Pre-Conference Workshops
November 19, 2024

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 | WORKSHOP B – APPLYING BIG DATA FOR MITOCHONDRIAL TARGETING

Harnessing Omics Technology for Biomarker Validation

Workshop Leader TBC

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

  • Dima Martini Lead- Global Early-Stage Medical Affairs, Mitochondrial and Rare Disease, Astellas Pharma

Synopsis

  • Analyzing the immensity of cost, time and investment required for mitochondrial drug development
  • Demonstrating the long-term value of mitochondrial therapeutics to regulators early on for expanded therapeutic opportunities
  • Creating incentives for mitochondrial disease programs to build a promising strategy for regulatory recognition

5:30 pm End of Pre-Conference Day