Defining a new era of oligonucleotides

Defining a new era of oligonucleotides

Events & Publications

Events & Publications

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
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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)
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)