Explore the Agenda
8:00 am Check In, Coffee & Light Breakfast
Fluid Biomarker Seminar Day
8:55 am Chair’s Opening Remarks
9:00 am Panel Discussion: Deciphering the Landscape of Emerging Neurodegenerative Fluid Markers Beyond p-tau & α-Synuclein
- GBA, LRRK2, and TREM2: Where do these emerging targets stand in terms of measurable, scalable biomarkers?
- What should define a ‘clinically useful’ biomarker when translatability remains uncertain?
- How do we ensure fluid biomarkers reflect causal disease biology rather than downstream noise?
10:00 am Morning Break & Refreshments
10:30 am The Use of TauPET & Blood GFAP in Clinical Trials & Practice to Enable Earlier Change Detection & Smaller Alzheimer’s Trials
- Leveraging biomarker stratification and early change detection through integrated analysis of cognitive and functional scores to reduce trial duration and enhance patient engagement.
- How close are Tau PET and blood GFAP being accepted as primary end‑points to enable smaller sample sizes once validated?
11:00 am Roundtable Discussion: Integrating Genetics, Fluid Markers & Digital Endpoints to Sharpen Trial Enrichment
- Leveraging GBA, APOE, and LRRK2 genetics alongside plasma and CSF biomarkers to refine patient stratification strategies
- Exploring how digital endpoints (motor, cognition, speech) complement fluid biomarker signals to create multidimensional risk profiles
- Discussing the practicalities of embedding composite biomarker strategies into protocol design to improve recruitment speed, reduce costs, and strengthen regulatory alignment
Preclinical Models Workshop Day
9:00 am Workshop A | Decoding Protein Aggregation In Vitro: Next-Generation Assays for Alzheimer’s & Parkinson’s Translation
Conventional animal models, from MPTP rodents to amyloid/tau transgenics, no longer provide sufficient confidence that Alzheimer’s or Parkinson’s candidates will translate to patients. As the field pivots to next-generation in vitro systems, this workshop compares advanced platforms across tau, α-synuclein, and emerging mechanisms. Attendees will explore how to link cellular potency to CSF biomarkers and translate those signals into IND-ready decision-making for both AD and PD.
Key Questions to be Addressed:
- Physiological relevance: How can we improve in vitro assays to capture endogenous tau and α-synuclein pathology without relying on artificial overexpression? What lessons can we draw from limitations of in vivo models like MPTP or transgenics?
- Assay design: What are best practices for high-throughput, image-based aggregation assays that can differentiate seeded aggregation from de novo misfolding, while retaining scalability and signal clarity?
- Biomarker linkage: How can in vitro tau and α-synuclein potency readouts be more reliably tied to in vivo CSF biomarker outcomes such as pTau181, pTau217, or α-synuclein seed assays? What magnitude of effect or PK/PD relationship should be demanded preclinically?
- Readouts for IND-enabling packages: Which cellular biomarkers (aggregation, synaptic function, neuroinflammation, resilience, mitochondrial stress) are most predictive for Alzheimer’s and Parkinson’s? How can imaging, transcriptomic, or multi-omic readouts guide clinical endpoints?
- Next-generation models: With mechanisms like gut–brain axis signaling, ferroptosis, lipid metabolism, and resilience genetics on the horizon, how should in vitro systems evolve? Are organoids, co-cultures, or iPSC-derived platforms ready for prime time in AD and PD drug discovery?
12:00 pm Lunch & Networking Break
Fluid Biomarker Seminar Day
1:00 pm NeuroInflammatory Blood Markers: From Exploratory to DecisionMaking Tools
- Serum NFL, cytokines and complement proteins in faster progressing AD/PD phenotypes
- Exploring multiplex panels for patient stratification
1:30 pm Quantitative Fluid Biomarkers for Precision Neurodegenerative Trials: From p-tau217 to Neurofilament Light
- Using plasma p-tau217 as a prognostic tool to enable early detection of pathological changes and improve trial enrichment and patient stratification
- Implementing neurofilament light (NfL) as a progression biomarker to provide scalable, blood-based measure of neuronal damage and support monitoring of disease-modifying effects
- Boosting regulatory confidence with multi-omic biomarker panels to integrate proteomics, immunoassays, and CSF–blood correlations to deliver robust endpoints
2:00 pm Break & Networking
2:30 pm Roundtable Discussion: Harmonising Fluid‑Biomarker Assays Across Sites for Regulatory Confidence
- Aligning plasma p‑Tau217 assays and shared cut‑offs across trial labs standardises early‑stage Alzheimer’s screening and cuts unnecessary PET scans and enrolment delays
- Quantifying the α‑synuclein RT‑QuIC seeding assay with synthetic calibrators converts a yes/no read‑out into a dose‑response biomarker, permitting smaller, mechanism‑rich Parkinson’s studies
- Benchmarking CSF dopa‑decarboxylase alongside NFL in AMP‑PD biobank samples harmonises Parkinson’s progression metrics across sites and boosts regulator confidence in multicentre data packages
3:30 pm Restoring Endo-Lysosomal Function: Translational Biomarkers to Accelerate Parkinson’s Therapeutics
- Discovering metabolomic and lipidomic signatures of endolysosomal dysfunction in PD patients and healthy controls to define disease-relevant biomarker baselines
- Validating pharmacodynamic biomarkers via small molecule target manipulation in cell and patient-derived samples to track treatment effects early in the pipeline
- Accelerating drug discovery by using these biomarkers to derisk candidate selection, inform dose selection, and shorten time to clinical proof-of-concept in Parkinson’s
Preclinical Models Workshop Day
1:00 pm Workshop B | Reimagining Translational Validity: Optimizing In Vivo Models for Predictive Power in Alzheimer’s & Parkinson’s Drug Development
Animal models remain the crucial, but often weakest, link between discovery and clinic. This workshop dissects why toxin‑based and first‑gen transgenic models miss human disease complexity and progression and showcases next‑generation options such as hybrid humanized strains, gut‑origin paradigms, and vector‑delivered pathology that better mirror biomarker change and clinical progression. Walk away with clear criteria and partnership tips for selecting, benchmarking, and refining in‑vivo systems that sharpen translational signals and cut downstream risk.
Key Questions to Be Addressed:
- What criteria should define a ‘fit-for-purpose’ in vivo model in neurodegenerative disease, especially when pathological complexity and slow disease progression challenge current paradigms?
- How can we enhance the translatability of in vivo findings to clinical endpoints, particularly in tracking neuroinflammatory, synaptic, or resilience-related pathways beyond amyloid and α-synuclein?
- With combinatorial and multi-target strategies becoming more common, how do we best model additive or synergistic effects across amyloid, tau, α-synuclein, and neuroinflammatory cascades?
- What role can newer disease paradigms, such as gut-brain axis induction or viral vector delivery, play in enhancing the physiological relevance of current models?
- How do we benchmark CROs and academic centres developing novel models? What best practices and vendor collaborations can accelerate model refinement and validation?