Ionis Pharmaceuticals / Biogen
Approach: Antisense Oligonucleotide
Status: Preclinical. Predecessor compound, ISIS-DMPKRx completed a Phase 1 study but proved not potent enough. Ionis is working on muscle-targeting ligands to increase potency.
Presentation at 2018 MDF Conference
2012 Press Release Announcing Biogen-Isis (now Ionis) deal
Article on Phase 1 results of predecessor compound
September 14, 2019 Presentation at Myotonic conference (starts at 27:00)
April 2, 2020 Article: Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3′UTR RNA