Proteopathies are human diseases caused by protein misfolding, which prevents proteins from assuming their native three-dimensional structure. Protein misfolding compromises the ability of proteins to carry out important biochemical reactions and, when accumulated, misfolded proteins can cause cell death. Unfortunately, there are many diseases caused by protein misfolding such as Huntington’s Disease, Lou Gehrig’s Disease, Glycogen Storage Diseases, Parkinson’s and Alzheimer’s Disease.
Chaperone’s expertise in the development of drugs that activate the cellular protein folding machinery, coupled with our powerful drug discovery platform technology, addresses multiple proteopathies by correcting the protein misfolding defect.
Our therapeutic development pipeline is advancing chemically and mechanistically distinct candidates in protein misfolding disease indications: