Dyn101: a dynamin 2 modulator as a treatment of centronuclear myopathies (CNM)
Dynacure is developing DYN101, an investigational antisense medicine using Ionis Pharmaceuticals’ proprietary antisense technology. DYN101 is designed to modulate the expression of dynamin 2 (DNM2) for the treatment of CNM.
Preclinical studies have demonstrated that DYN101 has the potential to be disease modifying in CNM, with compelling preclinical efficacy in treating animal models of XLCNM and ADCNM. Prevention and reversion of the disease was observed with a clear dose-dependent improvement in whole body strength and mice survival.
The development plan for DYN101 was designed to be very broad and it is the only known program being investigated for most CNM populations, XLCNM, ADCNM and ARCNM. In addition to investigating DYN101 for CNM, Dynacure aims to expand its use and explores additional indications where the overexpression of DNM2 is a disease-driving factor.
In April 2019, the Clinical Trial Application for DYN101 was approved in the EU and Dynacure starts a Phase 1 / 2 study.