Neurodegenerative diseases

As our understanding of the pathogenesis of neurodegenerative disease deepens, it has become possible to identify specific genetic sequences whose active downregulation using gene therapy has the potential to treat diseases for which no treatments exist. Direct administration into the spinal canal (Intrathecal administration) of MNM conjugated to siRNA provides a mode of direct administration via which our revolutionary delivery technology can be brought to bear on these inherited neurological diseases. Interna is working in collaboration with its partners on tailoring the MNM platform to a range of severe and untreated heritable diseases of the nervous system.