8:00 am REGISTRATION
8:25 am Chair’s Opening Remarks
8:30 am Discovering the Astellas Approach: How to Turn Innovative Science into Value for Patients Living with Mitochondrial Diseases
Synopsis
- Astellas takes on a long-term strategic view focusing on diseases with high unmet need
- Leveraging internal and external partnerships to deliver value for patients
MEASURING MITOCHONDRIAL DYSFUNCTION
9:00 am Targeting Mitochondrial Dysfunction in Neurodegenerative Diseases
Synopsis
- Identifying nexus points in mitochondrial function that impact neurodegeneration
- Developing new therapeutic approaches to target neurodegenerative diseases with selective small molecules that modulate key aspects of mitochondrial physiology
- Establishing translational approaches to objectively measure impacts on mitochondrial function, physiology and clinical outcomes
9:30 am Rapid, Accurate Detection of Mitochondrial Dysfunction Using the Agilent Seahorse XF Pro
9:40 am MORNING BREAK & NETWORKING
TREATING THE UNTREATABLE PREVALENT DISEASES BY MANIPULATING MITOCHONDRIA
10:00 am Modulating Mitochondrial Function in Patients at Risk for Post-Operative Complications following Cardiac Surgery
Synopsis
- Exploring the clinical benefits of modulating mitochondrial function in non-rare disease conditions
- Modulation of targeted gene expression in patients
10:30 am Targeting Mitochondrial Quality Control as a Therapeutic Strategy for Cancer
Synopsis
- Uncovering novel modulators of mitochondrial protein quality control as therapy for relapsed/refractory diffuse large B-cell lymphomas
- Explore the key challenges in developing agents directed against mitochondria for cancer therapeutics
- Discover novel clinical biomarkers related to mitochondrial biology
11:00 am Treating Chronic Diseases Associated with Mitochondrial Pathophysiology with a Novel siRNA Approach
Synopsis
- Demonstrated the role of MCJ, a key mitochondrial regulator, in NASH, related liver diseases, kidney and cardiovascular disease
- Developed a liver-targeted siRNA as a therapeutic approach to treating NASH; efficacy demonstrated in multiple preclinical disease models
- Conducting safety pharmacology and toxicity studies to support an IND filing with no adverse findings thus far
11:30 am Novel Therapeutic Strategies for Systemic Improvement of Mitochondrial Function
Synopsis
- Examining a multi-ingredient supplement to improve mitochondrial function in obesity and fatty liver disease
- Verifying nutraceutical therapy for primary mitochondrial disease
12:00 pm LUNCH & NETWORKING BREAK
CENTRING CLINICAL CARE FOR OUTSTANDING CLINICAL OUTCOMES IN RARE DISEASES
1:00 pm Using Natural History Studies for Rare Mitochondrial Diseases to Bolster Study Design
Synopsis
- Natural history studies are vital to all drug development projects but are especially difficult to obtain for inherited mitochondrial diseases due to their severity and rarity
- Discussing the power of natural history diseases to understand disease prevalence study disease progression and improve the efficiency of drug development
- Utilising natural history studies in conjunction to clinical trials due to the lack of alternate therapies as control treatment in rare mitochondrial diseases
1:30 pm Roundtable Discussion: Masterminding Mitochondrial Drug Development for Multiple Disease Indications
Synopsis
Join tables led by mitochondrial drug development experts to discuss the benefits and challenges of different mitochondrial modulation strategies for rare and prevalent disorders.
- Debating methods of modulation to inform early mitochondrial drug development decisions
- Balance the distinct safety and clinical considerations of rare and prevalent diseases in relation to modality for regulatory success
2:00 pm Feedback Panel
Synopsis
- Nominated speakers from each table will unite to share the alternate perspectives and discussions from each table
- Highlight the future of mitochondrial drug development across therapeutic areas
2:30 pm AFTERNOON BREAK & NETWORKING
2:50 pm Improving Patient Recruitment for Rare Mitochondrial Disease Clinical Trials to Rigorously Demonstrate Drug Efficacy
Synopsis
- Establishing the value of clinical care within clinical trials to maintain trust and improve recruitment for clinical trials in mitochondrial disease drug development
- Incorporating patient voice into decision making for mitochondria-disease specific endpoints
- Focusing on quality of life, patient experience, and pain management throughout mitochondrial therapy development, advancing clinical readiness for mitochondrial disease
3:20 pm A Case Study of Patient Engagement
Synopsis
- Understanding how patient advocacy groups and drug developers can work together throughout a drug development pipeline
- Centring patient experience to develop therapies that improve quality of life
- Utilizing digital technologies when conducting clinical trials
3:50 pm Connecting the dots: Understanding Patient Empowerment, Inclusion, and the Landscape of Patient-Developed Registries
Synopsis
- Leigh syndrome patient registry developed by the Cure Mito foundation – a comprehensive look
- Patient empowerment and inclusion – what do we really mean by that?
- Examining barriers and potential of patient-driven registries