Pre-Conference Discussion Day

Monday, March 28

8:00 am Networking & Registration

9.00am – 12.00pm | Workshop A – The Next Drug Development Frontier: Uncovering the Potential of Precision Neurology

The intricate and complex nature of neurodegenerative diseases have proved a burden for drug developers to create clinically effective therapies for Alzheimer’s, Parkinson’s, Huntington’s, ALS and more. Could a more precise therapeutic approach which integrates genomics, clinical data and carefully considers challenges with moving across the blood brain barrier be the solution? Is a precision neurology approach the next step industry must take to treat neurodegenerative disease once and for all? Attend this workshop to find out.

Defining Approaches for Precision Neurology

Synopsis

  • Fluid biomarkers for neurodegeneration
  • How to find the right indication and patient population in precision neurology
  • Utilizing forward and reverse translation

Utilizing Innovative Delivery Technology to Move Across the Blood Brain Barrier & Optimize Precision Targeting of Neurons

Synopsis

  • Exploring novel targets relevant to drug delivery across the BBB
  • Investigating BBB-shuttling pathways relevant to different formats of biologics
  • Understanding the rational and combinatorial design of BBB-crossing vehicles

12:00 pm Lunch & Networking

1.00pm – 4.00pm | Workshop B – Prioritizing & Progressing Pipeline in Rare Neurodegenerative Diseases as a Model to Expand into Broader Indications

Rare neurodegenerative disease comprise a vastly diverse group of pathologies with varying presentations. Although their prevelance in individual populations is low, they affect thousands of patients worldwide and can offer insight into optimizing treatment approaches against wider disease indications. Attend this workshop to be kept up to date with the latest, cutting-edge research on this fascinating topic.

Exploring RNA Targeted Approaches for Rare Movement Disorders

Synopsis

  • Exploring the medical landscape
  • Investigating RNA-mediated approaches
  • Outlining portfolio construction

Targeting Glycogen in Childhood Dementia with Expansion Potential to Larger Neurodegenerative Indications

Synopsis

  • Childhood dementias such as Lafora Disease (LD) are fatal and fewer than 5% have effective treatments
  • An antibody-enzyme fusion clears accumulated pathogenic glycogen in the brain of LD animal models, rescuing metabolism and glycosylation patterns
  • Highlights a putative strategy for currently untreatable neurodegenerative diseases with demonstrated elevated glycogen

Investigating Therapeutic Discovery Platforms for Oligonucleotides Targeting Neurodegenerative Diseases

Synopsis

  • High-throughput, high information content platform to study the function of neurons with single cell and single action potential resolution
  • Using AI/ML-based analyses to identify neuronal phenotypes to assess performance characteristics of therapeutic antisense oligonucleotides (ASOs)
  • Developing ASOs for genetically defined neurodegenerative diseases

4:00 pm End of Pre-Conference Discussion Day