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