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Dyne Therapeutics Presents New In Vivo Data from DYNE-101 at ASGCT Annual Meeting Demonstrating Low Monthly Dosing Leads to Robust DMPK RNA Knockdown
- Initiation of Dosing in Multiple Ascending Dose Clinical Trial for DYNE-101 in Patients with Myotonic Dystrophy Type 1 Anticipated in Mid-2022 - WALTHAM,
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Initiation of Dosing in Multiple Ascending Dose Clinical Trial for DYNE-101 in Patients with Myotonic Dystrophy Type 1 Anticipated in Mid-2022 - WALTHAM, Mass., May 16, 2022 (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, is presenting new in vivo data from DYNE-101, its myotonic dystrophy type 1 (DM1) candidate, at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting today demonstrating robust knockdown of DMPK RNA in multiple muscles with low monthly dosing. “We are excited to present these new data that show monthly dosing of DYNE-101 led to a substantial knockdown of toxic human nuclear DMPK RNA in cardiac and skeletal muscle in the hTfR1/DMSXL mouse model as well as reduction of wild-type DMPK RNA in muscles of non-human primates. Additionally, GLP toxicology data support a favorable safety profile for our DM1 candidate,” said Oxana Beskrovnaya, Ph.D., Dyne’s chief scientific officer. “These findings combined with previous data demonstrating correction of splicing, support the potential for DYNE-101 to be a disease-modifying therapy with low, infrequent dosing. We look forward to evaluating DYNE-101 in our planned global multiple ascending dose clinical trial in patients with DM1.” DM1 is a rare, progressive, genetic disease caused by an abnormal expansion in the number of CTG triplet repeats in a region of the DMPK gene that causes toxic RNA to cluster in the nucleus, forming foci and altering the splicing of multiple proteins essential for normal cellular function. As a result of this altered splicing, people living with DM1 typically experience progressive weakness of skeletal, cardiac and smooth muscle. There are no approved disease-modifying therapies for DM1. DYNE-101 is designed to enable targeted delivery to muscle tissue and address the genetic basis of the disease. The new data being presented during ASGCT evaluated monthly repeat doses of DYNE-101 in an innovative hTfR1/DMSXL mouse model developed by Dyne and in non-human primates (NHPs). The hTfR1/DMSXL model expresses the human transferrin 1 receptor (TfR1) and carries a human DMPK gene with more than 1,000 CTG repeats that represents a severe DM1 phenotype. In the hTfR1/DMSXL model, DYNE-101...