Modulating microRNAs.
Developing innovative medicines.

Discover our approach & focus

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