Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy
Preclinical data presented at Cell Symposia conference show a small molecule directed at a pathogenic RNA achieved reversal of myotonia in an animal model of myotonic dystrophy Type 1 (DM1)
Supports additional product development with Company’s multi-modality rSM drug platform, broadening the reach of genetic medicine and offering the potential to treat diseases at their root cause with oral medicines
