FUS small molecule

Transition Bio's FUS program is a preclinical drug discovery initiative focused on developing small molecules that target the **Fused in Sarcoma (FUS)** protein for the treatment of **amyotrophic lateral sclerosis (ALS)** and **frontotemporal dementia (FTD)**. FUS is an RNA-binding protein that is known to undergo pathological phase separation and aggregation in several neurodegenerative diseases. Transition Bio utilizes its proprietary **Condensomics™** platform, which integrates droplet microfluidics and machine learning, to identify compounds capable of modulating the phase behavior of intrinsically disordered proteins like FUS. The goal of the program is to prevent or reverse the formation of toxic FUS aggregates, thereby addressing the underlying pathology of ALS and FTD.

Other names

FUS programFUS targeting program