Changing the Lives of Patients with

Myotubular and Centronuclear Myopathies (CNM)

Our Vision

Our vision is to become a global leader in rare inherited disorders by developing and commercializing therapeutics that transform the lives of patients living with life-threatening disorders. Our core strength in translational research and development allows us to quickly identify and evaluate new treatment approaches. We select diseases with high unmet need that correspond to patient populations with particular genetic mutations where we believe can be treated by altering the expression of a particular gene. We aim to match the target with what we believe is the most suitable technical modality to address the disease. For our lead program, DYN101, we identified increased dynamin 2 protein, or DNM2, which is toxic for muscle cells, as the principal disease-causing mechanism in CNM. We selected ASOs as a treatment modality based on their unique properties and our previous experience with this technology combined with our preclinical studies.

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Our vision

Our vision is to become a global leader in rare inherited disorders by developing and commercializing therapeutics that transform the lives of patients living with life-threatening disorders. Our core strength in translational research and development allows us to quickly identify and evaluate new treatment approaches. We select diseases with high unmet need that correspond to patient populations with particular genetic mutations wh0m we believe can be treated by altering the expression of a particular gene. We aim to match the treatment approach with what we believe is the best technical modality. For our lead program, DYN101, we identified increased dynamin 2 protein, or DNM2, which is toxic for muscle cells, as the principal disease-causing mechanism in CNM. We selected ASOs as a treatment modality based on their unique properties and our previous experience with this technology combined with our preclinical studies.

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Company history/timeline

Dynacure receives CTA approval to begin Phase 1 / 2 Study “UNITE-CNM” for DYN101. 2018 Dynacure secures €47 M in financing from key investors and builds out management and clinical teams. Dynacure starts co-developing its lead program, DYN101, a generation 2.5 antisense product candidate targeting dynamin 2 for the treatment of CNM, with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery. Dynacure was founded as a spin-off from the IGBMC (Institute of Genetic and Molecular and Cellular Biology – Unistra/INSERM/CNRS) of Strasbourg. 2017 2019 2016 Announces $55M Series C financing led by US healthcare investors 1st patient dosed with DYN101 in Phase 1 / 2 UNITE-CNM study 2020 (April) 2020 (March)

Dynacure starts co-developing its lead program, DYN101, a generation 2.5 antisense product candidate targeting dynamin 2 for the treatment of CNM, with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery. Dynacure was founded as a spin-off from the IGBMC (Institute of Genetic and Molecular and Cellular Biology – Unistra/INSERM/CNRS) of Strasbourg. 2017 2016 Dynacure receives CTA approval to begin Phase 1 / 2 Study “UNITE-CNM” for DYN101. Dynacure secures €47 M in financing from key investors and builds out management and clinical teams. 2019
2018
Announces $55M Series C financing led by US healthcare investors 1st patient dosed with DYN101 in Phase 1 / 2 UNITE-CNM study 2020 (April) 2020 (March)

Dynacure
Management

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Dynacure
Supervisory Board

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