Pre-Conference Day
Tuesday April 23, 2024

Pre-Conference Workshop Day

Blood-Based Biomarkers Focus Day

7:30 am Registration & Coffee

8:00 am Workshop A: Next-Generation Blood-Brain Barrier Penetrant Therapeutic Antibodies

Synopsis

The recent approval of anti-Aβ antibodies has ushered in new possibilities and challenges in the realm of neurodegenerative diseases. A significant hurdle lies in the blood-brain barrier, permitting only 0.01-0.1% of antibodies to traverse into the brain, primarily through cerebrospinal fluid. The pivotal and complex challenge of achieving selective delivery of therapeutic antibodies with high bioavailability to the brain is at the forefront of drug development.

In this workshop, we delve into innovative approaches to surmounting the challenges associated with the blood-brain barrier, including:

  • Reviewing the challenges and improvements necessary for brain shuttles effectively cross the blood-brain barrier
  • Investigating the unique clustering pattern of transferrin receptors expressed on brain vessels, which are more densely distributed than in other tissues
  • Applying the principle of super-selectivity to engineer therapeutic antibodies, enhancing brain bioavailability without compromising systemic pharmacokinetics

Join this session to gain insights into the latest research, contribute to the discussion, and explore innovative solutions in the delivery of therapeutic antibodies across the blood-brain barrier

10:30 am Morning Break & Refreshment

10:45 am Workshop B: Advancing Gene Therapy to Treat Alzheimer’s & Parkinson’s Disorders

  • Ornit Chiba-Falek Professor of Neurology, Duke University & Co-Founder, CLAIRIgene
  • Krishna Subramanian Vice President & Head of Non-Clinical & Translational Science, Seelos Therapeutics Inc.

Synopsis

As the last couple of years have welcomed approved neurological gene therapies from Bluebird Bio, PTC Therapeutics, Novartis, Sarepta Therapeutics, what is the route forward for gene therapy development for Alzheimer’s & Parkinson’s disease? This workshop explores the most recent developments in gene therapy, including brain delivery, preclinical models, translation of gene therapy research into clinical trials, including viral, non-viral vectors.

  • Evaluating models of gene therapy treatment in in vivo and in vitro and exploring new innovations to recapitulate gene therapy efficacy preclinically
  • Reviewing technologies gaining approval for direct brain delivery in gene therapy
  • Translating gene therapy biomarkers for CNS indications including the validation
  • Comparing the safety and efficacy of viral and non-viral vectors for gene therapy including AAV, lentiviral, non-viral

1:15 pm Refreshment Break

1:30 pm Workshop C: Harnessing Digital Biomarkers for Internal Decision-Making in Alzheimer’s & Parkinson’s Disease

Synopsis

As the FDA moves towards defining digital biomarkers as digital health assessments, wearable technology becomes more refined and digital markers can provide early signs of drug efficacy. Digital biomarkers can support with earlier detection to provide more longitudinal and frequent assessment, but also support internal decision making for biopharma by better predicting drug efficacy.

Look forward to:

  • Exploring stride velocity as a newly approved digital marker by the EMA and the direction that regulation is moving in
  • Utilizing digital insoles to monitor stride velocity as a marker of motor symptoms for Parkinson’s Disease
  • Utilizing passive collections monitoring activity over time from walking, shopping, social engagement and interactions
  • Navigating the regulatory route to home testing to improve patient compliance
  • Harnessing home EEG devices for measuring sleep and sleep disturbance as a marker of Alzheimer’s Disease
  • Using artificial intelligence and machine learning to predict deviation from baseline to establish using digital biomarker

3:45 pm Refreshment Break

7:30 am Registration & Coffee

7:55 am Chair’s Opening Remarks

REFINING BIOMARKER DEVELOPMENT STRATEGY FOR EARLIER DETECTION & INTERVENTION OF AD & PD

8:00 am Integrating Non-Invasive Biomarkers to Allow for Earlier Intervention in Treating Neurodegenerative Disease

Synopsis

  • The hallmarks of Alzheimer’s Disease require a multimodal approach of biomarkers to evaluate heterogeneity of disease
  • Non-invasive biomarker modalities for diagnosis, staging and treatment response
  • Enabling a personalized approach using global proteomics and molecular subtyping to reflect disease heterogeneity

8:30 am Compiling Learnings from Biomarker Consortia PPMI & ADNI for Biomarker Target ID for More Reliable, Generalizable & Clinically Relevant Biomarkers

  • Holly Soares Vice President & Head of Precision Medicine, Pfizer

Synopsis

  • Harnessing single cell genomics, transcriptomics and GWAS to identify and validate markers of genetic variants
  • Implementing blood based biomarkers in primary care and community health settings for diagnosis and patient referral
  • Integrating blood based biomarkers, clinical presentation, patient history and imaging for more streamlined patient diagnosis

HARNESSING BLOOD-BASED BIOMARKER DISCOVERY & VALIDATION FOR IMPROVED SPECIFITY & MORE SENSITIVE QUANTIFICATION OF NEURODEGENERATION

9:00 am Identifying Pathological Tau Aggregates as a Biomarker in CSF of Alzheimer’s Disease Patients

Synopsis

  • Differentiating isolation approaches applied to identify seeding competent tau aggregates from AD brain homogenates
  • Generating high-affinity antibodies towards specific conformations and phospho sites of pathological tau
  • Harnessing them to identify tau aggregates in CSF of Alzheimer’s disease

9:30 am Morning Break & Refreshment

10:30 am Diagnostics and Therapeutics for Selective Targeting of Early Toxic Oligomers

Synopsis

  • Highly specific and sensitive plasma Soluble Oligomer Binding Assay (SOBA) with FDA breakthrough designation for AD
  • Soluble Oligomer Binding Inhibitor (SOBIN) for neutralization of toxic oligomers and their effects in vivo
  • SOBA test applicable to other amyloid diseases, including PD

11:00 am Identifying Patient-Derived Biomarkers through Lipid Signatures from Non-Invasive Patient Samples for Earlier Detection of Neurodegeneration

Synopsis

  • Assessing early PK/PD relationship to inform early trial dynamics of markers
  • Integrating blood based biomarkers, clinical presentation, patient history and imaging for more streamlined patient diagnosis
  • Harnessing biofluids for less invasive diagnosis, staging and prediction of treatment response in patients

11:30 am Lunch & Discussion

1:30 pm Unbiased Multiomic Analysis to Identify Blood Based Biomarkers in Neurodegeneration

  • Carlos Cruchaga Professor of Neurogenomics, Washington University School of Medicine in St. Louis

Synopsis

  • Integrating proteomics, metabolomics and methylation into a molecular map
  • Causal inference to identify novel genes driving associations to act as a druggable target
  • Integrating findings into refined prediction models

2:00 pm Distinguishing Pathological & Healthy Neuronal Signatures on Brain-Derived Analytes in the Blood for Neurodegenerative Disease

Synopsis

  • Concordance between CSF and blood-derived biomarkers
  • Predictive power of fluid biomarkers
  • Utility in patient selection and efficacy
  • Exploring the latest insights from Phase 1 and ongoing Phase 2 data from a disease-modifying Parkinson’s Disease trial

ENABLING NEURODEGENERATIVE BLOOD-BASED BIOMARKERS AS CLINICAL & SURROGATE ENDPOINTS

2:30 pm What can Parkinson’s Disease Learn from the Recent Progress in Alzheimer’s Disease? Lessons for HER-096

Synopsis

  • Learning from the recent Alzheimer’s Disease trial successes and how this can help inform PD treatment development, highlighting the importance of biomarkers
  • Understanding the importance of surrogate markers and development of clinical endpoints for a faster read on clinical trials
  • Pushing early biology and biomarker discovery to support clinical development, examples from HER-096

3:00 pm Panel Discussion: Overcoming Barriers to Clinical Utility of Emerging Non-Invasive Blood & Imaging Tests

Synopsis

  • Navigating accessibility challenges internationally to blood based biomarkers for early diagnosis
  • Employing consortia to strategically make biomarkers more reliable, generalizable and clinically relevant to enhance the quality and credibility of biomarker validation studies
  • Exploring future directions in biofluid sampling at-home for improved patient experience and retention in clinical trial

4:00 pm Workshop D: Translational De-Risking Toolkit – Enhancing Utility of Preclinical Models of Alzheimer’s & Parkinson’s Disease

Synopsis

Despite technological and scientific progress, a significant challenge in drug development for neurological disorders remains – capturing the complexity of the human brain in laboratory models. Historically, translation has been ineffective and contributed to a catalog of failures in human studies. To continue trialing new drug treatments, an optimized approach must be taken to utilize the currently available methodologies in early research and increase the chance of success at clinical phases.

Attend this practical, solution-focused workshop to:

  • Outline current rodent models, their limitations, and optimal functionality
  • Understand the implications of transgenic models on pathophysiology
  • Improve ability to review data gleaned in preclinical studies and translate into likely clinical effect

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7:00 pm End of Pre Conference Day