Our unique approach
All these diseases have been associated with the misfolding and aggregation of specific proteins or peptides to form oligomeres and insoluble 'amyloid fibers'. For many years the biopharma industry has focused on developing antibodies against these fibres and oligomeres However, this approach has completely failed as the insoluble fibers are not pathogenic.
Recent research has revealed that these disease-associated peptides also aggregate to form 'amyloid pores' within membranes of cells and organelles. These pores lead to dysfunction of cells and ultimately trigger cell death and degeneration .
Based on this emerging science, Amporin Pharmaceuticals was established to develop a new class of small molecule drugs that specifically target these amyloid pores, as potential disease-modifying treatments for all protein misfolding diseases.
While the biopharma industry has failed by targeting insoluble amyloid fibers, we specifically target amyloid pores because these are now known to cause dell death and degeneration by puncturing holes in cell membranes.
Our solution
We are developing three new classes of orally available, brain-penetrating drugs that specifically target amyloid pores within cell membranes:
- Amporbans – our First-in-Class amyloid pore-blocking agents effectively block amyloid pores like a plug, so that they are no longer permeable to calcium and other ions.
- Amporins – our First-in-Class amyloid pore inhibitors specifically inhibit and reverse the formation of amyloid pores within cell membranes, effectively dissolving them from the membrane like target-specific detergents.
- Amportacs – our First-in-Class amyloid pore-targeting chimeras actively promote the targeted ubiquitination and degradation of amyloid pores by the proteasome within the cell.
We are now building a strong and steady pipeline of drug candidates, which have the potential to become the first and most effective disease-modifying treatments for all protein misfolding diseases.