Transition Bio TDP-43 program

Transition Bio is developing a small molecule program targeting the TDP-43 protein for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Developed in collaboration with Voyager Therapeutics, the program utilizes Transition Bio's proprietary platform which integrates droplet microfluidics and machine learning to discover compounds that modulate biomolecular condensates. The therapeutic goal is to correct the pathological mislocalization and aggregation of TDP-43—a hallmark present in over 90% of ALS cases—without disrupting the protein's essential cellular functions. The program is currently in the preclinical discovery and lead optimization phase, with Transition Bio leading early development until the nomination of a development candidate.

Other names

TDP-43 targeting programTDP43 targeting programTDP 43 targeting programTransition Bio-Voyager TDP-43 small molecule program