TDP-43 targeting c-mod

TDP-43 targeting c-mod is a small molecule drug candidate developed to modulate biomolecular condensates formed by the TAR DNA-binding protein 43 (TDP-43). In amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, mislocalization and aggregation of TDP-43 in the cytoplasm are pathological hallmarks. These aggregates disrupt normal neuronal function. The "c-mod" class refers to "condensate-modifying drugs," which are designed to alter the physical properties or composition of disease-associated protein condensates. Dewpoint's TDP-43-targeting c-mods aim to reduce cytoplasmic inclusions of mislocalized TDP-43, restore its nuclear localization, improve neuron health, and rescue cellular functions impaired by toxic aggregates. This approach represents a novel therapeutic strategy for ALS and potentially other conditions involving aberrant phase separation or aggregation of RNA/DNA-binding proteins[2][3].

Other names

condensate-modifying drug targeting TDP-43Dewpoint c-mod for TDP-43