8:45 am Check In, Morning Coffee & Light Breakfast
ACCELERATING THE DISCOVERY OF MITOCHONDRIAL BIOMARKERS
9:00 am Chair’s Opening Remarks
9:15 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
9:45 am Panel Discussion: 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
10:30 am Morning Refreshments & Networking
11:30 am Session Reserved for Agilent Technologies
UNLOCKING NOVEL MECHANISMS OF MITOCHONDRIAL TARGETING
11:40 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
12:10 pm Roundtable Discussion: Developing Attractive Endpoints to Improve Clinical Trial Design for Disease Representation
Synopsis
- 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
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
2:30 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?
3:00 pm Fireside Chat: Building an Ecosystem for Stakeholders to Strengthen Patient Advocacy for Regulatory Success
Synopsis
- 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