Proven drug developers
and collaborators
We leverage our team’s core strength in translational research and development to identify what we believe to be the most suitable target and modality to address a particular rare disease, aiming to mitigate challenges faced by others in the development of therapeutics.
Myotubular and Centronuclear Myopathies (CNM) are no match
for us
We are initially focused on developing novel therapies to address myotubular and centronuclear myopathies, or CNM, a group of congenital myopathies characterized by severe muscle weakness, genetic heterogeneity, and defects in the structural organization of muscle fibers. The hallmark of CNM is a centrally located nucleus, or centronuclear, in muscle cells. In healthy muscle cells, the nucleus is usually located at the periphery of the cell.