ACE-2223

ACE-2223 is a first-in-class, orally bioavailable small molecule drug developed by Acelot. It is designed to specifically target and disrupt gain-of-function, misfolded forms of the protein TDP-43 (TAR DNA-binding protein 43), which are implicated in the pathology of several neurodegenerative diseases. In amyotrophic lateral sclerosis (ALS), TDP-43 accumulates as toxic aggregates in the cytoplasm, leading to loss of its normal nuclear function and subsequent neuronal death. ACE-2223 acts by disrupting these misfolded conformations and restoring functional TDP-43, thereby addressing a key driver of disease progression. The drug demonstrates excellent brain penetrance and favorable ADME properties. Its primary indication is ALS, but it also targets misfolded TDP-43 found in frontotemporal dementia (FTD) and Alzheimer’s disease[1][2][3].

Other names

Acelot-TDP-43Acelot-TDP43Acelot-TDP 43