8:30 am Morning Networking

9:00 am Chair’s Opening Remarks

9:10 am Driving Investment in Neurodegeneration

9:40 am Modeling Familial & Idiopathic Neurodegenerative Diseases in Mice

  • Cat Lutz Senior Director of the Mouse Repository & the Rare & Orphan Disease Center, The Jackson Laboratory

Synopsis

  • Extending common mechanisms in ALS to therapeutic strategies
  • How have the rare familial forms of the disease contributed to our knowledge
  • Exploring multiple pathogenic etiologies in neurodegeneration
  • The contribution of environment in mouse models
  • Linking models regeneration and neurodegeneration; exploring Stathmin2

10:10 am Gene Therapy for AADC Deficiency

  • Krystof Bankiewicz President & Chief Executive Officer, CEO, Brain Neurotherapy Bio, Columbus Children’s Foundation

Synopsis

  • Image guided administration of AAV2-AADC into the midbrain of AADC deficient children to maximize safety and anatomical coverage of gene therapy
  • Prospective clinical study design to utilize anterograde axonal transport to achieve physiological neurotransmission in the striatum
  • Long-lasting and life altering clinical responses seen in all subjects enrolled into the clinical and compassionate trials.

10:40 am Live Q&A With Your Expert Speakers

  • Cat Lutz Senior Director of the Mouse Repository & the Rare & Orphan Disease Center, The Jackson Laboratory
  • Jonathan Behr Partner, Dementia Discovery Fund
  • Krystof Bankiewicz President & Chief Executive Officer, CEO, Brain Neurotherapy Bio, Columbus Children’s Foundation
  • Christian Schubert Global Head of External Innovation, Research & Development, Servier

11:00 am Speed Networking

11:30 am Brain Delivery of Neuroprotective TrkB Agonist Antibody – Translation to Primates

Synopsis

  • TrkB agonist antibody delivery to the brain using a TfR1 specific VNAR antibody shuttle provides neuroprotection in a mouse model of Parkinson’s Disease
  • Development of efficient blood brain barrier (BBB) shuttle for primates as universal approach for delivery of CNS therapeutics.

12:00 pm Advances in PD Alpha-Synuclein Based Animal Models for Evaluation of Potential Therapeutics

Synopsis

  • Descriptions and examples of the use of AAV based rodent and macaque models of alpha-synucleinopathy.
  • Advances in preclinical alpha-synuclein preformed fibril models.
  • Discussions around model selection for different preclinical programs

12:30 pm Live Q&A With Your Expert Speakers

12:45 pm Lunch Break

Discovery Stream
Diving Into Human Genetics, Target Identification & Neuroinflammation

1:15 pm Unpacking the Role that Genetics Play in Neurodegenerative Diseases

  • Michael Nagle Director, Head of Human Genetics, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

Synopsis

  • Leveraging insights from human genetics, computational biology, and other data sciences to drive and maximise discovery programs
  • Exploring case studies of progressing genetically validated targets through discovery and development
  • Assessing different modalities and matching the target to the right modality using human genetics
  • Using genetics to identify the right biomarker and target patient population

1:45 pm Modern Target Identification: Exploration of the Hypotheses Around the Use of Genetics to Inform Decision Making

  • Jonathan Levenson Vice President of Translational Biology, Tiaki Therapeutics
  • Michael Nagle Director, Head of Human Genetics, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

Synopsis

  • What does it mean for a target to be genetically enabled?
  • How should we use genetics and genomics data to identify and prioritize drug targets for therapeutic development?
  • What is the role of preclinical tools if human genetic data are the primary driver for target selection?

2:15 pm Delving Into the Interface of Neuroinflammation & Neurodegeneration

Synopsis

  • Exploring the mechanisms of action for RIPK1-medicated neuroinflammation in CNS diseases, focusing on ALS,MS and beyond.
  • Reviewing RIPK1 biology and disease-associated mutations in RIPK1 signalling pathways
  • Pioneering applications of these RIPK1 inhibitors for the treatment of monogenic and polygenic neurodegenerative disorders
  • Early discovery work underway for future endeavours

2:45 pm Live Q&A With Your Expert Speakers

  • Dimitry Ofengeim Precision Neurology and Neuroinflammation Head, Sanofi
  • Michael Nagle Director, Head of Human Genetics, Eisai Center for Genetics Guided Dementia Discovery (G2D2)
  • Jonathan Levenson Vice President of Translational Biology, Tiaki Therapeutics
  • Rouba Kozak Senior Director, Neuroscience & Translational Medicine, Takeda

Translational Stream
Advancing the Application of Novel Biomarkers Into Clinical Practic

1:15 pm An Encyclopaedia of Currently Available Biomarkers for Neurodegenerative Diseases

Synopsis

  • Dissecting the current state of AD research, diving into new therapeutics and biomarkers
  • What do we have now and what needs to be developed?
  • How about biomarkers for non-AD neurodegenerative diseases?
  • What is needed for implementation of new biomarkers in clinical practice?
  • How can biomarkers be used today?
  • How will biomarkers be used the day we have effective disease-modifying therapy?

1:45 pm Exploring the Utility of P-tau as a BloodBased Biomarker in Neurodegeneration

  • Jeff Dage Senior Research Advisor, Eli Lilly & Company

Synopsis

  • The science and background of P-tau217 and P-tau181
  • Utility of P-tau in diagnosis and prognosis
  • Application of P-tau in clinical trials
  • Future opportunities that may alter our understanding of Alzheimer’s Disease

2:15 pm Evaluating the Role of CNS Exosomes in Diagnosing AD & PD

Synopsis

  • Delving into our approach to using biobanked clinical trials samples, human postmortem tissues and animal models
  • Purifying the source of proteins coming from the brain to track progression of neurodegenerative disease
  • Exosome trafficking and potential
  • Methodological considerations in exosome harvesting

2:45 pm Live Q&A With Your Expert Speakers

3:00 pm Afternoon Networking

Discovery Stream
Delving Into Gene Therapy: How Can We Best Implement New Clinical Trials?

3:15 pm Emerging New Gene Therapies for CNS Indications

  • Vanessa Almendro Head of Strategy & Operations, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

Synopsis

  • What is the current status for the emerging new Gene Therapies for CNS indications?
  • Choosing the right modality for the right target and the right patient
  • Understanding implications in the clinical implementation of Gene Therapies for CNS disorders

3:45 pm Designer DNA Drug Therapy for Neurodegenerative Diseases

  • Don W Cleveland Chair, Distinguished Professor of Cellular & Molecular Medicine & Neurosciences Member, Ludwig Institute for Cancer Research

Synopsis

  • Uncovering the mechanisms underlying the major genetic forms of ALS
  • Evaluating ongoing efforts and clinical trials in gene therapy for ALS, FTD, Huntington’s Disease, PD and beyond
  • Paving the path forward, what’s on the horizon?

4:15 pm Stoking Protein Production – Pioneering a Unique Therapeutic Approach Using Antisense Oligonucleotides

  • Gene Liau Executive Vice President; Head Research & Preclinical Development, Stoke Therapeutics

Synopsis

  • Designing a platform to address protein deficiency by precisely upregulating target protein expression
  • Targeted Augmentation of Nuclear Gene Output (TANGO) technology platform is being used to pursue disease-modifying treatment for severe genetic diseases via a precision medicine approach
  • Building data to the clinic for genetic epilepsies and advancing early programs focused on multiple therapeutics areas with initial emphasis on the CNS and the eye
  • How might our technology be applicable for neurodegeneration?

4:45 pm Live Q&A With Your Expert Speakers

  • Don W Cleveland Chair, Distinguished Professor of Cellular & Molecular Medicine & Neurosciences Member, Ludwig Institute for Cancer Research
  • Gene Liau Executive Vice President; Head Research & Preclinical Development, Stoke Therapeutics
  • Vanessa Almendro Head of Strategy & Operations, Eisai Center for Genetics Guided Dementia Discovery (G2D2)
  • Rouba Kozak Senior Director, Neuroscience & Translational Medicine, Takeda

Translational Stream
Striving for Clinical Validation of Biomarkers


3:15 pm Assessing Why Preanalytical Testing is Key in Performing High Quality Biomarker Studies

Synopsis

  • Cutting-edge developments in blood-based biomarkers for neurodegenerative diseases
  • Biomarker identification using proteomics methods, including mass spectrometry and array-based proteomics technologies
  • Analytical and clinical validation for diagnostic use of the most promising biomarkers by immunoassays
  • Ensuring the quality of biosamples

3:45 pm Exploring a Regulatory Perspective on Advancing Biomarkers for Neurodegenerative Diseases

Synopsis

  • Overview of the potential use of biomarkers for drug development for neurodegenerative conditions
  • Experience on EMA Qualification of novel methodologies (biomarkers)
  • Genetic markers for neurodegenerative conditions
  • Evaluating approaches of using biomarkers for enrichment purposes in clinical trials
  • Where does the field need to put more efforts to develop biomarkers that will be able to help us test new hypothesis across neurodegenerative diseases?
  • What is the regulatory willingness to accept such biomarkers, what do we need to see in order to validate biomarker endpoint as a primary endpoint for an approval as opposed to a clinical effect?

4:15 pm Evaluating Clinical Observations & Key Learnings From SIGNAL HD Clinical Trial

Synopsis

  • Neurons upregulate SEMA4D in response to stress during neurodegenerative disease progression
  • SEMA4D triggers inflammatory activation of astrocytes in HD and AD
  • Plans to execute SIGNAL AD in 2021, study design and patient recruitment

4:45 pm Live Q&A With Your Expert Speakers

5:00 pm Virtual Poster Session

Synopsis

View submitted Posters with Resources here:

Virtual Poster Session

5:35 pm Chair’s Closing Remarks & Final Reflections