9AM - 5PM EST | 3PM - 11PM CEST

8:00 am Online Registration & Virtual Coffee Networking

8:45 am Chair’s Opening Remarks

  • Philip Yeske Science & Alliance Officer, United Mitochondrial Disease Foundation

Overview of the Landscape:
The Role of Mitochondrial Dysfunction in Age-related Diseases & Rare Genetic Diseases

9:00 am Keynote Presentation: Mitochondria; How the Relic from Biology Class Could be a Powerhouse for Biotech Innovation

Synopsis

• Examine the potential of mitochondrial medicine for rare genetic diseases and common diseases of aging
• Understand targeting the mitochondria: Upstream, downstream, inner, outer, matrix
• Explore the platform approach to address metabolic cardiomyopathies and ophthalmic and neurological disorders

9:30 am New Technology for Assay of Mitochondrial Function in Greater Detail

Synopsis

  • Measure electron flow rates in mitochondria with 30 biochemical substrates
  • Measure sensitivity of mitochondria to 22 diverse inhibitors
  • Determine differences in mitochondria from different organs and tissues
  • Evaluate drug candidate effects on mitochondria with these new assays

9:55 am Panel Discussion: Evaluate the Current Challenges, Approaches & Opportunities in Targeting-Mitochondria for Age-related & Mitochondrial Diseases

  • Matthew Klein Chief Development Officer, PTC Therapeutics
  • Deanne Taylor Director of bioinformatics, Department of Biomedical & Health Informatics, The Children’s Hospital of Philadelphia
  • Natalie Ohana Yivgi Co-Founder & Chief Executive Officer, Minovia Therapeutics
  • Nicholas Hertz Chief Scientific Officer, Mitokinin
  • Pinchas Cohen Dean & Co-Founder, USC School of Gerontology & CohBar Inc.

Synopsis

• How can drug developers/research cut to the real barriers, and debate what is truly required to break through to successful treatments for mitochondrial diseases?
• What’s the impact of COVID-19 and its repercussions on existing clinical trials/research?

10:45 am Virtual Speed Networking

Synopsis

Grab a coffee from the comfort of your own kitchen and jump into this 1:1 networking session to make new connections, exchange virtual business cards and maybe even see a friendly face or two!

Early Discovery & Research

11:15 am Targeting Mitochondria for Skin Rejuvenation

  • Keshav Singh Joy & Bill Harbert Endowed Chair & Professor | Scientific Founder & Chief Scientific Officer, University of Alabama | Yuva Biosciences

Synopsis

• Examine how mitochondria play a vital role in the skin
• Discuss how mitochondrial dysfunction induces skin aging
• Consider how skin disorders manifest mitochondrial dysfunction
• Take a further look at how targeting mitochondria can help rejuvenate skin

11:45 am Using mitochondrial Ca2+ Uptake as a Therapeutic Target for Amyotrophic Lateral Sclerosis (ALS)

Synopsis

• Determine the contribution of skeletal muscle in ALS pathogenesis
• Characterize the progression of ALS in a mouse model using comprehensive longitudinal studies
• Explore the therapeutic potential of inhibiting mitochondrial Ca2+ uptake in ALS mice using a newly-generated inducible genetic mouse model (Genetic intervention)
• Assess the protective effect of a mitochondrial uncoupler (indirectly inhibit mitochondrial Ca2+ uptake), MP101/MP201, in a ALS mouse model (Pharmacological intervention)

Preclinical Development Drug Development & Studies

12:15 pm Novel Mitochondrial Uncoupler Therapeutics & Beyond

Synopsis

Novel Mitochondrial Uncoupler Therapeutics &Beyond

  • Examine new generation “smart-design” mitochondrial uncouplers
  • Enable technology to treat age-related diseases
  • Understand transformative therapies for metabolic diseases and tissue injury

12:45 pm Networking Lunch

1:45 pm Activating PINK1 Dependent Mitochondrial Quality Control to Treat Parkinson’s Disease

Synopsis

• Examine 3/A strategy to selectively activate PINK1 by stimulating autophosphorylation
• Take a Look at Identification of a potent, brain penetrant, PINK1 activator, MTK458
• Evaluate how MTK458 drives clearance of synuclein pathology in in-vivo Parkinson’s models
• Understand how MTK458 is a valuable pharmacological tool to interrogate PINK1 biology in vivo

2:15 pm USP30 inhibitors: Releasing the Brakes to Improve Mitochondrial Quality Control During Disease

Synopsis

• Consider how USP30 is the only mitochondrial deubiquitylating enzyme, perfectly placed to regulate mitochondrial ubiquitin and quality control
• Examine USP30 inhibitor target engagement and how pharmacodynamics can be followed in-vivo
• Explore Multiple disease pathologies and how they are prevented/reversed by USP30 inhibitors

2:45 pm Preclinical & Early Clinical Evidence of Stabilized Polyunsaturated Fatty Acid (PUFA) as Drug Therapy in Diseases of Mitochondrial Deficiency

Synopsis

• Examine how a worsening cascade of lipid peroxidation of PUFAs leads to cell dysfunction and death – often through mitochondrial loss of function
• View how reducing lipid peroxidation with the use of stabilized lipid drugs mitigates LPO-based cell death may halt or reverse progression of disease by restoring mitochondrial function
• Showcase how clinical and pre-clinical proof of concept has been established in several diseases

3:15 pm Virtual Networking & Scientific Poster Sessions

Synopsis

• Explore innovative research and success stories in mitochondria-targeted therapies, directly from your peers on our Poster Sessions
• Visit the Live Demo Area & Virtual Exhibition Hall

3:45 pm Mitochondrial Ion Channels as Therapeutic Targets

Synopsis

• Overview the field of exploring and targeting mitochondrial ion channels
• Examine Pano’s Novel, Non-selective Cation Channel (NMCC) in the inner mitochondrial membrane discovery and significance as target
• Explore connecting NMCC to complex diseases/disorders

Patient Perspective

4:15 pm Utilizing Technology to Better Understand Mitochondrial Disease & the Patient Journey

  • Kira Mann Chief Executive Officer, MitoAction

Synopsis

• Optimize patient-centric data-driven approaches to unlocking a meaningful understanding of day-to-day and real-life experiences of the mito patient
• Enable patient centricity in a way that transforms the patient experience in being an active partner in the clinical trials experience
• Provide reliable recording of symptoms and other relevant patient-reported outcomes data to complement trial data and to support better care decisions

4:45 pm Building the Mitochondrial Disease Patient Perspective, One Voice at a Time

  • Philip Yeske Science & Alliance Officer, United Mitochondrial Disease Foundation

Synopsis

• Examine why it is important to engage patient community early in the therapeutic development process
• Learn to understand burden of the disease as well as what meaningful treatments look like from the patient perspective
• Explore why regulatory agencies are increasingly interested in “Voice of the Patient” in addition to traditional efficacy and safety data
• Deep dive into data from patient-populated registries to understand why they are important complement to clinician-derived data

5:15 pm Chair’s Closing Remarks

  • Philip Yeske Science & Alliance Officer, United Mitochondrial Disease Foundation

5:30 pm End of Day One