DYN101

DYN101 is an investigational antisense oligonucleotide therapy designed to reduce the expression of dynamin 2 (DNM2) protein by targeting its mRNA. It is a synthetically manufactured constrained ethyl gapmer antisense oligonucleotide (ASO), administered intravenously, and developed for the treatment of centronuclear myopathies (CNM), including both X-linked and autosomal dominant forms. The drug works by inhibiting the production of dynamin 2 protein, which is implicated in CNM pathogenesis. Preclinical studies have shown that reducing DNM2 mRNA can prevent or reverse disease symptoms in animal models. Originally developed by Dynacure in collaboration with Ionis Pharmaceuticals, development was later continued by Flamingo Therapeutics following a merger[1][2][4][7].

Other names

5’-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-spdA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3’