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.