Lysosomal Ion Channels as a New Therapeutic Class in Neurodegeneration: Advancing TRPML1 and TMEM175 Small-Molecule Agonists

  • Scientific and genetic rationale for targeting lysosomal ion channels in age-related neurodegenerative diseases (AD and PD)
  • Development considerations, challenges, and translational promises in advancing a highly brain-penetrant, Phase 1–ready TRPML1 small-molecule agonist
  • Emerging preclinical data of a development candidate TMEM175 agonist for Parkinson’s disease