Arrakis Therapeutics Presents Data Highlighting Progress of Oral RNA-targeted Small Molecule to Treat Myotonic Dystrophy Type 1 (DM1)
Preclinical in vivo data demonstrate hyper-precise mechanism directly targets the disease-causing mRNA repeats leading to correction of splicing defects and reversal of myotonia Oral administration of lead drug candidate results in broad biodistribution, including to the muscle and heart Company plans to file an IND with U.S. FDA in 2026 for an oral drug candidate to […]