Dyne Therapeutics sharpens case for two muscle disease therapies with updated data 

Dyne Ther­a­peu­tics shared promis­ing clin­i­cal tri­al up­dates from two of its ex­per­i­men­tal RNA med­i­cines for my­oton­ic dy­s­tro­phy type 1 (DM1) and Duchenne mus­cu­lar dy­s­tro­phy.

The up­date, an­nounced Mon­day, in­cludes a first look at six pa­tients in a Phase 1/2 study who re­ceived a high dose of Dyne’s my­oton­ic dy­s­tro­phy ther­a­py called DYNE-101. It showed a dose-de­pen­dent in­crease in splic­ing cor­rec­tion — mea­sured on a pan­el of 22 genes — com­pared to ear­li­er co­horts that re­ceived low­er dos­es of the ther­a­py. Re­searchers and an­a­lysts look for dose de­pen­den­cy be­cause that’s a sign that a treat­ment has an ef­fect.

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