Table 1.

Examples of clinical trials for DM1 which are based on correction of mechanisms, causing DM1 pathology (see text for details). AOC is antibody-oligonucleotide conjugate.

Targeted Mechanism of DM1	Company	Phase I	Phase II	Outcome of Phase II	Phase III
Correction of RNA-binding protein CUGBP1 and degradation of the mutant RNA by small molecule GSK3 inhibitor tideglusib	AMO Pharma	Drug safety is known	Phase 2 completed	Reduction of CNS and muscle defects	Active
Correction of splicing of Insulin Receptor and other splicing events by metformin	Tor Vergata	Drug safety is known	Phase 2a completed	Mobility and gait improvement	Active
Correction of MBNL1 activity, reduction of CUG foci, reduction of myotonia by erythromycin	Osaka University Hospital	Drug safety is known	Active
Degradation of the mutant DMPK mRNA by AON	Ionis	Phase ½ completed	Phase ½ completed	Poor penetration into skeletal muscle
Degradation of the mutant DMPK mRNA by AOC	Avidis	Phase ½ in progress