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Protein misfolding diseases

A highly complex and to-date unsolvable challenge.

  • What are misfolded proteins?

    Our 100,000 different types of proteins must fold into unique structures to function….. but sometimes this goes wrong, generating "misfolded" proteins, which then clump together into toxic aggregates. Protein aggregation can be driven by ageing, environmental stress and lifestyle, genetic mutations, and medical interventions.

  • Manifestations across organs

    Misfolding and aggregation of proteins is central to pathological conditions ranging from Alzheimer's and Parkinson's diseases in the brain, Type-2 diabetes where deposits form in the pancreas, to systemic conditions, in which deposits can be found in multiple organs, including the liver and the heart. Overall, more than 50 human protein misfolding diseases have been described to date.

  • Significant unmet need with few disease modifying therapies

    Protein misfolding diseases are arguably the most critical global healthcare challenges of the 21st century. Unfortunately, current strategies - in particular those driven by traditional drug discovery and biological approaches - have proven, at least to date, to be ineffective for a large number of these disorders despite several decades of intensive academic and industry research efforts.

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