleotide Induced Hepatotoxicity
    Source
    17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
    Impact of Nitrogen-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
    Source
    17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
    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)
    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)
    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)
    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)
    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)
    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)
    Stereopure Oligonucleotides in Development for the Treatment of Genetically Defined Diseases
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    TIDES: OLIGONUCLEOTIDES AND PEPTIDE THERAPEUTICS 2020 MEETING (September 2020)
    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)
    RNA Editing via Endogenous ADARs Using Stereopure Oligonucleotides
    Source
    1ST INTERNATIONAL CONFERENCE ON BASE EDITING - ENZYMES AND APPLICATIONS (DEAMINET 2020) (January 2020)
    Publications
    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
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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)