8:00 am Check In, Morning Coffee & Light Breakfast
8:55 am Chair’s Opening Remarks
ACCELERATING THE DISCOVERY OF MITOCHONDRIAL BIOMARKERS
9:00 am Uncovering Mitochondrial & Related Pathways for Parkinson’s Disease & Dementia to Discover Biomarkers
Synopsis
- 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
9:30 am Fireside Chat: Adopting an Integrated Approach to Measuring Mitochondrial Activity for Validating Biomarkers
Synopsis
- 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
10:15 am Morning Refreshments & Networking
UNLOCKING NOVEL MECHANISMS OF MITOCHONDRIAL TARGETING
11:00 am Pioneering Mito-1041: A Novel siRNA Approach to Modulate Mitochondrial Function for Tissue Selectivity & Specificity
Synopsis
- 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
11:30 am Cracking the Code With Mitochondrial Transplant: Reversing the Age of the Immune System
Synopsis
• Unpicking cellular energetics to understand disease indications and drug translation in older patients
• Examining how transplanted mitochondria enhance cellular function and survival for evolutionary advantages
• Discovering Mitrix Bio’s approach to demonstrating age-reversal effects in the immune system of older mice to highlight the potential benefits for elderly human patients
12:00 pm Lunch & Networking
1:00 pm Linking the Convergence of Mito & Lysosomal Quality Control as Key Components of Age-Related Disease Progression
Synopsis
- 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
TRANSFORMING OUR APPROACH TO MITOCHONDRIAL TREATMENT
1:30 pm Patient Registries: Thinking Beyond the Data Collection for Expanding Therapeutic Avenues
Synopsis
- 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?