Amyl Therapeutics is Belgian preclinical biotech company developing a technology platform to bind various types of amyloid fibrils and induce their clearance, potentially offering a breakthrough approach to treating protein misfolding diseases.
Amyloidosis is a group of diseases characterized by an endogenous production of misfolded proteins that aggregate as insoluble amyloid fibrils and form deposits in various organs and tissues, resulting in organ dysfunction, serious morbidity and even death.
The company’s novel proprietary technology simultaneously targets multiple misfolded proteins implicated in both progressive peripheral diseases and neurodegenerative diseases, potentially creating a more robust response that could be suitable for patients at all stages of disease.
The therapies use a novel mechanism to bind to the amyloid fold of toxic protein aggregates of various types, including antibody light chain (AL) or transthyretin (TTR) aggregates, which accumulate in peripheral organs to cause systemic amyloidosis diseases, but also amyloid-β (Aβ), Tau, and α-synuclein aggregates, which accumulate in the brain to cause Alzheimer’s and Parkinson’s disease.
The company’s therapies are designed to prevent the further accumulation of aggregates and clear existing aggregates from the peripheral organs and the brain, while also blocking further cell-to-cell spread of misfolded proteins. Amyl Therapeutics believes these therapies offer a breakthrough approach to treating protein misfolding diseases.