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Scientific Publications & Presentations

June 23-26, 2024
FORCE™ platform for the Development of Targeted Therapeutics for Rare Muscle Diseases Presented at the New Directions in Biology and Disease of Skeletal Muscle Conference
June 13-14, 2024
The FORCE™ platform achieves robust and durable DUX4 suppression and functional benefit in FSHD mouse models Presented at the 31st Annual FSHD Society International Research Congress
May 5-8, 2024
Costs and Healthcare Resource Utilization Evaluation in Myotonic Dystrophy Type 1: Results from the Real-world CARE-DM1 Study Presented at ISPOR 2024
April 13-18, 2024
Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping
Presented at the American Academy of Neurology (AAN) Annual Meeting Oral Presentation Poster
April 9-13, 2024
Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1) Presented at the 14th International Myotonic Dystrophy Consortium (IDMC) Meeting
April 9-13, 2024
Costs and Healthcare Resource Utilization Evaluation in Myotonic Dystrophy Type 1: Results from the Real-World CARE-DM1 Study Presented at the 14th International Myotonic Dystrophy Consortium (IDMC) Meeting
March 3-6, 2024
Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping Presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
March 3-6, 2024
Initial Data from the ACHIEVE Trial of DYNE-101 in Adults with Myotonic Dystrophy Type 1 (DM1) Presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
February 16, 2024
Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials Presented at Parent Project Italy’s XXI International Conference on Duchenne and Becker Muscular Dystrophy
January 2, 2024
Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials Published in Research Involvement and Engagement
May 17, 2023
The FORCE™ Platform Delivers Oligonucleotides to the Brain in a DM1 Mouse Model and in NHPs Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
May 7-10, 2023
The Humanistic Burden of Myotonic Dystrophy Type 1: A Literature Review Presented at ISPOR 2023
April 22-27, 2023
ACHIEVE Trial, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals With Myotonic Dystrophy Type 1 (DM1)
Presented at the American Academy of Neurology (AAN) Annual Meeting Oral Presentation Poster
March 19-22, 2023
ACHIEVE Trial, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals With Myotonic Dystrophy Type 1 (DM1) Presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
March 19-22, 2023
DELIVER, a Randomized, Double-blind, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-251 in Boys With DMD Amenable to Exon 51 Skipping Presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
March 19-22, 2023
FORCE™ Platform Achieves Robust Exon Skipping, Restores Dystrophin at the Sarcolemma and Halts Progression of Fibrosis in the Severe D2-mdx Model of DMD Presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
October 11–15, 2022
Building a FORCE™ platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping Presented at the 27th International Hybrid Annual Congress of the World Muscle Society
August 10, 2022
Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice Published in Nucleic Acids Research (NAR)
May 16, 2022
Repeat Dosing with DYNE-101 is Well Tolerated and Leads to a Sustained Reduction of DMPK RNA Expression in Key Muscles for DM1 Pathology in hTfR1/DMSXL Mice and NHPs Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
October 1, 2021
FORCE™ Platform Delivers Exon Skipping PMO, Leads to Durable Increases in Dystrophin Protein in mdx Mice and Is Well Tolerated in NHPs Presented at the 2021 Muscle Study Group Annual Scientific Meeting
September 20-24, 2021
The FORCE™ Platform Achieves Durable Knockdown of Toxic Human Nuclear DMPK RNA and Correction of Splicing in the hTfR1/DMSXL Mouse Model. Presented at the World Muscle Society 2021 Virtual Congress
June 25, 2021
FORCE™ Platform Enables Muscle-Targeted Delivery of Antisense Oligonucleotide and Silencing of DUX4 Activity in an FSHD Cell Line Presented at 28th Annual FSHD Society International Research Congress
May 14, 2021
Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides Presented at American Society of Gene & Cell Therapy Annual Meeting (ASGCT)
May 14, 2021
The FORCE™ Platform Achieves Robust Knock Down of Toxic Human Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in Newly Developed hTfR1/DMSXL Mouse Model Presented at American Society of Gene & Cell Therapy Annual Meeting (ASGCT)
May 12, 2020
Targeted Delivery of ASOs Demonstrates Potential to Treat Duchenne Muscular Dystrophy Presented at ASGCT 2020

Supporting Literature

Platform
Development of Antibody-siRNA Conjugate Targeted to Cardiac and Skeletal Muscles Journal of Controlled Release
Metabolic Catastrophe in Mice Lacking Transferrin Receptor in Muscle EBio Medicine
DM1
Targeting Nuclear RNA for In Vivo Correction of Myotonic Dystrophy Nature
Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1 Nucleic Acids Research (NAR)
In Situ Fluorescence Analysis Demonstrates Active siRNA Exclusion From the Nucleus by Exportin 5 Nucleic Acids Research (NAR)
RNase H1-Dependent Antisense Oligonucleotides Are Robustly Active in Directing RNA Cleavage in Both the Cytoplasm and the Nucleus Molecular Therapy
The Current State and Future Directions of RNAi-based Therapeutics Nature Reviews Drug Discovery
Quantitative Fluorescence Imaging Determines the Absolute Number of Locked Nucleic Acid Oligonucleotides Needed for Suppression of Target Gene Expression Nucleic Acids Research (NAR)
Cellular Localization of Long Non-Coding RNAs Affects Silencing by RNAi More Than by Antisense Oligonucleotides Nucleic Acids Research (NAR)
RNAi Factors Are Present and Active in Human Cell Nuclei Cell Reports
RNA-mediated Therapies in Myotonic Dystrophy Drug Discovery Today
DMPK Gene Deletion or Antisense Knockdown Does Not Compromise Cardiac or Skeletal Muscle Function in Mice Human Molecular Genetics
FSHD
Morpholino-Mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics Molecular Therapy
Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy (FSHD) Genes
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