PROTEIN MISFOLDING DISEASES
The cause of many rare diseases is a protein malfunction due to a mutation in the wild-type sequence.
This mutation causes a decrease in the protein’s stability leading to misfolded protein, which is eventually degraded by the quality control system of the cell.
The absence of functional protein with the subsequent accumulation of toxic substrate is the primary cause of these diseases.
Small-molecule pharmacological-regulator therapy aids in protein folding and stabilization as well as lysosomal translocation, thus preventing further development of the disease.
Through its proprietary technological platform, Gain Therapeutics develops structurally targeted allosteric regulators that:
are non-competitive with the natural substrate,
have high specificity for the targeted enzyme,
are small molecules with improved drug-like properties (oral availability and BBB penetration),
and therefore, offer a wide therapeutic window.