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
    Application of ADAR-mediated RNA editing to modulate gene expression
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    American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
    PN modification of stereopure GalNAc-siRNAs enhances durability of human HSD17B13 silencing in transgenic mouse model
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    American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
    Phosphoryl guanidine (PN)-containing oligonucleotides support exon skipping in skeletal muscle in mice and boys with DMD
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    American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
    RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
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    American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
    Application of ADAR-mediated RNA editing to modulate protein-protein interactions
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    European Society of Gene & Cell Therapy 29th Congress (October 2022)
    RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
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    18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
    Synthesis of stereopure chimeric oligonucleotides containing PN and PS backbone: A systematic evaluation of chiral auxiliaries
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    18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
    Stereopure oligonucleotides incorporating phosphoryl guanidine backbone increase durability of gene silencing by RNAi
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    18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
    Effect of stereochemistry and backbone chemistry on AIMer RNA editing efficiency
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    18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
    Phosphoryl-guanidine backbone chemistry: understanding its impact on stereopure oligonucleotides
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    18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
    Synthesis and application of stereopure guanidine containing backbone to multiple oligonucleotide modalities in preclinical studies
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    24th International Roundtable on Nucleosides, Nucleotides and Nucleic Acids (August 2022)
    Targeting pathological transcriptional variants in C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD): Initial results from the ongoing FOCUS-C9 clinical trial
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    ENCALS Meeting (June 2022)
    Stereopure AIMers for the treatment of Alpha-1 antitrypsin deficiency (AATD)
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    TIDES USA: Oligonucleotides and Peptide Therapeutics (May 2022)
    Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing
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    7TH ANNUAL OLIGONUCLEOTIDE & PRECISION THERAPEUTICS CONGRESS (March 2022)
    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)
    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)
    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
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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
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    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)
    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 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)
    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
    Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference
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    NUCLEIC ACIDS RESEARCH (April 2023)
    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)