Our Science and Approach
Neuropore Therapies discovers and develops novel small molecule orally bioavailable drugs for the treatment of neurodegenerative disorders. The agents being developed by Neuropore target fundamental underlying causes of these disorders, namely the accumulation of neurotoxic misfolded proteins and the associated neuroinflammation, and thus are expected to slow disease progression.
There is compelling evidence that the intracellular accumulation of specific proteins and their aggregation into toxic oligomeric conformations, as caused by dysfunction in the mechanisms responsible for removing these proteins, is an underlying cause of most neurodegenerative disorders. Thus, misfolded and aggregated forms of alpha-synuclein in Parkinson’s disease, amyloid beta and tau proteins in Alzheimer’s disease and huntingtin protein in Huntington disease are all associated with neurodegeneration, neuroinflammation and disease progression.
Images depicting the predominant neuropathology
associated with common neurodegenerative disorders
Neuropore Therapies is pursuing three distinct but inter-related therapeutic approaches to address the convergence of protein aggregation, failed protein clearance and neuroinflammation. The strong interrelationship between chronic inflammation, autophagy (the cellular mechanism for clearing protein aggregates) and neurodegeneration is depicted by a “triad hypothesis”. A key concept of the triad hypothesis is that interventions mechanistically focusing on inflammation, protein clearance and/or protein aggregation can lead to desired outcomes of reduced levels of neurotoxic proteins and slowed disease progression. The general approaches and mechanisms targeted by Neuropore are summarized below:
- Inhibit the formation of toxic oligomeric protein aggregates of misfolded proteins
- Inhibit brain inflammatory processes involved in disease pathogenesis
- Rectify defects in the cellular mechanisms for clearing aggregates of misfolded proteins
Triad hypothesis incorporates protein dysregulation, inflammation and
autophagy as key components leading to neurodegenerative diseases