Arrakis Therapeutics Inc. has announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1).

Arrakis Therapeutics Inc. has presented data on its RNA-targeted small-molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1). The company’s proprietary RNA‐specific chemical, biological and structural methods and RNA-directed medicinal chemistry enabled structure-based small-molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology.