TMEM175 program (WCNDD)

The TMEM175 program at the Warren Center for Neuroscience Drug Discovery (WCNDD) is focused on the development of small-molecule modulators of the TMEM175 protein for the treatment of Parkinson's Disease (PD) and Lewy Body Dementia (LBD). TMEM175 is a potassium-selective channel located in the lysosomal membrane that is essential for maintaining lysosomal pH and ion homeostasis. Genetic studies have identified TMEM175 variants as significant risk factors for PD, with loss-of-function mutations leading to lysosomal dysfunction and the accumulation of alpha-synuclein aggregates. This program, supported by the Wellcome Trust, aims to identify and optimize small molecules that can restore or enhance TMEM175 function to improve lysosomal health and provide neuroprotection. It is currently in the lead optimization stage of preclinical development.

Other names

TMEM175 small molecule programTMEM-175 small molecule programTMEM 175 small molecule programTMEM175 modulatorTMEM-175 modulatorTMEM 175 modulator