8:25 am Chair’s Opening Remarks

  • Neil Cashman Chief Scientific Officer & Co-founder, ProMIS Neurosciences

Interrogating Causative Genes & Cellular Pathways with Robust ALS Models to Recapitulate Disease Pathogenesis & Validate Targets

8:30 am Harnessing New Insights into Human Genetics & Stem Cell Technology to Provide Genetically-Validated Targets for the Sporadic ALS Patient Populations

  • Kasper Roet Founder & Chief Executive Officer, QurAlis

Synopsis

  • Investigating genetically validated targets in ALS patient-derived cells to develop precision therapeutics
  • Examining the emerging role of aberrant splicing in the development of disease
  • Highlighting the need of human model systems to select the right therapeutic candidate molecules

9:00 am From Monotherapy to Combination Therapy: Considering a Paradigm Shift to Target Complexity of ALS

Synopsis

  • Pursuing combination therapy to decelerate disease progression and improving QoL
  • Demonstrating therapeutic efficacy in Zebrafish to assess a synergistic combination therapy
  • The role of biomarkers in the regulatory approval process of novel therapies in ALS

9:30 am Revolutionizing Discovery & Development of ALS Therapeutics Using Transformational, AI-Powered ‘All-in-Human’ Platforms

Synopsis

  • Target discovery using human tissue, genomics and AI-machine learning platforms
  • Proof of biology: validating new therapeutic targets
  • Advancing candidates towards clinical development

10:00 am Morning Break & Networking

Enlightening Pathophysiology of ALS & Clarifying Novel Pathways for Therapeutic Intervention

11:00 am Panel Discussion: Mechanisms, Targets, & Therapeutic Potential: Dissecting Exciting Opportunities to Drive Novel Therapeutic Strategies

Synopsis

  • Dissecting causative genes and cellular processes involved in ALS pathogenesis to shape future directions of therapeutic development
  • C9orf72, SOD1 & TDP-43: mapping promising targets
  • Can we move the needle in genetically defined patient populations and translate learnings to patients with sporadic ALS?

11:30 am Characterizing Pre-symptomatic ALS to Identify Opportunities for Early Therapeutic Intervention

Synopsis

  • Conceptual framework for describing and studying pre-symptomatic ALS
  • Prodromal ALS: mild motor impairment, mild cognitive impairment and mild behavioral impairment
  • Strategies for early therapeutic intervention to delay or prevent onset of clinically manifest ALS

12:00 pm SARM1 Inhibition as a Potential Therapeutic Strategy in ALS

  • Miroslaw Brys Senior Director, Neurodegeneration Clinical Head, Eli Lilly & Company

Synopsis

  • SARM1 is the central driver of axonal degeneration
  • Genomics analysis suggest SARM1 as an ALS candidate risk gene
  • Downstream effects of SARM1 inhibition might potentially translate to robust biomarker, electrophysiology and clinical efficacy signals in ALS trial

12:30 pm Lunch & Networking

1:30 pm Investigating Pathways for Intervention: Targeting RIPK1 in ALS

  • Dimitry Ofengeim Head of Precision Neurology & Neuroinflammation Cluster, Sanofi

Synopsis

  • Assessing RIPK1 kinase activity modulates inflammatory signaling in microglia and astrocytes
  • RIPK1 is elevated in ALS patient spinal cords and RIPK1 kinase inhibition delays ALS disease progression in the SOD1G93A mouse model
  • Reviewing the Himalaya trial using a RIPK1 inhibitor to treat ALS patients

2:00 pm Generating Effective Therapeutic Strategies for Sporadic Disease with Gene Therapies in ALS

  • Neil Cashman Chief Scientific Officer & Co-founder, ProMIS Neurosciences

Synopsis

  • Gene therapies for sporadic disease: an emerging concept
  • Understanding heterogeneity of disease mechanisms
  • Translating improving understanding of ALS pathogenesis to identify promising targets

2:30 pm Stratifying ALS Patients into Clinically Relevant Subpopulations to Develop Targeted Therapeutics

Synopsis

  • Pursuing targeted approaches to drug discovery: unravelling the biological heterogeneity of ALS
  • Employing biomarkers to distinguish distinct patient subtypes and provide rational for individualized treatments
  • Developing targeted therapeutics for stratified patient populations

3:00 pm Afternoon Break & Networking

3:30 pm Exploring Mastocytosis & Kinases as the Basis of the History of Masitinib in ALS Mechanism of Action & Reviewing Clinical Results

Synopsis

  • Investigating the mechanism of action of masitinib in ALS with the SOD1 rat model
  • Assessing Masitinib to block the activity of tyrosine kinases and decrease nerve cell dysfunction in ALS
  • Evaluating clinical results of Masitinib in combination therapy

4:00 pm Developing a Synaptic Regenerative Therapy in ALS Using a Novel Spinogenic Small Molecule

Synopsis

  • ALS is a synaptopathy that involves an early and progressive loss of glutamatergic synapses in motor and cognitive centers
  • Developing novel small molecule therapeutics for the synaptopathies that regenerate lost synapses to physiologically relevant levels and has a lead clinical candidate (SPG302) selected for ALS
  • In preclinical studies, SPG302 improved motor function and survival in an aggressive TDP43 mouse model of ALS, and led to a recovery of respiratory function in a very defined and quantitative model of cervical spinal cord injury

4:30 pm Chair’s closing remarks

  • Neil Cashman Chief Scientific Officer & Co-founder, ProMIS Neurosciences