Ionis is the leading pharmaceutical company developing ground breaking‘antisense’ drugs – such as the recently approved treatment for spinal muscular atrophy – SPIRANZA . Togetherwith Dynacure, Ionis have identified an antisense drug target that has the potential to work for centronuclear myopathies, by ‘turning down’ or ‘antisense-ing’ the protein dynamin 2.  Dynacure’s collaboration with the laboratory of Jocelyn Laporte at IGBMC has already demonstrated that targeting dynamin 2 can potentially treat a number of genetic forms of myotubular myopathy including BIN1, DNM2 and MTM1
“We are delighted with the productivity and efficiency of our collaboration with Ionis. The identification of a development candidate is an important step for our alliance with Ionis and the Dynacure team. Importantly, this development puts us one step closer to potentially finding a new treatment for patients suffering with CNM, a devastating disease with no adequate treatments”, Stephane van Rooijen, chief executive officer of Dynacure.