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
    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
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    TIDES USA: OLIGONUCLEOTIDES & PEPTIDE THERAPEUTICS MEETING (September 2021)
    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)
    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)
    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
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    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
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    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
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    KEYSTONE eSYMPOSIA: NEURODEGENERATIVE DISEASES: GENES, MECHANISMS AND THERAPEUTICS (June 2021)
    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)
    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)
    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)
    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
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    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
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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)