Advancing Lysosomal Ion Channel Programs from Early Discovery into Confident Lead Optimization

• Clarifying target rationale for lysosomal ion channels implicated in neurodegenerative disease to support early investment decisions
• Integrating electrophysiology, functional cell assays & chemistry iteration to progress intracellular channel hits toward development candidates
• Managing assay design & mechanistic uncertainty when optimizing compounds against non‑plasma membrane ion channels
• Achieving high brain penetrance while maintaining potency

Speakers:

Alexei Pushechnikov

Vice President MedChem

Mair Therapeutics