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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Dynamin 101

            Therapeutic Area: Musculoskeletal Product Name: DYN101

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 30, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM).

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            Lead Product(s): DYN101

            Therapeutic Area: Musculoskeletal Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 05, 2020

            Details:

            This clinical trial, based on Ionis' technology platform, is the first investigational study testing an antisense medicine to treat Centronuclear Myopathies.