Conference Day Two
*Please note this event has been cancelled*
9:20 am Chair’s Opening Remark
TRANSLATING DISCOVERIES: ADAPTING OVER THE LIMITATIONS OF ANIMAL MODELS TO ASSESS CNS DRUG EFFICACY
9:30 am Artificially Driving Neurodegenerative Pathologies in In Vivo Models
Synopsis
- Reimagine how we profile animal models to understand disease progression when using artificial strategies to improve characterisation of preclinical models and improve translation
- Incorporating multiple models and characterizing tissues from different species to assess expression profiles and disease progression in context
- Scrutinize human-derived signatures used to evaluate in vivo models amongst the lack of access to human samples
10:00 am Intelligent Use of Multiple Data Streams for Comprehensive Computational Support During Drug Trial Phases
Synopsis
- Utilize AI and machine learning models to incorporate known genetic risk factors and discover novel associated genes steering neurodegeneration
- Understand the approaches used to address problems while working within limited patient-centred data
- Creating and interpreting webs of information to determine what neurological targets should not proceed
10:30 am Morning Break & Networking
11:30 am Roundtable Discussion: Unleashing a New Era of Preclinical Studies for Neuroscience Drug Development
Synopsis
- Debate the validity of using animal models and assess the timeline of non-clinical toxicity studies in relation to CNS disorders to prevent unforeseen side effects
- Explore how to leverage cell-based assays, microphysiological systems, and computational modelling in conjunction to demonstrate mechanism of action and safety of novel CNS therapies
- Recalibrate how models can be used to reassess and reengineer potentially flawed drugs earlier to maximize their success rate in clinic
12:00 pm Feedback Panel
Synopsis
- Nominated speakers from each table will unite to share the alternate perspectives and discussions from each table
- Highlight the future of CNS model development to improve drug development across neuroscience disease areas
12:30 pm Lunch & Networking
ENSURING MODEL INTEGRITY ACROSS CNS DISORDERS & FUTURE PERSPECTIVES
2:00 pm Mastermind Discussion: Highlighting the Value of Collaboration & Pre-Competitive Sharing – Moving Towards Universal CNS Models & Predictive Models
Synopsis
Across methods of modelling disease access to data and legality of data acquisition remains a challenge, discuss aspects from iPSC access, validation of academic models, and messy clinical data for computational models
- Discovering how clinicians collaborate with computational biologists to ensure high quality data to be incorporated as priors
- Explore the considerations to ensure clinical data remains anonymous when developing models
- Discuss precompetitive sharing and assess the value of creating universal disease models
2:30 pm Considerations of Acute In Vivo Mouse Models to Interrogate Neurological Disease and Drug Development
Synopsis
- Using viral gene editing methods to create new models to recapitulate symptomology in vivo in mice
- Understand considerations that must be made when attempting to study preclinical behavioural and genetic differences
- Dive into the solutions and challenges of studying chronic disorders compared to ones with clear genetic causes
3:00 pm Afternoon Break
BOLSTERING LIMITED HUMAN CNS SAMPLES TO PREDICT DRUG OUTCOMES
3:30 pm Creating a Virtuous Cycle of In Vitro & In Silico Models Informing their Own Improvement
Synopsis
- Collecting functional measurements from in vitro samples at high throughput to inform computational models that can be used for in silico screening and predictions
- Applying machine learning to identify disease phenotypes in human cellular models, enabling a quantitative approach for optimizing therapeutic candidate choice
- In silico models of functional activity can be used for target deconvolution, disease phenotyping, or drug repurposing
4:00 pm Blood Brain Barrier & Beyond: Developing Effective Models to Study Penetration & Therapeutic Dose in Humans
Synopsis
- Ranking molecule capability to cross human blood brain barrier models to understand the human therapeutic dose
- Achieving a paralleling as blood brain barrier defects in neurodegenerative disease, understanding more of the biology to identify new targets and potential to modify the blood brain barrier
- Developing assays in-house to understand how brain cell types interact with blood brain barrier
4:30 pm Validation of Demyelinating Models for Small Molecule Discovery of Multiple Sclerosis Therapies
Synopsis
- Use of electrophysiology and biomarkers as surrogate endpoints of remyelination in autoimmune encephalomyelitis (EAE) and cuprizone preclinical models
- PIPE-791 a brain-penetrant LPA1 antagonist induces OPC differentiation and remyelination in demyelinating models