Conference Day Two

Day One

Wednesday, October 8

Day Two

Thursday, October 9

8:00 Check-In & Coffee

8:45 Chair’s Opening Remarks

  • Jon Levenson Chief Scientific Officer, FireCyte Pharmaceuticals

Refining Biomarker Clinical Readiness to Enable Better-Defined Trials & Accelerate Clinical Validation

9:00 Defining Context of Use to Anchor Biomarker Relevance & Streamline the R&D Pipeline

Synopsis

  • Determining Context of Use: Diagnostic, prognostic, predictive and pharmacodynamic
  • Assay validation across research, development, and clinical stages
  • When is a biomarker meaningful enough to replace or complement clinical outcomes?
  • From correlation to mechanistic specificity: Tracking vs. proxy biomarkers

9:30 Decoding Co-Pathologies to Refine Disease Definitions, Enhance Target Engagement, & Optimize Patient Stratification in Neurodegeneration

  • Terina Martinez Executive Director, Critical Path For Rare Neurodegenerative Diseases (Cp-Rnd), Critical Path Institute

Synopsis

  • Navigating overlapping pathologies in AD, PD, ALS, and MS: tau, amyloid, αsynuclein, TDP-43, and others
  • Quantifying and differentiating overlapping pathologies and their impact on diagnosis and treatment
  • What deciphering co-pathologies means for patient stratification and therapeutic targeting

10:00 Morning Break & Networking

Regulatory & Clinical Validation Pathways: Clinical-Grade Biomarkers, Regulatory Guidelines, Surrogate Endpoints, & Biomarker Qualification

11:00 How Imaging Can Be Used in a Clinical Setting to Improve the Credibility of Assessments – A Case Study

Synopsis

  • Combining biomarker modalities to support development of therapies in neurodegenerative diseases
  • Application of neurophysiological biomarkers in Parkinson’s and Alzheimer’s diseases
  • Lost in translation: selecting efficacious drug exposures based on experimental findings

11:30 Panel Discussion: Optimizing Imaging Endpoints, Mapping the Current Imaging Landscape & Emerging Tools in Neuroscience

  • Mihaly Hajos Scientific Advisor, Cognito Therapeutics
  • Terina Martinez Executive Director, Critical Path For Rare Neurodegenerative Diseases (Cp-Rnd), Critical Path Institute

Synopsis

  • Review of Current Imaging Modalities: Overview of PET, MRI, fMRI, and other techniques used in neurodegenerative disease trials (e.g., AD, PD, MS, ALS)
  • Limitations of Imaging Biomarkers in Clinical Trials: Challenges including accessibility, cost, patient burden, and limitations in sensitivity or specificity
  • Innovations on the Horizon: Discussion of emerging imaging techniques and tools, including novel radiotracers, high-resolution modalities, and multimodal approaches
  • Integrating Imaging with Other Biomarker Modalities: How imaging can complement fluid biomarkers, digital measures, and omics data for a more holistic disease profile
  • What regulatory criteria must be met for a surrogate endpoint to be accepted as a validated substitute for a clinical endpoint?

12:15 Lunch Break & Networking

1:15 Outlining the Regulatory Roadmap for Efficient Official Validation of Biomarkers

  • Jiri Aubrecht Vice President & Head of Clinical Biomarkers, Prothena Corporation

Synopsis

  • Presenting the necessary steps for different pathways of biomarker approval, minimal requirements, metrics and best practice
  • Appropriately validating assays from an analytical, clinical and regulatory standpoint for with the correct context of use
  • Outlining a strategic approach to IND filings by aligning timing, data readiness, and regulatory requirements
  • Knowing when and how to push for approval based on the strength of supporting biomarker data 

From Molecules to Machines: Harmonizing Technologies, Imaging & AI in Data Analysis

1:45 Intra-net of Cellular Things: Multi Omics and Secretome Based Discovery of Endogenous Fibroblast Cell States in Inflammation and Fibrosis

  • Jamila Siamwala Senior Scientist - Cell, Molecular Biology & Exploratory Research, Vertex Pharmaceuticals

Synopsis

  • Exploring how multi-omics and secretome profiling can illuminate diverse fibroblast cell states and their roles in inflammatory and fibrotic processes
  • Discussing the value of integrative approaches to capture cellular communication networks and pathways underlying disease progression
  • Highlighting opportunities for biomarker discovery and therapeutic targeting by decoding the cellular “intra-net” that connects fibroblast activity with broader tissue outcomes

2:15 Roundtable Discussion: Piecing It All Together: How Multi-Omics Reveals Hidden Patterns to Unlock a Deeper Understanding of Disease

Synopsis

Exploring how integrative multi-omics approaches are transforming our ability to decode disease complexity, particularly in heterogeneous neurological disorders like Alzheimer’s, Parkinson’s, MS, and ALS. Gain insight into how combining omics layers can improve biomarker discovery, support precision medicine, and drive better patient stratification.

  • The limitations of relying on single-omic data in complex, multi-pathway diseases
  • Overview of multi-omics: Combining layers to contextualise molecular interactions
  • How integrated data enhances the resolution and specificity of biomarker discovery
  • The Omics Spectrum: Genomics, transcriptomics, proteomics, epigenomics, lipidomics & metabolomics
  • Discussion of emerging omics layers: Glycomics, single-cell omics and secretomics

3:00 Chair’s Closing Remarks & End of Conference

  • Jon Levenson Chief Scientific Officer, FireCyte Pharmaceuticals
Conference Day One
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