Clinical trials are expected to start next year with an antisense clinical candidate for the rare disease, centronuclear myopathy, developed by French start-up Dynacure as part of its collaboration with US antisense specialist Ionis Pharmaceuticals.
Strasbourg, France-based Dynacure may only have been set up two years ago, but in some ways it is a "mature start up", and has been able to raise a hefty €47m ($55m) from VCs and institutional investors in its first substantial financing round to support the move of its lead product candidate, Dyn101, into the clinic for the rare and often fatal muscle disease, centronuclear myopathy (CNM).
Stephane van Rooijen, CEO of Dynacure
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A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to bring about a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the key players that have been part of pricing discussions.
