Dyne Therapeutics' Myotonic Dystrophy Gene Therapy Shows Encouraging Action In Animal Studies

  • Dyne Therapeutics Inc DYN has announced new preclinical data from its myotonic dystrophy type 1 (DM1) program, including results demonstrating sustained knockdown of toxic human nuclear DMPK RNA, the genetic basis of the disease.
  • Data were presented at the American Society of Gene & Cell Therapy Annual Meeting.
  • Data showed reductions in toxic human nuclear DMPK RNA with twice the duration and half the dose compared to the data reported in January of this year.
  • In January 2021, Dyne reported data showing that two doses (2 x 10 mg/kg) of its lead DM1 candidate resulted in significant toxic human nuclear DMPK knockdown at 14 days.
  • New data demonstrated an approximately 405 reduction in DMPK heart foci at 14 days.
  • Dyne expanded its analysis with a single, low 10 mg/kg dose. These new data show sustained DMPK knockdown at four weeks: 51% in the diaphragm, 46% in both the heart and tibialis anterior, and 42% in the gastrocnemius.
  • DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac, and smooth muscles. It is caused by an abnormal expansion in a region of the DMPK gene.
  • Price Action: DYN shares are trading 3.7% higher at $17.61 during the market session on the last check Friday.
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