10:00 AM | WORKSHOP A – ENSURING PRECLINICAL SUCCESS FOR MITOCHONDRIAL DISEASES
Enhancing Preclinical Models for Seamless Translatability
Synopsis
- Addressing limitations with current preclinical models for mitochondrial dysfunction to clear the roadblock to clinical translation
- Unpicking must-have attributes of a preclinical model that can support the transition from an in vitro to an animal system
- Exploring alternative models that can be used to capture the safety and efficacy of molecules and mechanisms beyond the lab setting
12:00 pm LUNCH BREAK
1:00 PM | HARNESSING MITOCHONDRIA FOR THERAPEUTIC INNOVATION
Unraveling Mitochondrial Biology: Translating Science to Therapy
Synopsis
- Unlocking bioinformatics tools to screen and identify relevant targets with the desired functions on mitochondrial activity
- Applying multi-omics to understand the multifactorial nature of mitochondrial diseases and expand avenues for biomarker development
- Leveraging big data to uncover protein-protein interactions that can lead to pharmacodynamically attractive targets
3:00 pm AFTERNOON BREAK
3:30 PM | WORKSHOP C – REDEFINING STRATEGIES FOR MITOCHONDRIAL THERAPEUTICS
Evaluating Decision-Making Behind Therapeutic Development
Synopsis
- Analyzing the immensity of cost, time and investment required for mitochondrial drug development
- Demonstrating the long-term value of mitochondrial therapeutics to patients, investors, and regulators early on for expanded therapeutic opportunities
- Creating incentives for mitochondrial disease programs to build a promising strategy for regulatory recognition