7:30 am REGISTRATION
8:25 am Chair’s Opening Remarks
TACKLING TOXICITY: LEVERAGING OUR SAFETY & EFFICACY TESTING FOR A NEW ERA OF MITOCHONDRIAL THERAPEUTICS
8:30 am Utilising our Understanding of Mitochondrial Toxicity of Existing Drugs to Guide Mitochondrial Drug Development
Synopsis
- Leveraging our understanding of the mitochondrial toxicity of existing drugs to bridge the gap towards development of safe treatments
- Applying in vitro and in silico models to drug screening funnels
9:00 am Overcoming the Technical Limitations of In Vivo Models for Representing Human Mitochondria-Related Metabolic Disease
Synopsis
- Analysing the translation of in vitro to in vivo preclinical metabolic models and exploring additional models to put in place that improves translation
- Evaluating the translatability of in vivo models for mitochondrial targeting, the differences of rodent and human cellular metabolism
- Holistically assessing the quality of in vivo models to represent the severity and progression of mitochondrial diseases
9:30 am MORNING BREAK & SPEED NETWORKING
Synopsis
Our structured networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in the field and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond
OUT WITH THE OLD & IN WITH THE NEW: MITOPHAGY & IMPROVING CELLULAR HOMEOSTASIS
10:15 am VNA-318: An Inducer of Mitophagy to Address Mild Cognitive Impairment & Neurodegeneration
Synopsis
- VNA-318 is a safe inducer of mitophagy in vitro and in vivo with excellent potency, selectivity, and brain penetration profile
- Unique mechanism of action able to regulate short-term memory and induce mitophagy
- First-in-human studies expected to kick-off in Q2 2024
10:45 am Targeting USP30 & Parkin: Boosting Mitophagy & Mitochondrial Quality Control as a Treatment Strategy for Neurodegenerative Disease
Synopsis
- Mitophagy as an area of convergence in neurodegenerative disease
- Selecting patients that may best respond to mitochondrial therapeutics with feature-based selections and regression AI algorithms
- Securing the therapeutic window for safe levels of target modulation that lead to disease modification for mitochondrial therapeutics
11:15 am Identification of a New Target to Enhance Mitophagy Flux & Treat Muscular Dystrophy
Synopsis
- Unveiling of a novel target that potently enhances mitophagy flux in cells and in vivo when activated
- Overview of molecular data supporting mechanism of action which lead small molecule induces mitophagy
- In vivo data supporting beneficial effect of mitophagy induction in preclinical model
11:45 am Pharmacological PINK1 activation Ameliorates Pathology in Parkinson’s Disease Models
Synopsis
- Improving mitochondrial quality control as a treatment for Parkinson’s and chronic diseases of mitochondrial dysfunction
- Combining genetically validated targets with small molecules to create a novel therapeutic modality
12:15 pm LUNCH & NETWORKING BREAK
1:15 pm Panel Discussion: Exploring the Safety & Strategy Considerations of Different Treatment Modalities of Mitochondrial Therapies
Synopsis
- Explore the rationale behind decision making of different treatment modalities, positioning small molecules amongst biologics, gene editing therapies and mitochondrial transplantation
- Discuss the needs of specific disorders and why this may favour one modality against anot
GENE EDITING & GENE THERAPIES TARGETING MTDNA MUTATIONS
1:45 pm Rewriting Mitochondrial Genomes as Therapy
Synopsis
- Strategies and considerations for correction of pathogenic mitochondrial mutations
- Translating recent advances in gene editing into mitochondrial therapies
- Considerations for modeling mitochondrial genetic therapy without established animal models
2:15 pm Advancing Gene Therapy for Mitochondrial Eye Diseases: LHON and ADOA
Synopsis
- Diving into the world of gene therapy and developing advanced gene therapy drugs for LHON (Leber’s Hereditary Optic Neuropathy) caused by ND4 and ND1 mutations, offering new hope for patients
- Exploring the progress of Neurophth’s NFS-01 gene therapy, currently in clinical stages, designed to target and treat LHON associated with ND4 mutations
- Discovering IND enabling gene therapy designed to combat ADOA (Autosomal Dominant Optic Atrophy), offering a promising avenue for addressing this rare genetic disorder affecting the optic nerves
2:45 pm AFTERNOON BREAK & SCIENTIFIC POSTER SESSION
Synopsis
This is an informal session to help you connect with your peers in a relaxed atmosphere and continue forging new and beneficial relationships. With an audience of mitochondria enthusiasts eager to hear the latest cutting-edge advancements, you will have the opportunity to display a poster presenting your own work. Additionally, you will have the chance to review other’s posters displaying novel approaches to targeting mitochondria, optimizing mitochondrial dysfunction, biomarker discovery and more.
PROTECTING THE POWERHOUSE OF THE CELL
3:15 pm Developing Brain-Penetrant Mitochondrial Protective Small Molecules for Rare Epilepsies, Mitochondrial Diseases, & Parkinson’s Disease
Synopsis
- Screening strategy that lead to the discovery of mitochondrial and neuroprotective compounds
- Development of mitochondrial disease models in zebrafish to understand progression of disease and as a screening tool
3:45 pm Preventing Neurodegeneration by Protecting Mitochondrial Structure & Function
Synopsis
- Characterization of a novel cellular target for the protection of mitochondria from metabolic and oxidative stress
- Examining preclinical data demonstrating delivery of therapeutic compound to target tissues
- Analysing preclinical data in ophthalmic indications of retinitis pigmentosa and dry age-related macular degeneration