8:00 am Check In, Morning Coffee & Light Breakfast
8:55 am Chair’s Opening Remarks
CAPITALIZING ON OUR UNDERSTANDING OF MITOCHONDRIAL BIOLOGY
9:00 am Deciphering the Mitochondrial Genome to Model Mitochondrial DNA Mutations
Synopsis
- Unraveling the intricacies of mitochondrial DNA and associated mutations to uncover disease mechanisms
- Presenting methods related to de novo mutagenesis in mitochondrial DNA to implement correction
- Characterizing molecular phenotypes in mitochondrial DNA-edited mouse models
9:30 am A Novel Take on Treatment: Shedding Light on Mitochondrial Transplantation for Neurological Diseases & Aging
Synopsis
- Exploring the role of mitochondrial transplantation in slowing and reversing neurological diseases and aging
- Targeting neurological diseases, aging, and rare disorders with mitochondrial transplantation
- Streamlining mitochondrial transplantation for clinical trials
10:00 am
Transforming Evaluation of Mitochondrial Function: the Seahorse XF Pro Analyzer
Virtual Presentation
Synopsis
• Mitochondrial dysfunction contributes to many common pathologies and can be targeted during drug discovery to reinstate a healthy phenotype
• The Seahorse XF Pro Analyzer provides label-free direct measurements of mitochondrial function and the flexibility for researchers to customize experiments to address specific mechanistic questions, validate therapeutic targets, identify lead compounds, characterize engineered cells, and mitigate potential toxicity risks
• The XF Pro Analyzer is equipped with advanced software, standardized workflows, and advanced data analytics to simplify the entire XF assay experience, from assay design to data QC and interpretation
10:10 am Morning Refreshments & Speed Networking
NARROWING THE PRECLINICAL & CLINICAL GAP TO TACKLE MITOCHONDRIAL DYSFUNCTION
11:10 am ALS, Secondary Progressive MS & Alzheimer’s Disease: Commonly Rooted in Mitochondrial Dysfunction in a 14-Day MP-101 Clinical CSF Biomarker Basket Study
Synopsis
- MP-101: Lowers damage while promoting repair
- MP-101: Mitochondrial-specific, biophysics MOA, non-genomic pharmacology cascading into cellular remodeling
- MP-101: Oral brain penetrating once-per-day treatment waking up compensatory mechanism Pro-Survival
11:40 am Micro-Dose MP-101 Preserves Motor & Muscle Function in ALS Mice With Potential Functional Recovery After Disease Onset
Synopsis
- Micro-dose MP-101 delays ALS disease onset and progression in hSOD1G93A mice
- ALS mice treated with MP-101 exhibit improved motor function, muscle innervation and muscle contractile function
- MP-101 treatment in hSOD1G93A from disease onset promotes recovery in mouse running ability
12:00 pm Mild Mitochondrial Uncoupling Attenuates Cerebral Mitochondrial Dysfunction After Traumatic Brain Injury
Synopsis
- Mild mitochondrial uncoupling with MP-201 mitigates oxidative stress in both contusive and concussive traumatic brain injury (TBI)
- Mitochondrial bioenergetic improvements are observed in a dose-dependent manner after TBI
- TBI-induced behavioral impairments are mitigated with MP-201 treatment
12:20 pm Understanding Mitochondrial Metabolism: More Than ATP
Synopsis
- Harnessing novel CEMS technology to measure mitochondrial metabolism
- Creating multi-pathway maps to decipher complex mitochondrial functions
12:30 pm Lunch & Networking
1:30 pm MP-201 Decreases Neuropathic Pain
Synopsis
- MP-201 attenuates neuropathic pain determined in a chronic trigeminal craniofacial pain model
- Molecular analysis of trigeminal sensory neurons from mice treated in vivo with MP-201 finds 98 unregulated and 33 downregulated genes
EXPEDITING THE DEVELOPMENT OF MITOCHONDRIAL THERAPEUTICS
1:50 pm Panel Discussion: Translating Modality Into Value for Patients to Establish Clearly Defined Clinical Objectives
Synopsis
- Building on the industry’s failures to re-evaluate how to select and measure the right clinical outcome
- Steering the direction of research to acquire robust data that can illustrate clinical translatability and expedite discovery towards Phase I of clinical trials
- Positioning patients at the heart of the modality to demonstrate its viability, thereby eliminating the need for a cost-risk analysis
2:35 pm Afternoon Refreshments & Networking
3:25 pm Modulating Novel Mitochondrial Targets to Treat Mitochondrial Disease, Diseases of Aging & Metabolism
Synopsis
- Unveiling small molecule activators of the mitochondrial DNA polymerase, POLG, to treat mitochondrial DNA depletion syndromes and beyond
- Exploring inhibitors of the mitochondrial RNA polymerase, POLRMT, to treat obesity and metabolic disorders
- Leveraging a unique mitochondrial biology discovery platform to develop a pipeline of novel therapeutics
3:55 pm Preserving Sight in Patients With Neurodegenerative Diseases Through a First-in-Class Target
Synopsis
- Highlighting the implications of mitochondrial dysfunction underlying the pathological basis for tissue damage in retinal diseases
- Building a library of mitochondrial protective compounds to design a new class of small molecules that can treat neurodegeneration through the protection of mitochondrial homeostasis