Conference Day Two
November 21, 2024

8:45 am Check In, Morning Coffee & Light Breakfast


9:00 am Chair’s Opening Remarks

9:15 am Uncovering Mitochondrial & Related Pathways for Parkinson’s Disease & Dementia to Discover Biomarkers


  • Analyzing various methods to discover pharmacodynamic biomarkers for Parkinson’s disease and dementia
  • Identifying specific pathways that could outperform standard markers to overcome the transient nature of metabolic products
  • Leveraging the genetic linkage of Parkinson’s disease and dementia with mitochondrial dysfunction to potentially trace back to the root cause of pathogenesis

9:45 am Panel Discussion: Adopting an Integrated Approach to Measuring Mitochondrial Activity for Validating Biomarkers


  • Determining disease-specific biomarkers that can accurately reflect disease progression over time
  • Formulating a shared mechanism to identify biomarkers that can be reliably and non-invasively measured for application across disease indications
  • Interrogating organelle cross-talk to explore non-traditional avenues for biomarker validation

10:30 am Morning Refreshments & Networking

11:30 am Session Reserved for Agilent Technologies


11:40 am Pioneering Mito-1041: A Novel siRNA Approach to Modulate Mitochondrial Function for Tissue Selectivity & Specificity


  • Transforming siRNA as a state-of-the-art therapeutic for treating mitochondrial impairment through gene specificity and organ selectivity
  • Highlighting various methods of siRNA delivery to address distinguishing features of organs for restoration of mitochondrial function
  • Creating a roadmap to develop MITO-1041 and related compounds as a novel, safe and effective mitochondrial therapy for treating metabolic-associated liver disease (MASH), acute liver, kidney injury and heart disease

12:10 pm Roundtable Discussion: Developing Attractive Endpoints to Improve Clinical Trial Design for Disease Representation


  • Finding strategies to enhance enrolment that can tap into an existing patient pool at a specific stage of diagnosis
  • Exploring innovative trial designs that can provide statistical significance to address the heterogeneous nature of mitochondria
  • Creating eligibility for a trial to demonstrate the efficacy of drugs that can satisfy regulators and boost clinical development

1:00 pm Lunch & Networking

2:00 pm Linking the Convergence of Mito & Lysosomal Quality Control as Key Components of Age-Related Disease Progression


  • Diving into the interplay between mitochondrial and lysosomal quality control in the context of age-related diseases
  • Understanding how disruptions in cellular processes contribute to disease progression and potential therapeutic targets
  • Shedding light on C-Path Institute’s latest research on improving cellular health to combat age-related conditions


2:30 pm Patient Registries: Thinking Beyond the Data Collection for Expanding Therapeutic Avenues


  • Delving into Cure Mito’s global patient registry for Leigh syndrome to uncover what makes it unique
  • Assessing registry data for regulatory use and recognition
  • Evaluating the addition of a new registry: A meaningful contribution or a redundant effort? 

3:00 pm Fireside Chat: Building an Ecosystem for Stakeholders to Strengthen Patient Advocacy for Regulatory Success


  • Uniting patient advocates, regulators and health technology assessors to provide patients with predictive disease indications
  • Creating an alliance with regulators at the early stages of discovery to embrace the unique pathway to drug approval for mitochondrial diseases
  • Looking beyond the epidemiology to maximize the potential of drug developers and commercial success

3:45 pm Chair’s Closing Remarks

4:00 pm End of Day Conference