Dyn101: a dynamin 2 modulator as a treatment of centronuclear myopathies (CNM)
Dynacure’s lead program, Dyn101, is modulating the expression of dynamin 2 for the treatment of centronuclear myopathies.
Dyn101 is an antisense oligonucleotide (ASO) targeting the mRNA of dynamin 2 and was developed in collaboration with IONIS pharmaceuticals (Carlsbad USA).
ASO technology represents a promising therapeutic approach for neuromuscular diseases caused by a gain-of-function mechanism (Arechavala-Gomeza et al. 2012; Wheeler et al. 2012). It has been demonstrated that Dyn101 downregulates the expression of dynamin 2, which leads to the prevention and reversion of the disease in a relevant mouse model.