Commonly known as “Lou Gehrig’s Disease”, amyotrophic lateral sclerosis is a rare, progressive motor neuron disease. Sufferers of ALS experience ever-increasing muscle weakness, gradually losing the ability to walk, to speak, to eat… and eventually, to breathe. The vast majority of ALS patients die within 10 years of diagnosis – there is no cure.
Given that one of the hallmarks of ALS is aggregation of TAR DNA binding protein (TDP) within the central nervous system, it is a protein misfolding disease. Treventis is leveraging its CCM technology for discovery of drugs that broadly impact on TDP isoform misfolding, with the potential for disease-modifying effects in ALS as well as in other TDPopathies such as limbic-predominant age-related TDP-43 encephalopathy (LATE).