Cambridge Healthtech Institute’s 3rd Annual

CNS Targets and Translational Strategies

Advancing CNS Drug Development

June 18-19 2019

A greater understanding of CNS related disease biology and the emergence of new, improved targets and technologies is bringing renewed interest, excitement and investment into this transformative area of medicine.

Cambridge Healthtech Institute’s CNS Targets and Translational Strategies conference focuses on the key issues of CNS drug development – including CNS target discovery and validation, biomarkers, bridging the preclinical/ clinical translation gap, evaluating the strengths and weaknesses of current preclinical models, challenging “gold-standards”, understanding mechanism of action, dose selection, neuroimaging, neuroinflammation, neuroimmunology, and more. 2019’s meeting will focus on progress being made in the field of CNS drug development rather than historical reflection.

Final Agenda

Stay on to attend Wednesday, June 19 - Thursday, June 20

Blood-Brain Barrier

Tuesday, June 18

7:00 am Registration Open and Morning Coffee


8:00 Chairperson’s Remarks

Dario Doller, PhD, Senior Director, Exploratory Science, SAGE Therapeutics

8:10 Current Challenges and Opportunities in CNS Drug Development

Gopalakrishnan_MuraliMurali Gopalakrishnan, PhD, Senior Director, Head Search & Evaluation Neuroscience, AbbVie

CNS drug development represents an exciting area, particularly for neurodegenerative indications. This presentation details current understanding of CNS-related disease biology, emerging targets, new technologies and the major hurdles facing developers bringing CNS therapies to market.

8:40 KEYNOTE PRESENTATION: Translational Strategies to Optimize Drug Development for Neurodegenerative Disorders

Soares_HollyHolly D. Soares, PhD, Head, Translational Neuroscience, AbbVie

9:10 KEYNOTE PRESENTATION: Lysosomal Dysfunction in Parkinson’s Disease: From Genetics to the Clinic

Sardi_PabloPablo Sardi, PharmD, PhD, R&D Director, Sanofi

This presentation will discuss: clinical, genetic and experimental evidence underlies the relevance of lysosomal dysfunction in Parkinson’s disease (PD); mutations in the lysosomal glucocerebrosidase gene (GBA) accelerate PD progression; First trial has begun testing a GBA pathway modulator in a genetically defined population, and Modulation of the lysosomal pathway may also benefit a larger sporadic patient population.

9:40 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing


10:25 Featured Presentation: Targeting KCC2 for Epilepsy and Other Brain Disorders

Brandon_NickNicholas Brandon, PhD, Chief Scientist, Neuroscience, IMED, AstraZeneca

Deficits in GABAergic inhibition result in the abnormal neuronal activation and synchronization that underlies seizures. However, the molecular mechanisms responsible for transforming a normal brain into an epileptic one remain largely unknown. Here we discuss the growing evidence that 2K+-Cl- cotransporter (KCC2) dysfunction has a central role in the development and severity of the epilepsies.

10:55 Behavior & Biomarker Characterization of the SOD1G93A Model of ALS Following Chronic Dosing with a Brain Penetrant HDAC6 Inhibitor Compound

Niroomand_ShahriarShahriar Niroomand, PhD, Senior Scientist, Neuroscience, Merck Research Laboratories

11:25 Zebrafish Models for Early Research and Development in Neurodegenerative and Neuromuscular Diseases

Arantza Muriana, Co-Founder & CEO, Biobide USA

Zebrafish presents numerous advantageous that make it a powerful animal model in neuroscience research. During this presentation we will review several neurodegenerative and neuromuscular rare disease zebrafish models (ALS, Duchnne, Dravet Syndrome), their application in CNS drug screening, target validation and other early research and development activities.

11:40 Sponsored Presentation (Opportunity Available)

11:55 Transition to Lunch

12:00 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:30 Session Break


1:05 Chairperson’s Remarks

Pablo Sardi, PharmD, PhD, R&D Director, Sanofi

1:10 Targeting Nucleocytoplasmic Transport in Amyotrophic Lateral Sclerosis and Dementia

Freyermuth_FernandeFernande Freyermuth, PhD, Massachusetts General Hospital, Mass General Institute for Neurodegenerative Diseases

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disorder characterized by the degeneration of motor neurons leading to fatal paralysis. Mutations in the FUS gene are responsible for familial cases of ALS, and cytoplasmic mislocalization of the protein is a pathological hallmark in a form of frontotemporal dementia. We determined that FUS mutations lead to defects in the nuclear membrane integrity that can be restored by targeting FUS nuclear cytoplasmic transport.

1:40 The TSC Preclinical Consortium: De-Risking Drug Discovery and Repurposing for Tuberous Sclerosis Complex

Roberds_StevenSteven Roberds, PhD, CSO, Tuberous Sclerosis Alliance

The TSC Preclinical Consortium is a public-private partnership catalyzing development of new treatments for tuberous sclerosis complex. The TS Alliance holds licenses to use specific mouse models at CROs, including rights to test compounds for commercial entities. This has attracted nine pharmaceutical companies to join the consortium, testing 15 proprietary drug candidates. Fifteen additional compounds have been tested by the TS Alliance, with data shared to all consortium members.

2:10 Presentation to be Announced

2:25 Refreshment Break in the Exhibit Hall with Poster Viewing


3:10 New Targets and Biology in Neuroimmunology

Hasson_SamSamuel Hasson, PhD, Senior Scientist, Neuroscience, Amgen

Information coming from human genetic studies has implicated several key pathways in the pathogenesis of Alzheimer’s disease (AD). These include processing of the Amyloid Precursor Protein and activation of the innate immune response. Here we describe efforts to understand the biology of targets in each of these pathways in order to help translate that into potential, disease-modifying therapeutics for the treatment of AD.

3:40 Microglial Immune Checkpoint in Neurodegeneration and Brain Tumors

El_Khoury_JosephJoseph El Khoury, MD, Associate Professor of Medicine, Harvard Medical School

Similar to lymphocytes, microglia, the innate immune cells of the CNS, have several immunological checkpoints that prevent their overreaction to external stimuli. These checkpoints are different from those of lymphocytes. They include Trem2, Cx3cr1/fractalkine and progranulin that keep the inflammatory response in check. Dysregulation of any of these checkpoints occurs in neurodegeneration and affects the response to brain tumors. As with T cell immune checkpoints, microglial immune checkpoints may be important targets for therapy.

4:10 Transition to Keynote


5:20 Welcome Reception in the Exhibit Hall with Poster Viewing

6:35 Find Your Table, Meet Your Moderator

6:40 Breakout Discussion Groups

7:30 Close of Day

Wednesday, June 19

7:00 am Registration Open and Morning Coffee


8:00 Chairperson’s Remarks

Dario Doller, PhD, Senior Director, Exploratory Science, SAGE Therapeutics

8:05 Harnessing the Gut-Brain Axis to Discover Novel CNS Therapeutics

Donabedian_DavidDavid H. Donabedian, PhD, Co-Founder & CEO, Axial Biotherapeutics

In ASD, a human commensal bacterial therapy has shown to correct gut permeability, restore a healthy microbial composition and ameliorate core and non-core behavioral symptoms associated with ASD. In addition, Axial has developed a gut-selective, nonbacterial-based therapy that has shown to improve barrier integrity and ameliorate core and non-core behavioral symptoms associated with ASD. The findings suggest that targeting the gut microbiome may provide a new approach for diagnosing and treating PD and ASD.

8:35 Preclinical Translational Strategies for the Neuroinflammatory Aspects of Neurodegenerative Disease

Levenson_JonathanJonathan Levenson, PhD, Vice President, Translational Biology, Tiaki Therapeutics

Chronic neuroinflammation is a hallmark of Alzheimer’s disease and other neurodegenerative disorders. Tiaki Therapeutics has developed a novel ex vivo platform that faithfully models the neuroinflammatory signature observed in patients afflicted with a neurodegenerative disease. Tiaki has focused on the role of neuroinflammation in Alzheimer’s disease, specifically microglial dysfunction. Tiaki has identified druggable, molecular targets on microglia that when modulated mitigate neuroinflammation and improve neuronal health.

9:05 Using Simoa Ultra-Sensitive Biomarker Detection Technologies to Speed Approval of Therapeutics

David Duffy, PhD, CTO, Quanterix Corporation

We will describe several Simoa ultra-sensitive multiplexed technology platforms which allow biomarkers to be measured at previously undetectable concentrations. We will discuss how they are being used to improve decision making in the drug development process and provide examples of how researchers have used Simoa for ultrasensitive bioanalysis, immunogenicity, and biomarker detection assays. Other benefits of ultra-sensitive measurements, including reduction of matrix effects, single cell analysis, and small sample volume assays will also be illustrated.

9:20 Sponsored Presentation (Opportunity Available)

9:35 Coffee Break in the Exhibit Hall with Poster Viewing


10:20 KEYNOTE PRESENTATION: Translational Opportunities for Brain Proteinopathies, Alzheimer’s Disease and Parkinson’s Disease, through Pathophysiology-Based Imaging and Biofluid Biomarkers

Johan Luthman, PhD, SVP, Neuroscience Clinical Development, Neurology Business Group, Eisai, Inc.

Amyloid pathophysiology-based PET imaging and CSF biofluid biomarkers are now used routinely in major clinical Alzheimer’s disease trials for eligibility screening when studying amyloid-directed therapeutics. Moreover, quantitative amyloid PET imaging is providing important possibilities to conduct clinical proof of principle trials for anti- amyloid investigational therapeutics. Tau PET imaging and CSF tau markers are now emerging as a similar tool for patient identification as well as PoP in studies on tau-directed therapeutics.

10:50 Novel Markers that Significantly Enhance the Prediction of Alexandra’s Disease Progression

Devanarayan_ViswanathViswanath Devanarayan, PhD, Adjunct Professor, University of Illinois, Chicago

The 2018 NIA-AA research framework proposes a ATN classification system with beta-Amyloid deposition (A), pathologic Tau (T), and neurodegeneration (N) for the diagnosis and staging of Alzheimer’s Disease (AD). We use proteomics data from the AD neuroimaging initiative to develop simple cut-point based signatures that reinforces this ATN research framework. When applied to a separate MCI group at baseline, subjects satisfying these signature criteria experience over two-fold faster progression to AD compared to signatures based on the ATN research framework.

11:20 Electrophysiological Biomarkers and Translational Animal Models

Stephen Morairty, PhD, Senior Director, Center for Neuroscience, SRI Biosciences

The use of behavioral assays in rodents has proven to provide, at best, mixed results for predicting effects in the clinic leading to a very low success rate for new CNS active. Since the electroencephalogram (EEG) is used in humans, we have studied EEG in rodents and non-human primates in combination with behavioral tests in the pursuit of biomarkers that are predictive across species.

11:50 Transition to Lunch

12:00 pm Bridging Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:30 Transition to Plenary


2:20 Dessert and Coffee Break in the Exhibit Hall with Poster Viewing

3:05 Close of Conference

Stay on to attend Wednesday, June 19 - Thursday, June 20

Blood-Brain Barrier

* The program is subject to change without notice, due to unforeseen reason.

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