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

     2019 Article: Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates

    2018 Article: Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1 

    2017 Article: Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

    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