Defining a new era of oligonucleotides

Defining a new era of oligonucleotides

Downloads

Investor Inquiries

Scientific Presentations

Scientific Presentations

Investor Events Scientific Presentations Publications
Application of ADAR-mediated RNA editing to modulate gene expression
Source
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
Source
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
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
Source
American Society of Gene & Cell Therapy 26th Annual Meeting (May 2023)
Application of ADAR-mediated RNA editing to modulate protein-protein interactions
Source
European Society of Gene & Cell Therapy 29th Congress (October 2022)
RNA base editing for the treatment of Alpha-1 antitrypsin deficiency
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
Effect of stereochemistry and backbone chemistry on AIMer RNA editing efficiency
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
Phosphoryl-guanidine backbone chemistry: understanding its impact on stereopure oligonucleotides
Source
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
Source
18th Annual Meeting of the Oligonucleotide Therapeutics Society (October 2022)
Stereopure oligonucleotides incorporating phosphoryl guanidine backbone increase durability of gene silencing by RNAi
Source
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
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
Source
ENCALS Meeting (June 2022)
Stereopure AIMers for the treatment of Alpha-1 antitrypsin deficiency (AATD)
Source
TIDES USA: Oligonucleotides and Peptide Therapeutics (May 2022)
Chemically Optimized Stereopure Oligonucleotides Direct ADAR-mediated RNA Editing
Source
7TH ANNUAL OLIGONUCLEOTIDE & PRECISION THERAPEUTICS CONGRESS (March 2022)
A Novel Quantitative Wild-type Huntingtin (wtHTT) Protein Biomarker Method for Human Cerebrospinal Fluid
Source
CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
Innovations that Led to SELECT-HD, a Phase 1b/2a Clinical Trial of an Allele-selective Therapy for Huntington’s Disease
Source
CHDI 17TH ANNUAL HD THERAPEUTICS CONFERENCE (March 2022)
Towards the Development of a Therapeutic RNA Editing Platform
Source
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
Source
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
Source
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
Source
17TH ANNUAL MEETING OF THE OLIGONUCLEOTIDE THERAPEUTICS SOCIETY (September 2021)
Predicting Exon Criticality from Primary Protein Structure
Source
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
Source
KEYSTONE eSYMPOSIA: PRECISION ENGINEERING OF THE GENOME, EPIGENOME AND TRANSCRIPTOME (March 2021)
Stereopure Oligonucleotides in Development for the Treatment of Genetically Defined Diseases
Source
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
Source
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)