Day One
Wednesday, February 21, 2018
Day Two
Thursday, February 22nd, 2018
08.25 Chairs Welcoming & Opening Remarks
Novel Therapeutic Approaches for Targeting Neuroinflammation & Neurodegeneration
08.30 (Re) Introduction to Microglia as Intrinsic Brain Cells
Synopsis
- 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
08.50 Shedding Light on the Role of TREM2 in Alzheimer’s Disease
Synopsis
- 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
09.10 The Importance of Microtubular Proteins & Cytokines in Neuroplasticity & Neuroinflammation
Synopsis
- 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
09.30 Session Q&A Panel
Synopsis
- 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?
10.00 Speed Networking & Morning Refreshments
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Discovery Track: Novel Approaches to Target & Phenotypic Based Discovery
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Development Track: Therapeutic Strategies for Effectively Crossing the Blood Brain Barrier
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
discovery - 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,
Ossianix
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
12.30 Lunch & Networking
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Discovery Track: Addressing The Need for Integrative Disease Modelling
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Development Track: Assessing the Therapeutic Potential of Gene Therapy for Neurodegenerative Diseases
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.
15.00 Afternoon Refreshments & Networking
16.00 Presentation Title To Be Confirmed
Exploring the Utility of Non Human Primate Models in the Study of Neurodegeneration
16.30 Evaluating Translatability: Relevance of Rodent & Nonhuman Primate Preclinical Models of Cognitive Impairment to Clinical Drug Development
Synopsis
- 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
16.50 Translational Value of Non-Human Primate Models of Motor Impairment and Disease Progression in Parkinson’s Disease
Synopsis
- 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
17.30 Closing Remarks & Close of Conference Day One
17.30 Session Q&A Panel
Synopsis
- 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
Synopsis
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.
