WALTHAM, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that new preclinical data from its myotonic dystrophy type 1 (DM1) program will be featured in presentations during the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, to be held virtually May 11-14, 2021. ASGCT abstracts are available on the meeting website, and Dyne’s presentations are noted below.
Presentation: Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
Scientific Symposium: Hot Topics and Remaining Challenges in RNAi and Oligonucleotide Therapy for 2021
Date/Time: Friday, May 14, 2021 at 10:26 a.m. ET
Presenter: Romesh Subramanian, Ph.D., Chief Scientific Officer
Oral Presentation: The FORCETM 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 (Abstract #247)
Session: Oligonucleotide Therapeutics
Date/Time: Friday, May 14, 2021 at 1:15 p.m. ET
Presenter: Stefano Zanotti, Ph.D., Director, Mechanistic Biology
The presentations will include new data, expanding upon initial findings reported in January 2021 utilizing Dyne’s innovative hTfR1/DMSXL mouse model expressing human TfR1 and carrying a human DMPK gene that represents a severe DM1 phenotype with more than 1,000 CTG repeats, as well as results from in vitro studies.
Dyne’s FORCE™ platform leverages the importance of transferrin 1 receptor, TfR1, in muscle biology as the foundation for its novel approach. TfR1, which is highly expressed on the surface of muscle cells, is required for iron transport into muscle cells. Dyne links therapeutic payloads to its TfR1-binding fragment antibody (Fab) to develop targeted therapeutics for muscle diseases. Dyne’s lead DM1 candidate consists of a Fab conjugated to an antisense oligonucleotide (ASO) to enable targeted delivery to muscle tissue to reduce accumulation of toxic DMPK RNA in the nucleus, release splicing proteins, allow normal mRNA processing and translation of normal proteins, and potentially stop or reverse disease progression.
DM1 Program Webcast
Following the presentations on May 14, 2021, Dyne plans to host a live webcast event at 4:00 p.m. ET to review the company’s DM1 program and preclinical data, and to provide an overview of the disease and treatment challenges. Joining management on the webcast will be Charles Thornton, M.D., the Saunders Distinguished Professor of Neuromuscular Research at the University of Rochester. Dr. Thornton directs the Muscular Dystrophy Cooperative Research Center in Rochester and the Myotonic Dystrophy Clinical Research Network, a multicenter consortium for clinical research and therapeutic trials. He has been engaged in bench and clinical research on myotonic dystrophy for 30 years.
The live and archived webcast can be accessed in the Investors & Media section of Dyne’s website at: https://investors.dyne-tx.com/news-and-events/events-and-presentations. The archived webcast will be made available shortly after the event and accessible for 90 days.
About Dyne Therapeutics
Dyne Therapeutics is building a leading muscle disease company dedicated to advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue seen with other approaches. Dyne’s broad portfolio of therapeutic candidates for serious muscle diseases includes programs for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com/, and follow us on Twitter, LinkedIn and Facebook.
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