8AM - 5PM EST | 2PM - 11PM CEST

8:00 am Registration, Morning Coffee & Structured Networking

8:45 am Chair’s Opening Remarks

Overview of the Mitochondrial Medicine Landscape

9:00 am Bench to Bedside – Targeting Mitochondria for Aging Skeletal Muscle

  • David Marcinek Professor of Radiology, Adjunct of Bioengineering and Laboratory Medicine and Pathology, University of Washington Medical School

Synopsis

  • Discussing the role of mitochondria in chronic disease and age-related dysfunction with an emphasis on aging skeletal muscle
  • Presenting new developments on mechanisms of mitochondrial aging
  • Examining successes and challenges translating mitochondrial targeted interventions to human skeletal muscle aging

9:30 am Spotlight Session– BACH1 Inhibitor as Therapeutics for Sickle Cell Disease

Synopsis

  • Developing therapeutics modulating mitochondrial function to address various diseases
  • Developing a BACH1 inhibitor that can regulate antioxidant response genes through Nrf2 pathway
  • Reviewing the preclinical data supporting the therapeutic efficacy of BACH1 inhibitor to treat sickle cell disease

The Role of Ex Vivo Mitochondrial Transfer to Improve Stem Cell Therapy Outcomes (Mitochondrial Transfusion)

10:00 am Mitochondrial Augmentation Therapy (MAT): First Steps in Elucidating Mechanism of Action

Synopsis

  • Evaluating the systemic effect of MAT on different organs
  • Examining the effect of MAT on hematopoietic and immune system
  • Connecting pre-clinical and clinical data to explain mechanism of action of MAT

10:30 am Morning Refreshments & Structured Networking Break

11:00 am Therapeutic Mitochondria Transplantation – A New Category of Medicines?

Synopsis

  • Exploring how viable mitochondria are transplanted into cells to induce the therapeutic effect and how this approach has shown great promise in ischemiareperfusion injury
  • Examining the latest insights to support the notion of augmentation and replacement, to affect the cell energy metabolism
    (Therapeutic Mitochondria Transplantation) as a treatment modality with broad applications
  • Highlighting select challenges the field faces

11:30 am Mitochondrial Bioreactors & Transplant Therapy for Neurodegeneration & Age-Related Diseases

  • Ben Albensi Scientific Advisor, Mitrix Bio, Nova Southeastern University

Synopsis

  • Why is targeting the mitochondria important in disease and injury?
  • What is mitochondrial transplantation/transfusion?
  • Exploring new sources of mitochondria for larger-scale therapy

The Relationship of Mitochondrial DNA (mtDNA) Mutations/Damage & Mitochondrial Reactive Oxygen Species (ROS) Dysfunction

12:00 pm Activating Native Thermogenic Pathways to Treat Metabolic Disorders

  • Jonah Sinick Chief Executive Officer, Equator Therapeutics

Synopsis

  • Reviewing the evidence linking mitochondrial thermogenesis with the reversal of metabolic diseases
  • Examining the physiological mechanisms underlying thermogenic H+ leak through the inner mitochondrial membrane
  • Presenting data on novel activators of these mechanisms

12:30 pm Lunch & Structured Networking

1:30 pm Fumarates for Rescue of Mitochondrial Functions

Synopsis

  • Demonstrating how MMF supports increased mitochondrial function through a novel MoA
  • Examining how Ixchel has identified a novel MMF that has improved PK over existing fumarates
  • Exploring how this molecule rescues mitochondrial function and longevity in multiple animal models of mitochondrial disease

2:00 pm Development of a New Medication for the Ultra-Rare Mitochondrial Disease Thymidine Kinase 2 Deficiency Disorder (TK2d)

  • David Schaaf Vice President, US Medical Affairs, Senior Medical Officer, Zogenix

Synopsis

  • Reviewing TK2d disease whilst navigating natural history
  • Evaluating the creation of NDA-supporting dataset
  • Examining top-line results supporting NDA-submission

Current Progress of Mitochondrial Quality Control (MQC) Signaling Pathways Mitochondrial Biogenesis, Mitochondrial Dynamics & Mitochondrial Autophagy (Mitophagy)

2:30 pm Mitochondrial Uncoupling for Therapeutic Development in Metabolic Diseases and Cancer

Synopsis

  • Investigating how mitochondrial function deterioration is an important causal factor for several diseases, including NASH, type 2 diabetes, neurodegenerative diseases
  • Exploring mitochondria-related metabolic changes (e.g. the Warburg Effect) and how it’s essential for the transformation of normal cells to cancer cells
  • Examining how mitochondrial uncoupling provides a unique opportunity for effective treatment of these major diseases through eliminating the corresponding root causes
  • Presenting strategies, approaches and examples in targeting mitochondrial uncoupling for therapeutic development

3:00 pm Afternoon Refreshments & Structured Networking Break

3:30 pm Small Molecule Activator of PINK1 in Preclinical Models of Parkinson’s Disease & Mitochondrial Disease

Synopsis

  • Examining the discovery and optimization of small molecule activator of PINK1, MTK458
  • Exploring how MTK458 reduces mitochondrial damage signature in multiple orthogonal models of mitochondrial disease and Parkinson’s disease
  • Demonstrating pathological and functional rescue in pre-clinical alpha synuclein PFF model of Parkinson’s disease

4:00 pm Rejuvenating Cells via Mitophagy to Address Unmet Medical Need

Synopsis

  • Examining the benefits of mitophagy induction via Urolithin A on mitochondrial function were demonstrated across a broad range of models
  • A closer look at Vandria, a spin-off of Amazentis dedicated to the development of novel pharmaceuticals, possesses a growing pipeline of next generation small molecule mitophagy inducers
  • Evaluating results and target pharmaceutical applications of new chemical entities

4:30 pm Panel Discussion: Evaluate the Current Challenges in Targeting-Mitochondria for Chronic Mitochondrial Diseases

  • Gregory Flesher President & Chief Executive Officer, Reneo Pharma
  • Keshav Singh Joy & Bill Harbert Endowed Chair & Professor/Scientific Founder & Chief Scientific Officer, University of Alabama, Birmingham/Yuva Biosciences

Synopsis

  • How can drug developers/research cut to the real barriers?
  • Debate what is truly required to break through to successful treatments for mitochondrial diseases
  • Understand how to make good disease models in which one can test potential therapeutic pathways

5:00 pm Chair’s Opening Remarks

5:15 pm End of Day 1