Our lead investigational program is an anti-miR for Myotonic Dystrophy
New Target
Upregulated miRNA in tissues of patients with myotonic dystrophy
First-in-class Mechanism
of Action
Impact on the root cause of the disease
(toxic DMPK and insufficient MBNL)
Improved Delivery
to Muscle Tissue
Oligonucleotide conjugated to a fatty acid allowing for very low active dose
