Re-establishing Mitochondrial Homeostasis in Parkinson’s Disease: Advancing Parkin Gene Therapy to the Clinic
- Exploring the full discovery-to-IND pathway for Parkin gene therapy, showing how novel models and mitochondrial endpoints were established, providing a roadmap for advancing first-in-class neurodegenerative targets from concept to clinical readiness
- Demonstrating translational biomarker strategies that track mitochondrial recovery across patient-derived cells, rodent studies, and non-human primates
- Highlighting lessons from IND-enabling studies, including delivery, safety, and endpoint design, to inform trial design and de-risk early clinical development