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Dyne Therapeutics’ Preclinical Data Demonstrating FORCE™ Platform Delivery to CNS Featured in Oral Presentation at ASGCT Annual Meeting
- Broad Distribution in CNS Demonstrated in Non-Human Primates - - Achieved Reduction of Toxic Nuclear DMPK RNA and Foci in the Brain in a DM1 Disease Model -
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Broad Distribution in CNS Demonstrated in Non-Human Primates - - Achieved Reduction of Toxic Nuclear DMPK RNA and Foci in the Brain in a DM1 Disease Model - WALTHAM, Mass., May 17, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is delivering an oral presentation today at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles highlighting new preclinical data demonstrating the FORCE™ platform achieved delivery to the central nervous system (CNS) in non-human primates and robust pharmacological effects in the brain in a model of myotonic dystrophy type 1 (DM1). Oral Presentation: FORCE™ Platform Delivers Oligonucleotides to the Brain in a DM1 Mouse Model and in NHPs (abstract #82)Session: Nucleic Acid TherapeuticsDate/Time: Wednesday, May 17, 2023, at 4:00 p.m. PTPresenter: Stefano Zanotti, Ph.D., Executive Director, Head of Neuromuscular Research, Dyne The presentation will be available in the Scientific Publications & Presentations section of Dyne’s website following the session. The FORCE platform was designed to overcome the limitations of delivering oligonucleotide therapeutics to muscle tissue by leveraging transferrin receptor 1 (TfR1). TfR1-mediated delivery also has been shown by the field to facilitate uptake of therapeutics by the CNS. Many people living with rare muscle diseases experience CNS symptoms that contribute to the burden of disease, including cognitive deficits and dysregulated sleep, which affect individuals with DM1. Data being presented at ASGCT show that intravenous (IV) administration of FORCE conjugate, a TfR1-binding Fab antibody conjugated to an antisense oligonucleotide (ASO), achieved delivery to the CNS via TfR1 in both non-human primates (NHPs) and the innovative hTfR1/DMSXL mouse model. The hTfR1/DMSXL model, developed by Dyne, expresses the human TfR1 and carries a human DMPK gene with more than 1,000 CTG repeats that represents a severe DM1 phenotype. In these studies, FORCE conjugate was well tolerated. Highlights from the ASGCT data include: In NHPs, FORCE conjugate achieved superior delivery compared to unconjugated ASO when both were administered via IV. In addition, IV administration of...