Defining a new era of oligonucleotides

Defining a new era of oligonucleotides

Events & Publications

Events & Publications

Scientific Presentations
Synthesis and application of stereopure guanidine containing backbone to multiple oligonucleotide modalities in preclinical studies
Source
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
Source
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
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
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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
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
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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
Source
1ST INTERNATIONAL CONFERENCE ON BASE EDITING - ENZYMES AND APPLICATIONS (DEAMINET 2020) (January 2020)
Publications
Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD
Source
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
Source
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)