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February 20-22, 2018
Boston, USA
Only 1 Week Remaining

Day One
Wednesday, February 21, 2018

Day Two
Thursday, February 22nd, 2018

Chairs Welcoming & Opening Remarks

  • Richard Wyse Director of Research & Development Research & Development , The Cure Parkinson's Trust

Novel Therapeutic Approaches for Targeting Neuroinflammation & Neurodegeneration

(Re) Introduction to Microglia as Intrinsic Brain Cells

  • Richard Ransohoff Previously VP & Senior Research Fellow Neuroimmunology (Biogen), Harvard Medical School


  • Demonstrating the role of microglia enter the developing CNS in early embryogenesis and carry out crucial developmental tasks
  • Highlighting how basal and reactive microglial transcriptomes point towards mechanisms underlying functions
  • Showcasing how our microglial research tool kit continues to grow but still lacks some desirable utensils

Shedding Light on the Role of TREM2 in Alzheimer’s Disease

  • Damian Crowther Director, R&D; Neuroscience IMED Biotech Unit, AstraZeneca


  • Demonstrating how TREM2 shedding from the microglia leaves them “blind” to debris in the brain
  • Exploring how shed TREM2 is increased in neurological diseases and accelerated shedding is linked to Alzheimer’s disease
  • Proteolysis occurs at a single position in the protein, providing a target for therapy

The Importance of Microtubular Proteins & Cytokines in Neuroplasticity & Neuroinflammation


  • Exploring the interplay between alterations in microtubular proteins and cytokines in neuroinflammation and neuroplasticity
  • Demonstrating CSF data highlighting the relationship between a-syn, Tau and microtubules and their effects on plasticity
  • Evaluating the interaction between microtubular proteins and cytokines and possible pharmacological interventions

Session Q&A Panel

  • Richard Ransohoff Previously VP & Senior Research Fellow Neuroimmunology (Biogen), Harvard Medical School
  • Damian Crowther Director, R&D; Neuroscience IMED Biotech Unit, AstraZeneca
  • Massimiliano Bianchi General Manager & Scientific Director, Transpharmation Ltd


  • What is the pathological role of neuroinflammation and the immune system in the development of neurodegenerative diseases?
  • How should we be targeting and/or developing dynamic therapeutic strategies for tackling neuroinflammation?
  • How can genetics drive the identification of novel targets for immune-based therapy towards microglial and neuroinflammation in neurodegenerative disorders?

Speed Networking & Morning Refreshments

11.00 Systems Biology Approaches to Accelerating Neurodegenerative Drug Discovery

  • Demonstrating how large-scale genomic datasets can be used to tackle three core challenges in CNS drug discovery and development:
  • Identifying next-generation therapeutic targets; Selection of appropriate preclinical disease models; Patient enrichment strategies and biomarker
  • Exploring how can patient derived iPSc’s can be combined with patient genetic and gene expression data to create an “all-in-human” approach to drug discovery

Alice Zhang, CEO, Verge Genomics

11.20 Transcriptomic Profiling & Experimental Validation to Explore Drug Repurposing in Parkinson’s Disease

  • Exploring the value of using iPSC-derived dopamine neurons generated from Parkinson’s patients in research
  • Highlighting the importance of experimental validation of an in silico prediction in robust models of Parkinson’s
  • The central role of lysosomal biology in Parkinson’s
  • Does transcriptomic analysis of disease models provide a short-cut to drug repurposing?

Richard Wade-Martins, Professor Molecular Neuroscience, University of Oxford

11.40 Modulation of Histone Modifying Enzymes for Treatment of Neurodegenerative Disease

  • Demonstrating the modulation of the histone lysine demethylase, LSD1, has positive effects on cognition and neuroinflammation in different animal models of neurodegenerative disease
  • ORY-2001 has finalized Phase I studies and is ready for the trials in patients with neurodegenerative disease

Tamara Maes, CSO and VP, Oryzon Genomics S.A.

12.00 Session Q&A; Panel

  • Where can genetics and big data be best utilized to drive the identification of novel pathway and target selections?
  • Can genetic profiles be utilized to identify higher risk patient populations in earlier phases of disease initiation?
  • How should we factor in the dynamic relationships, epigenetic drivers and pathology of neurodegenerative disease?
  • How does the heterogeneity of AD and other degenerative diseases dictate focusing on distinct subpopulations?

Alice Zhang, Verge Genomics
Richard Wade-Martins, University of Oxford
Tamara Maes, Oryzon Genomics S.A.

11.00 Single Domain Antibody Platform for Delivery of Biologics to the CNS

  • Combination of in vivo and in vitro phage selections allowed for identification of efficient, cross-species reactive, and safe CNS shuttles specific to TfR1 receptor
  • The shuttle mediates uptake of small peptides, antibodies and enzymes to the brain parenchyma, where these cargos can exert their physiologic/ therapeutic action

Krzysztof Wicher, Principal Scientist and Group Leader,

11.20 Strategies to Improve Penetration & Delivery to the Brain

  • Highlighting current data on innovative technological approaches such as chemical modifications and physical disruption for the delivery of therapeutics to the CNS
  • Exploring novel shuttle and trojan approaches for delivery of therapeutics across the brain-blood barrier

Robert Bell, Senior Principal Scientist & Lab Head of
Neurovascular Biology, Pfizer

11.40 Nose to Brain Delivery of Neuroprotective Amnion Derived ST266 Secretome

  • Intranasal delivery is an effective means to deposit large molecular weight proteins to the brain
  • Amnion derived secretome is neuroprotective and anti-inflammatory in optic nerve disease and traumatic injury models

Larry Brown, Executive Vice President R&D, Chief
Scientific Officer, Noveome Biotherapeutics

12.00 Session Q&A; Panel

  • What passive and active mechanisms should be considered during the development and delivery of effective therapeutic interventions to central nervous system targets?
  • How can we best harness innovative methods including chemical modifications, Trojan horse approaches, physical targeting and disruption, nanoparticles,
    ultrasound, and other technologies?
  • What potential opportunities are there to catalyze development of novel treatments that cross the BBB from the preclinical to clinical phase with an emphasis on risks, levers, and potential collaborative efforts among sectors

Krzysztof Wicher, Ossianix
Robert Bell, Pfizer
Larry Brown, Noveome Biotherapeutics

Lunch & Networking

13.30 Progress and Challenges in the Development of Translational in Vivo Disease Modification Assays

  • Reviewing approaches to demonstrate disease modification in preclinical in vivo assays for neurodegenerative disorders
  • Exploring progress and hurdles towards translational assays: keeping the clinical goal in sight
  • Optimizing the application of currently available in vivo assays to exploit their strengths
  • Highlighting Huntington’s disease, ALS, and Parkinson’s disease research

Susan Browne, Director, Teva Pharmaceuticals

13.50 Profiling Specific Cell Populations in Human Brain Tissue to Identify New Targets

  • Demonstrating new insights about healthy and disease states as well as aging through Cerevance’s molecular profiling of neuronal and glial cell nuclei
  • Highlighting how our large-scale approach provides an alternative to relying on animal models, iPS cells and single cell analysis
  • Through comparative analysis, we are identifying potentially new drug targets that are highly selectively expressed in cell populations and circuitry that are most vulnerable in neurodegenerative diseases

Brad Margus, Co-founder & CEO, Cerevance

14.10 Preclinical Electromyography and Muscle Force Recording for Natural History and Disease Modification in Neurodegeneration Models

Laurent Bogdanik, Senior Study Director; Lead Scientist in Neurobiology, The Jackson Laboratory

14.30 Session Q&A; Panel

  • In vitro, in vivo and/or in silico analyses: where should be applying our resources to bridge the translational gap in neurodegenerative research?
  • How can the study of genotype-phenotype interactions be integrated to enhance the translation of modelling systems?
  • Are we able to cost-effectively scale up advanced blood-brain barrier models to make high throughput and or high content screening a reality?
  • Which of these dynamic models most effectively and accurately models the blood-brain barrier to prove selective drug delivery and distribution?
  • What advances have been made using human iPScderived neurons to model key mechanisms of pathology and their applications to stimulate drug discovery?

Susan Browne, Director, Teva Pharmaceuticals

Brad Margus, Co-founder & CEO, Cerevance 

13.30 AAV2-AADC Gene Therapy for Advanced Parkinson’s Disease: Interim Data, Clinical Development Insights

  • Demonstrating the potential for gene therapy to restore lost CNS function by creating an alternative source of the enzyme responsible for the conversion of levodopa to dopamine (AADC)
  • Exploring updated biomarker and clinical interim data readouts from VY-AADC01 clinical studies
  • Discussing unique aspects of clinical development required in “one-and-done” therapies

Brendon Boot, Medical Director, Voyager Therapeutics

13.50 Targeting pre-mRNA splicing with small molecules for treatment of neuromuscular disorders

  • The potential of small molecules for therapeutic targeting of pre-mRNA splicing
  • Targeting alternative splicing of SMN2 exon 7 for treatment of spinal muscular atrophy
  • Targeting alternative splicing of exon 20 in mutant IKBKAP gene for treatment of familial dysautonomia

Nikolai Naryshkin, Executive Director, Biology – Genetic Disorders, PTC Therapeutics, Inc.

14.10 Presentation Title To Be Confirmed

Speaker to be Confirmed

14.30 Session Q&A; Panel:

  • What does it really take for regulatory approval with gene therapy?
  • How many patients, especially in the rare and ultra rare disease do you really need to show convincing efficacy and safety profile (particularly long term safety) in pivotal and supplementary studies?
  • What are the best methods for the in vivo characterization of balancing potency of drug product with delivery and ensuring retention/control of functionality?
  • Are we able to platform gene therapies to leverage across preclinical development programs? What would the challenges be here?

Brendon Boot, Medical Director, Voyager Therapeutics
Nikolai Naryshkin, Executive Director, Biology – Genetic Disorders, PTC Therapeutics, Inc.

Afternoon Refreshments & Networking

Presentation Title To Be Confirmed

Exploring the Utility of Non Human Primate Models in the Study of Neurodegeneration

Evaluating Translatability: Relevance of Rodent & Nonhuman Primate Preclinical Models of Cognitive Impairment to Clinical Drug Development

  • Jay Schneider Professor, Pathology, Anatomy and Cell Biology, Director, Parkinson’s Disease Research Unit, Thomas Jefferson University


  • Reiterating cognitive impairment as a key component of various CNS disorders as well as an important potential adverse effect of medications
  • Reviewing similarities and differences in homology between rodent/human and nonhuman primate/human in relationships between brain organization and cognitive capacity
  • Highlighting advantages and disadvantages of rodent and nonhuman primate models of cognitive dysfunction
  • Demonstrating translational successes/failures of rodent and nonhuman primate cognition models

Translational Value of Non-Human Primate Models of Motor Impairment and Disease Progression in Parkinson’s Disease


  • Demonstrating the value of MPTP-lesioned non-human primates in predicting Phase II efficacy of novel treatments for motor symptoms, and motor side-effects of treatment, in Parkinson’s disease
  • Reviewing the limitations of MPTP-lesioned non-human primate in predicting Phase II efficacy in providing disease modifying benefit in Parkinson’s disease
  • Exploring recent progress and applications of alpha-synuclein-based non-human primate models for evaluating novel approaches to disease modification in Parkinson’s disease

Closing Remarks & Close of Conference Day One

Session Q&A Panel

  • Jay Schneider Professor, Pathology, Anatomy and Cell Biology, Director, Parkinson’s Disease Research Unit, Thomas Jefferson University
  • Jonathan Brotchie Co-Founder and Chairman, Atuka Inc.


  • How are new technologies converging to enhance primate specific neurodegenerative research?
  • Despite advances, how should we seek to overcome the technical challenges in the creation and analysis of transgenic primate models?
  • For which applications should primate models act as a critical tool in the advancement of neurodegenerative research?
  • How do we build the business case for their use, given cost and ethical considerations amongst recent advances in the development of lower order and cellular based models?

Drinks Reception & Scientific Poster Session: Hosted by Transpharmation


After the formal presentations have finished, the learning and networking carries on. The poster session is an informal part of the conference agenda, allowing you to connect with your peers in a relaxed atmosphere and to continue to forge new and existing relationships. During this session, scientific posters will be presented on novel models, biomarkers and validated targets, highlighting innovative mechanisms for potential disease modification of neurodegenerative diseases.