8:00 am Check In, Morning Coffee & Light Breakfast
8:45 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 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
10:00 am Morning Refreshments & Speed Networking
NARROWING THE PRECLINICAL & CLINICAL GAP TO TACKLE MITOCHONDRIAL DYSFUNCTION
11:00 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
11:30 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
11:50 am 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
12:10 pm Lunch & Networking
1:10 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
1:30 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
2:15 pm Afternoon Refreshments & Networking
2:45 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
PROTECTING THE POWERHOUSE OF THE CELL
3:15 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