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
Management

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Dynacure
Supervisory Board

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