Lysoway Therapeutics has developed unique technological approaches to screen and develop potent modulators of lysosomal ion channels, including TRPML1 and TMEM175. These small-molecule compounds can restore autophagic flux and resolution, as well as enhance plasma membrane repair through direct activation of lysosomal exocytosis. These modulators have great potential to treat neurodegenerative diseases with genetic defects in lysosomal function or rare diseases characterized by toxic accumulation of cellular wastes.
Restoring lysosomal function via ion channel modulation
Normal functions of critical lysosomal ion channels are often disrupted due to either genetic defect or inhibition by accumulated cellular waste material, which in turn results in lysosomal functional deficiency, a trademark observation in a number of neurodegenerative diseases and metabolic disorders. Activation or inhibition of ion channels via small molecule agents can help restore lysosomal function, which can then better regulate critical downstream processes (including autophagic flux and resolution, exocytosis, plasma membrane repair, and lysosomal biogenesis) to provide therapeutic benefit to patients.
Our Pipeline: Synergistic Programs To Address Multiple Distinct Lysosomal/Autophagy Deficiencies
Our management team has tremendous relevant experience and expertise in lysosomal biology, drug discovery and development.