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CNS Diseases Summit

2018-01-242018-08-142018-06-25
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The 2018 full agenda is currently in process.

Please come back and visit this page for updates.

BELOW IS THE FULL AGENDA FROM 2017.

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Day 1 - Monday, September 11, 2017
7:00
Registration & Continental Breakfast
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8:15
Welcome & Opening Remarks by Dr. Satish Medicetty, President, GTCbio
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10:00
Morning Networking Break
1
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Neurodegenerative Diseases Research & Development 2017
Experimental Therapeutics & Approaches in Alzheimer’s Disease & Related Dementias
Moderator: Katrina Paumier, Washington University School of Medicine
10:45
Chaperone Alterations in AD as a Target of Intervention
 
Gabriela Chiosis
Gabriela Chiosis
Professor
MSKCC
About Speaker: Dr. Gabriela Chiosis received her graduate training at Columbia University in New York and joined Memorial Sloan Kettering Cancer Center in 1998, first as a fellow and, since 2005, as faculty. She has authored over 130 scientific articles which were ... Read Full Bio 
 
 
Gabriela Chiosis
Gabriela Chiosis
Professor
MSKCC
 
About Speaker:

Dr. Gabriela Chiosis received her graduate training at Columbia University in New York and joined Memorial Sloan Kettering Cancer Center in 1998, first as a fellow and, since 2005, as faculty. She has authored over 130 scientific articles which were published by virtually all well respected scientific and medical journals, holds over 190 patents and patent applications which are related to the discovery of novel compounds as therapeutic agents or diagnostics in human medicine, is serving as a reviewer for over 50 well-known scientific and medical magazines and on several scientific panels. She is also a co-founder of Samus Therapeutics Inc, and also serves on its Board of Managers. Novel compounds and diagnostics discovered by her lab are the platform for the development of inhibitors currently in clinical evaluation in Alzheimer’s disease and cancer patients.

 
Abstract: An increasing number of genetic studi...Read More 

An increasing number of genetic studies suggest that the pathogenesis of certain neurodegenerative diseases and cancer have commonalities in that they share certain genes, pathways, and mechanisms. Nonetheless, the links between molecular mechanisms that cause and are associated with cancer and neurodegenerative diseases, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD), have thus far remained largely unexplored. This talk aims to present and discuss key mechanisms of disease propagation found under both conditions. Will also discuss how these commonalities can lead to potential new strategies for therapeutic intervention in AD and PD.

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CNS Partnering & Deal-Making 2017
What’s New in CNS – Targets, Models, Assays, etc.
Moderator: Linda Hedley, Hedley Consulting
10:45
Transforming AD Therapy Development: from Targets to Trials
 
Suzana  Petanceska
Suzana Petanceska
Senior Advisor for Strategic Development and Partnerships
National Institute on Aging, NIH
About Speaker: Dr. Petanceska is a senior advisor for strategic development and partnerships and a program director for systems biology and systems pharmacology at the Division of Neuroscience of the National Institute on Aging (NIA).  During her tenure at the NIA... Read Full Bio 
 
 
Suzana  Petanceska
Suzana Petanceska
Senior Advisor for Strategic Development and Partnerships
National Institute on Aging, NIH
 
About Speaker:

Dr. Petanceska is a senior advisor for strategic development and partnerships and a program director for systems biology and systems pharmacology at the Division of Neuroscience of the National Institute on Aging (NIA).  During her tenure at the NIA she has been overseeing and developing a number of research portfolios and innovative programs in basic and translational research for Alzheimer’s disease (AD).  Her recent program development efforts have been focused on developing systems biology and systems pharmacology capabilities for AD research and drug development within an open science framework.    Dr. Petanceska was instrumental for the development of NIA’s AD Translational Research Program and leads NIA’s open-science, systems biology programs for target and biomarker discovery: the AMP-AD Target Discovery and Preclinical Validation Project and the M2OVE-AD Consortium

 

Dr Petanceska holds a Ph.D. in Pharmacology from New York University.  Following her postdoctoral training at Rockefeller University and Cornell University, she established her independent research career at the Nathan Kline Institute in Orangeburg, N.Y., and joined the faculty of New York University Medical Center. Her research focused on the role of disrupted sterol metabolism in Alzheimer’s disease pathogenesis and on the mechanisms by which estrogens and cholesterol-lowering drugs exert neuroprotection.

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11:10
Targeting the Neurovascular Unit in Neurodegeneration
 
Robert Bell
Robert Bell
Associate Research Fellow, Gene Therapy Translational Science, Rare Disease Research Unit
Pfizer
About Speaker: Robert “Bob” received a PhD in Pathology studying the role of cerebral vascular dysfunction in Alzheimer’s disease under the supervision of Dr. Berislav Zlokovic at the University of Rochester. He then completed an AHA funded postdoctoral fello... Read Full Bio 
 
 
Robert Bell
Robert Bell
Associate Research Fellow, Gene Therapy Translational Science, Rare Disease Research Unit
Pfizer
 
About Speaker:

Robert “Bob” received a PhD in Pathology studying the role of cerebral vascular dysfunction in Alzheimer’s disease under the supervision of Dr. Berislav Zlokovic at the University of Rochester. He then completed an AHA funded postdoctoral fellowship in cardiovascular biology in the Laboratory of Dr. Joseph Miano and held a Research Assistant Professor position in the Department of Neurosurgery at the University of Rochester before joining Pfizer in 2012. Bob’s research has helped to elucidate cellular and molecular mechanisms that regulate neurovascular functioning in health and disease.  As the Neurovascular and BBB Biology Lab head at Pfizer, Bob led a group focused on identifying neurovascular targets for the treatment of CNS disorders and the delivery of therapeutics across the blood-brain barrier. Bob has recently taken on a new role at Pfizer in the Rare Disease Research Unit, Gene Therapy Translational Sciences department where he leads a laboratory group focused on the development of novel gene therapy clinical candidates for peripheral and CNS genetic disorders.

 

 
Abstract: Highly coordinated molecular and cell...Read More 

Highly coordinated molecular and cellular interactions across the neurovascular unit, comprised of vascular endothelium, smooth muscle, pericytes, extracellular matrix, glia and neurons, are essential for CNS health. The neurovascular unit controls cerebral blood flow (CBF) dynamics, blood-brain barrier (BBB) integrity, peripheral immune cell infiltration into the CNS and drug transport. I will highlight neurovascular anatomy, dysfunction and biomarkers in the context of dementia and neurodegenerative conditions. A key emphasis will be placed on how neurovascular dysfunction can directly contribute to key aspects of neuroinflammation, neuronal injury and impaired brain energetics. Potential therapeutic targets and pathways to directly improve neurovascular function or indirectly impact downstream pathological processes will be highlighted. In summary, the neurovascular unit plays a key role in neurodegenerative disease processes and presents unique opportunities for novel therapeutics to improve CNS health.

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11:10
ANAVEX 2-73, a Sigma-1 Receptor Agonist, Targeting Neurodegenerative and Neurodevelopmental Diseases
 
Daniel Klamer
Daniel Klamer
​Vice President, Business Development​ & Scientific Strategy​
Anavex Life Sciences
About Speaker: Dr Daniel Klamer, Vice President of Business Development and Scientific Strategy of Anavex, has more than 15 years of experience in neuroscience and the orphan disease space, with acquisition, partnering and R&D experience in Europe and the USA. ... Read Full Bio 
 
 
Daniel Klamer
Daniel Klamer
​Vice President, Business Development​ & Scientific Strategy​
Anavex Life Sciences
 
About Speaker:

Dr Daniel Klamer, Vice President of Business Development and Scientific Strategy of Anavex, has more than 15 years of experience in neuroscience and the orphan disease space, with acquisition, partnering and R&D experience in Europe and the USA. Prior to Anavex he worked at e.g. Retrophin and NeuroSearch. At NeuroSearch, Dr Klamer led and evaluated multiple discovery-phase neuropharmacological research products with an emphasis on strategic evaluation of preclinical and clinical development and commercialization.

Dr. Klamer earned his PhD in Pharmacology at The Sahlgrenska Academy at University of Gothenburg, Sweden, his MBA at Fordham Gabelli School of Business, and his Post-Doctoral training at the Department of Psychiatry, Yale University School of Medicine. In addition, Dr. Klamer holds a position as an Associate Professor at the Department of Pharmacology, at The Sahlgrenska Academy at the University of Gothenburg.

 
Abstract: ...Read More 

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded clinical stage biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders. Anavex’s lead candidate, ANAVEX 2-73, is an orally available compound that restores cellular homeostasis by targeting Sigma-1 and muscarinic receptors. ANAVEX 2-73, has demonstrated good safety, bioavailability, and tolerability in Phase 1 and Phase 2 clinical trials. In addition, data from an ongoing Phase 2a clinical trial in Alzheimer’s Disease demonstrates dose dependent cognitive improvements. The compound also exhibits anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat a broad range of CNS disorders.

ANAVEX 2-73 significantly improves multiple behavioral phenotypes in the Rett Syndrome mouse model in a dose-related manner. Based on these data, Anavex Life Sciences will start a U.S. multicenter Phase 2 clinical trial of ANAVEX 2-73 for the treatment of Rett Syndrome with the support of Rettsyndrome.org.

The Michael J. Fox Foundation for Parkinson’s Research awarded Anavex Life Sciences a research grant to develop ANAVEX 2-73 for the treatment of Parkinson’s Disease. Ongoing preclinical research in this therapeutic area demonstrates that ANAVEX 2-73 ameliorates motor deficits in spontaneous rotational activity and forelimb use asymmetry, and exerts significant neurorestorative effects on the damaged nigrostriatal dopamine system.

The scientific rationale, preclinical data, and clinical development path of ANAVEX 2-73 as a novel treatment strategy for Rett Syndrome, Parkinson’s Disease, and Multiple Sclerosis will be discussed in more detail. This presentation will explore the links between preclinical findings and clinical trial development trajectory in our ongoing translational research efforts.

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11:35
Clearance of Beta-Amyloid Is Facilitated By Apolipoprotein E and Circulating High-Density Lipoproteins in Bioengineered Human Vessels
 
Cheryl  Wellington
Cheryl Wellington
Clinical Professor, Djavad Mowafaghian Centre for Brain Health
University of British Columbia
About Speaker: Dr. Wellington, Professor at the University of British Columbia, integrates research on genetic and environmental risk factors for dementia, with particular attention to lipoprotein metabolism, cerebrovascular dysfunction, and traumatic brain injury.... Read Full Bio 
 
 
Cheryl  Wellington
Cheryl Wellington
Clinical Professor, Djavad Mowafaghian Centre for Brain Health
University of British Columbia
 
About Speaker:

Dr. Wellington, Professor at the University of British Columbia, integrates research on genetic and environmental risk factors for dementia, with particular attention to lipoprotein metabolism, cerebrovascular dysfunction, and traumatic brain injury. Over the past 16 years, her laboratory has used a variety of genetic and pharmacological approaches to show that the amount of fats carried on apoE regulates clearance of beta-amyloid peptides, which are neurotoxic species that accumulate as amyloid in the AD brain. She has also demonstrated that apoE plays additional critical roles in diffuse axonal injury after closed-head traumatic brain injury TBI and in endothelial function. The newest area of research in Dr. Wellington’s laboratory explores tissue engineered human cerebral blood vessels as model to investigate lipoprotein mediated beta-amyloid clearance across cerebrovasculature.

 

 
Abstract: In the last decade, the complex inter...Read More 

In the last decade, the complex interrelationship between cerebrovascular dysfunction and AD is increasingly appreciated. Established vascular risk factors for AD include hypertension, dyslipidemia, and type II diabetes, exacerbate cognitive decline and cerebral small vessel diseases, including cerebral amyloid angiopathy (CAA) and microvascular degeneration, are comorbidities in 60-90% of AD autopsy cases. Interestingly whereas high-density lipoproteins (HDL) plasma level positively correlates with cognitive function in AD patients, its effects on human cerebrovascular integrity remain poorly understood. In the present study we developed a novel human based in vitro model of the vasculature to study the role of HDL on the cerebrovascular function in the context of AD. Primary human endothelial (EC) and smooth muscle cells (SMC) were cultivated in the absence (bipartite) or presence (tripartite) of human astrocytes (apoE3/E3) using a scaffold-directed dynamic, pulsatile flow bioreactor system. Histological analysis demonstrated the formation of a dense tissue composed of a tight layer of ECs in the lumen and several layers of SMCs for the bipartite tissue and several layers of astrocytes on the ablumen for the tripartite mimicking the structure of pial, leptomeningial and penetrating cerebral arteries. We have injected Aß directly into the tissue chamber to mimic amyloid coming from the brain side of the vessel. To further analyze the role of lipoproteins on vascular amyloid accumulation we circulated HDL isolated form healthy young individuals in the lumen and/or added apoE with Aß. Histological staining and ELISA quantifications confirmed the accumulation of Aß within our vessel equivalent time and dose dependently. We further show that lipoproteins on both sides of the vessel synergize to facilitate Aß transport across the human cerebral vessel. Importantly, our data also show that lipoproteins facilitate Aß42 transport more efficiently than Aß40, which is consistent with human data showing that Aß40 is the primary species that accumulates in CAA. Moreover, apoE4 is less effective than apoE2 in promoting Aß transport, which is also consistent with the well-established role of apoE4 in Aß deposition in AD. Taken together, our results establish the utility of human engineered cerebral vessels as a highly innovative in vitro platform to study key mechanistic questions relevant to AD.

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11:35
Modulating Microglial-mediated Neuro-inflammation - NTRX-07 as a Novel Treatment for Pain and Neurodegenerative Disease
 
Joseph Foss
Joseph Foss
Founder
NeuroTherapia
About Speaker: Dr. Foss is a founder of NeuroTherapia and Staff at the Cleveland Clinic in the Anesthesiology Institute. . He received  training in clinical pharmacology at the University of Chicago and led the development of methylnaltrexone, a peripherally activ... Read Full Bio 
 
 
Joseph Foss
Joseph Foss
Founder
NeuroTherapia
 
About Speaker:

Dr. Foss is a founder of NeuroTherapia and Staff at the Cleveland Clinic in the Anesthesiology Institute. . He received  training in clinical pharmacology at the University of Chicago and led the development of methylnaltrexone, a peripherally active opioid antagonist. He later worked f or Adolor, a small biotech company, which was developing alvimopan, another peripherally acting opioid antagonist, which also has been approved for clinical use. He was also responsible for the translational program there that was developing novel analgesics.He has been working with Dr. Naguib, co-founder of NeuroTherapia, to support to the development program for NTRX-007, a novel CB2 agonist, for the treatment of pain and neurodegenerative diseases.

 
Abstract:  Neuroinflammation is now identi...Read More 

 Neuroinflammation is now identified as a common feature in a wide spectrum of diseases and conditions such as Alzheimer’s Disease, ALS, multiple sclerosis, Parkinson’s disease, stroke, traumatic brain injury, neuropathic pain and chemotherapy induced peripheral neuropathy. While the pathology of each of these conditions varies, activation of the microglial cells initiate an inflammatory cascade characterized by increases in inflammatory mediators, appears a common pathway in a broad range of neurologic disorders. The upregulation of cannabinoid type 2 (CB2) receptors on the microglia has been shown to have important regulatory effects on neuroinflammation. NTRX-07 is a CB2 receptor agonist, with distinct functional activity, that has been shown in a variety of models (specifically Alzheimer’s disease, neuropathic pain induced by varying types of injury and chemotherapy-induced peripheral neuropathy) to be effective in ameliorating microglial activation, neuroinflammation and restoring function.

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12:00
Attenuating Hippocampal Overactivity as a Therapeutic Strategy for Mild Cognitive Impairment Due to Alzheimer’s Disease
 
Sharon  Rosenzweig-Lipson
Sharon Rosenzweig-Lipson
Vice President of R&D
Agenebio
About Speaker: Sharon Rosenzweig-Lipson, Ph.D., is President of IVS Pharma Consulting. In 2011, Dr. Rosenzweig-Lipson founded IVS Pharma Consulting to bring her expertise in screening strategies, in vivo models, translation and early clinical development strategy t... Read Full Bio 
 
 
Sharon  Rosenzweig-Lipson
Sharon Rosenzweig-Lipson
Vice President of R&D
Agenebio
 
About Speaker:

Sharon Rosenzweig-Lipson, Ph.D., is President of IVS Pharma Consulting. In 2011, Dr. Rosenzweig-Lipson founded IVS Pharma Consulting to bring her expertise in screening strategies, in vivo models, translation and early clinical development strategy to the neuroscience scientific community in pharma, biotech and academia. She has over 20 years experience developing compounds for psychiatric and neurologic indications in the pharmaceutical industry. She has successfully led teams from the earliest exploratory studies through to Phase II Proof of Concept Trials. Prior to her current positions, Dr. Rosenzweig-Lipson held the roles of Head of Translational Neuroscience and In Vivo Head of Psychiatry at Wyeth Research.   Dr. Rosenzweig-Lipson received her B.A. in Biological Basis of Behavior from the University of Pennsylvania and her Ph.D. in Behavioral Neuroscience from Harvard University.

 
Abstract: There is strong support from preclini...Read More 

There is strong support from preclinical AD models and human patients, particularly in early stages of AD, that neuronal circuits in the hippocampus become excessively active contributing to neuronal pathology and brain dysfunction (Bero et al., 2011; Busche et al., 2012; 2015; Ewers et al. 2011; Varga et al., 2014; Hall et al. 2015; Wu et al. 2016). The notion that AD pathology contributes to the augmentation of hippocampal hyperactivity is supported by the clinical finding that hyperactivity is most pronounced in the MCI clinical/cognitive phase in patients with amyloid positive PET imaging compared to those who are amyloid negative (Huijbers et al., 2015). Further, AD pathophysiological pathways involving both amyloid and tau have been functionally linked to excessive neural activity, with heightened neural activity causing dysfunction and progressive spread of pathology in the disease from an origin in the medial temporal lobe/hippocampus (Busche et al. 2015; Wu et al. 2016; Yuan and Grutzendler, 2016; Leal et al., 2017).  Substantial clinical evidence indicates that hippocampal overactivity longitudinally predicts subsequent cognitive decline/conversion to a dementia diagnosis (Dickerson et al., 2005; Sperling et al, 2007; Miller et al., 2008) and is significantly correlated with the extent of neuronal injury affecting the brain (Putcha et al., 2011). The evidence that hippocampal overactivity is a potential driver of neurodegeneration during prodromal AD supports the reduction of overactivity as a novel approach to the treatment of patients in this phase of disease with potential to delay/slow progression (Haberman et al., 2017). 

 

This talk will highlight two novel approaches (AGB101 – low dose levetiracetam and GABA-A a5 positive allosteric modulators) to reducing hippocampal overactivity at differing stages of therapeutic development.   Recent preclinical and Phase 2 clinical studies using low doses of the atypical antiepileptic levetiracetam have supported the concept that reduction of hippocampal overactivity may be therapeutically beneficial (Bakker et al., 2012, 2015). Ranging from research on age-associated memory impairment in rodents to clinical studies in patients with amnestic MCI, beneficial effects on key circuits in the medial temporal lobe/hippocampus and on memory performance have been demonstrated by treatment at low doses of levetiracetam that reduce hippocampal overactivity (Bakker et al., 2012; 2015; Sanchez et al., 2012; Koh et al., 2013; Shi et al., 2013).  AGB101 (220 mg; low dose levetiracetam) is poised to enter Phase III clinical development for the treatment of aMCI due to AD (HOPE4MCI) trial.

 

The localization and functional properties of GABA-A a5 receptors are ideal for control of the condition of hippocampal overactivity in MCI due to AD (Sur et al., 1998; Glykys and Mody, 2006; Hörtnagl et al., 2013).  Across species, the highest expression occurs in the hippocampal formation (Hörtnagl et al., 2013), where those receptors mediate tonic inhibition of hippocampal neurons (Glykys and Mody, 2006). Reduction in GABA-A a5 receptor subunits in knockout mice disrupts memory for location of objects in mice (Prut et al., 2010).  Even more specifically, knockout of GABA-A a5 receptor subunits in the dentate gyrus in the mouse results in impairments in cognitive tasks characterized by high interference, without any deficiencies in low-interference tasks, suggesting specific impairment of pattern separation, which is an essential computational function for episodic memory (Engin et al., 2015). Pattern separation impairments begin in the elderly and are exacerbated during MCI (Stark et al. 2013) and the dentate gyrus plays an important role in mediating this process (Berron et al., 2016). Preclinically, GABA-A a5 PAMs improve memory function in rats with age-associated memory impairments (Koh et al., 2013).

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12:00
Translational Neuroscience for Developing CNS Targeted Therapeutics: Electrophysiological Biomarkers and Translational Animal Models
 
Stephen Moriarty
Stephen Moriarty
Director, Neurosciences
SRI International
About Speaker: Stephen Morairty received his Ph.D. in Neuroscience from the University of California, Los Angeles where his research focused on homeostatic regulation of sleep.  He joined SRI in 1999 following a posdoctoral position at Harvard University in the la... Read Full Bio 
 
 
Stephen Moriarty
Stephen Moriarty
Director, Neurosciences
SRI International
 
About Speaker:

Stephen Morairty received his Ph.D. in Neuroscience from the University of California, Los Angeles where his research focused on homeostatic regulation of sleep.  He joined SRI in 1999 following a posdoctoral position at Harvard University in the laboratories of Dr. Robert Greene and Dr. Robert McCarley.  Current research focuses on the search for translational electrophysiological biomarkers for therapeutic efficacy in rodents and non-human primates.  Using the electroencephalogram (EEG), Dr. Morairty investigates electrophysiological changes following pharmacological manipulations and that are associated with disease, disease progression and cognitive processes.  Recent investigations study the relationship between behavior, EEG patterns and neurodegenerative diseases. Quantitative analysis of the EEG (qEEG) provides a rich data set that shows measurable changes prior to the onset of behavioral changes. His hope is to discern reliable EEG biomarkers for the presence and progression of disease.

 

 
Abstract: For CNS indication, the use of behavi...Read More 

For CNS indication, 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 compounds making it to market. While behavioral outputs from rodents can be difficult to translate to humans, the basic neural networks underlying behavior are remarkably similar between rodent, non-human primates (NHPs) and humans. Importantly, the basic neurophysiological properties defining network function are the same across these species. Thus, electrophysiology is the most translational CNS biomarker know to date. Since the electroencephalogram (EEG) is used in humans, we study EEG in rodents and NHPs in combination with behavioral tests in the pursuit of biomarkers that are predictive across species. Some of the paradigms we investigate are:

  • Electrophysiological measures across the sleep/wake cycle
  • Resting state quantitative EEG (qEEG) profiles
  • Evoked response potentials (ERPs) and sensory gating paradigms such as mismatched negativity (MMN) an auditory odd-ball task.
  • Combining ephys/ERP analysis and behavioral performance during cognitive tasks in NHPs such as during the continuous performance task (CPT; i.e. sustained attention) and the delayed match to sample task (DMTS; i.e. working memory).
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12:25
Lunch Workshop Presentation
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Neurodegenerative Diseases Research & Development 2017
Advances in Parkinson’s Disease Research & Therapeutics
Moderator: Arti Gaur, Dartmouth Geisel School of Medicine
1:55
Targeting Glucocerebrosidase for Parkinson’s Disease
 
Gayathri Ramaswamy
Gayathri Ramaswamy
Lab Head, Internal Medicine Research Unit
Pfizer
About Speaker: Dr. Gayathri Ramaswamy is the head of Lipids and Lysosomes lab and Senior Principal Scientist in the Internal Medicine Research Unit at Pfizer Inc. Dr. Ramaswamy obtained her Ph.D. from St. Jude Children’s Research Hospital, University of Tennessee... Read Full Bio 
 
 
Gayathri Ramaswamy
Gayathri Ramaswamy
Lab Head, Internal Medicine Research Unit
Pfizer
 
About Speaker:

Dr. Gayathri Ramaswamy is the head of Lipids and Lysosomes lab and Senior Principal Scientist in the Internal Medicine Research Unit at Pfizer Inc. Dr. Ramaswamy obtained her Ph.D. from St. Jude Children’s Research Hospital, University of Tennessee (Memphis) and did her post-doctoral training at Gladstone Institute of Neurological Diseases, University of California (San Francisco). She has more than 18 years of experience in lipid metabolism and is considered an expert in apolipoprotein E (apoE) and its role in Alzheimer’s disease. Dr. Ramaswamy has made seminal contributions to understanding the underlying mechanism(s) by which apoE4 contributes to AD. Additionally, she is an expert in small molecule phenotypic screen deconvolution, molecular biology and cell biology. She has also developed reagents for identifying bio-threat agents and for evaluating target gene expression. She has co-authored several peer-reviewed publications. Currently, Dr. Ramaswamy is leading drug discovery programs for treating Alzheimer’s disease and Parkinson’s disease respectively. Areas of research in her laboratory include understanding the underlying mechanisms that regulate apoE in the brain, mechanistic role of apoE4 in AD, and interrogating the role of lipids and lysosome dysfunction in neurodegenerative diseases.

 
Abstract: ...Read More 

Glucocerebrosidase 1 (GBA1) is a lysosomal hydrolase that breaks down the glycolipid, glucocerebroside into ceramide and glucose. Deficiency in GBA1 caused by mutations in the GBA1 gene leads to accumulation of the glycolipid substrate in lysosomes in Gaucher’s disease, a rare lysosomal storage disorder. More recently, mutations in GBA1 have been found to be associated with increased risk and reduced age of onset of Parkinson’s disease (PD). Furthermore, these mutations lead to a decrease in GBA1 enzyme activity and have been found to be associated with increased alpha-synuclein accumulation in brains from PD patients. Given the compelling genetic association and underlying pathophysiology, targeting GBA1 seems to be an attractive strategy for treating PD. This presentation will address the following:
1. What is the association of GBA1 to PD?
a. Present data on the genetic link.
b. Discuss mechanistic link to the disease.
2. What are the potential strategies for targeting GBA1 for treating PD?

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CNS Partnering & Deal-Making 2017
Industry Partnering with Academia & CROs to Advance Research
Moderator: Nadim Shohdy, New York University
1:55
Tango, Waltz or Milonga: Dancing to the Same Tune is Key to Successful Academia-Industry Partnerships
 
Parag Mehta
Parag Mehta
Chief Executive Officer
Aveta Biomics
About Speaker: Parag G. Mehta, PhD, is the Co-founder and CEO of Aveta Biomics, a clinical stage pharmaceutical company based in Bedford, MA.    Parag is a serial entrepreneur with a track record of commercializing novel chemistry based innovations. He has... Read Full Bio 
 
 
Parag Mehta
Parag Mehta
Chief Executive Officer
Aveta Biomics
 
About Speaker:

Parag G. Mehta, PhD, is the Co-founder and CEO of Aveta Biomics, a clinical stage pharmaceutical company based in Bedford, MA. 

 

Parag is a serial entrepreneur with a track record of commercializing novel chemistry based innovations. He has strong background in biotechnology, organic, macromolecular and biochemistry and material science. His experience encompasses product R&D, manufacturing, global business development, strategic alliances, and capitalization of companies through public and private financing. 

 

At Aveta Biomics, Parag played a key role in developing robust pipeline of three drug candidates and gaining IND approval for company’s first-in-class multi-targeted, polypharmaceutical drug. He helped establish three academic partnerships and two industrial partnerships encompassing pre-clinical research & development and a clinical trial.

 

Parag has been active in Boston area entrepreneurial community mentoring many entrepreneurs. He has authored/co-authored twenty-six scientific papers and is inventor/co-inventor of 18 patents. Parag received his PhD in Chemistry from Rensselaer Polytechnic Institute, Troy, NY and MSc in Chemistry from Indian Institute of Technology, Mumbai, India.

 

 
Abstract: The discovery and development of brea...Read More 

The discovery and development of breakthrough therapies will require collaboration among the best minds.  Highly effective alliances and partnerships, therefore, are critical to harnessing and executing the best ideas. The most common models of partnerships are transactional in nature and rest largely on asymmetric economic incentives and risks between the partners. These arrangements often fail to account for the most basic requirements needed for the successful partnerships.  Not unlike highly interdependent dance forms, successful partnerships require the development of well-aligned goals, passion for the purpose, the flexibility of approach, the empowerment of everyone in the participating organizations to contribute to development efforts and ultimately the sharing of rewards. We will share examples from the vantage point of early stage companies where high-performance partnerships are established, based on above principles, to fast track the drug development efforts.

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2:20
The Gut-brain Inflammation Axis in Parkinsons Disease
 
Malu Tansey
Malu Tansey
Professor, Physiology
Emory University
About Speaker: ... Read Full Bio 
 
 
Malu Tansey
Malu Tansey
Professor, Physiology
Emory University
 
About Speaker:
 
Abstract: Parkinson’s disease (PD) is a p...Read More 

Parkinson’s disease (PD) is a progressive disorder that currently is not diagnosed until characteristic motor impairments appear, at which time extensive, irreversible neurodegeneration has already occurred. An emerging paradigm of PD suggests that an extended prodromal period which includes gastrointestinal (GI) disease manifestations could present the opportunity for earlier diagnosis and intervention. We predicted that evaluation of intestinal inflammatory mediators could provide mechanistic information about PD-related GI dysfunction and possibly identify valuable early-stage biomarkers.

We observed trends for PD-associated increases in levels of immune and angiogenesis factors in stool and significant indicators of inflammation in colonic biopsies from PD patients. PD patients reported anxiety, sleep disorders, and digestive problems more frequently than controls, and we detected associations between digestive problems and factors related to T cell recruitment. Interestingly, household controls reported digestive problems more frequently than non-household controls and exhibited similar tissue inflammatory markers. A history of smoking, known to affect PD risk, was associated with reductions in levels of multiple stool analytes.

Our findings suggest that intestinal inflammation persists in PD, is associated with digestive problems and other non-motor manifestations of the disease, and could act as a key driver of neurodegeneration.

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2:20
A Community-based Approach for Systems Biology Based AD Target Discovery
 
Lara  Mangravite
Lara Mangravite
President
Sage Bionetworks
About Speaker: Lara Mangravite, PhD is President of Sage Bionetworks. This organization is focused on the development and implementation of practices for large-scale collaborative biomedical research. Previously, Dr. Mangravite served as Director of the Systems Bio... Read Full Bio 
 
 
Lara  Mangravite
Lara Mangravite
President
Sage Bionetworks
 
About Speaker:

Lara Mangravite, PhD is President of Sage Bionetworks. This organization is focused on the development and implementation of practices for large-scale collaborative biomedical research. Previously, Dr. Mangravite served as Director of the Systems Biology research group at Sage Bionetworks where she focused on the application of collaborative approaches to advance understanding of disease biology and treatment outcomes at a systems level with the overriding goal of improving clinical care. Dr. Mangravite obtained a BS in Physics from the Pennsylvania State University and a PhD in Pharmaceutical Chemistry from the University of California, San Francisco. She completed a postdoctoral fellowship in cardiovascular pharmacogenomics at the Children’s Hospital Oakland Research Institute.

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2:45
Targeting Autophagy and Neuroinflammatory Mechanisms to Modulate Alpha-Synuclein for the Treatment of Parkinson's Disease
 
Errol  De Souza
Errol De Souza
President & Chief Executive Officer
Neuropore Therapies
About Speaker: Dr. De Souza is currently president and Chief Executive Officer of Neuropore Therapies a biopharmaceutical company developing disease modifying treatment for Parkinson’s Disease, Alzheimer’s Disease and other neurodegenerative disorders by ta... Read Full Bio 
 
 
Errol  De Souza
Errol De Souza
President & Chief Executive Officer
Neuropore Therapies
 
About Speaker:

Dr. De Souza is currently president and Chief Executive Officer of Neuropore Therapies a biopharmaceutical company developing disease modifying treatment for Parkinson’s Disease, Alzheimer’s Disease and other neurodegenerative disorders by targeting molecular mechanisms of autophagy and neuro-inflammation to facilitate clearance of misfolded proteins. He has substantial experience as an executive in the biopharmaceutical industry, having founded companies (Neurocrine Biosciences, Nasdaq: NBIX) and served as President and Chief Executive Officer of several public (Biodel, Nasdaq: BIOD; Synaptic Pharmaceutical Corp., Nasdaq: SNAP) and private (Archemix) biotech companies.

Dr. De Souza has raised hundreds of million dollars in capital in private (venture) and public sectors. Also, he has been involved in taking companies public (Neurocrine Biosciences IPO), executed M&A deals including Synaptic Pharmaceuticals sale to H. Lundbeck A/S, and has served in a number of high-ranking R&D roles, including Senior Vice President and U.S. head of R&D for Aventis (now Sanofi; 1998-2002), co-founder and Executive Vice President of Research and Development at Neurocrine Biosciences (1992-1998), and Head of CNS Diseases Research at DuPont Merck (1990 – 1992). He has extensive Board experience and serves or has served on the Board of Directors of several private companies and public companies.

Dr. De Souza received his B.A in physiology and his Ph.D. in neuroendocrinology from the University of Toronto and he received his postdoctoral fellowship in neuroscience from The Johns Hopkins University School of Medicine.

 
Abstract: ...Read More 

Numerous lines of evidence derived from human genetic studies, patient pathology and extensive nonclinical research in animal models have implicated the cellular accumulation of misfolded and aggregated forms of the synaptic protein alpha-synuclein (ASYN) in the pathogenesis of Parkinson’s disease (PD). These aggregated proteins disrupt cellular integrity and evoke inflammatory responses leading to cell death and functional decline in the patient. In some rare familial cases of PD, the underlying cause of the disease is an over production of the protein. However, for most patients, it is more likely that failure of cellular housekeeping mechanisms responsible for degrading these aberrant proteins (referred to as autophagy) is the underlying driver of disease pathology. Recent findings have revealed tight reciprocal relationships between autophagy and neuroinflammation such that not only do failures in autophagy lead to protein accumulation and neuroinflammation, but also that ASYN-evoked inflammation leads to failures of neuronal autophagy thus resulting in a self-propagating pathological process. The presentation will provide an overview of the tight link between neuroinflammation, autophagy and cell pathology and focus on specific drug discovery targets for the development of small molecule, disease-arresting therapies for the treatment of Parkinson’s disease and related disorders.

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3:10
In Vivo Metabolism of Alpha-synuclein in Human Central Nervous System
 
Katrina Paumier
Katrina Paumier
Assistant Professor of Neurology
Washington University School of Medicine
About Speaker: Dr. Paumier received her undergraduate training in psychology at the University of Illinois Champaign-Urbana and her PhD in neuroscience from the University of Cincinnati. She then completed her postdoctoral training in the laboratories of Dr. John D... Read Full Bio 
 
 
Katrina Paumier
Katrina Paumier
Assistant Professor of Neurology
Washington University School of Medicine
 
About Speaker:

Dr. Paumier received her undergraduate training in psychology at the University of Illinois Champaign-Urbana and her PhD in neuroscience from the University of Cincinnati. She then completed her postdoctoral training in the laboratories of Dr. John Dunlop and Dr. Warren Hirst at Pfizer Inc, where she focused on autophagy enhancers for Parkinson’s disease. She was previously an Assistant Professor on the neuroscience faculty at Michigan State University where she focused on treatment strategies for Parkinson’s disease. Dr. Paumier joined the Bateman team in 2015 and is actively involved in several research activities related to misfolded proteins involved in neurodegeneration including alpha-synuclein and amyloid-beta. At present, Dr. Paumier contributes to several studies including: 1) Development of a stable isotope labeling kinetic (SILK) method to measure the turnover rate of alpha-synuclein in human CSF; 2) Investigation of genetic protective factors against Alzheimer's disease; 3) Examination of effects of ApoE4 on beta-amyloid clearance in the human central nervous system. She also serves as the Administrative Core Leader and Deputy Director of the Dominantly Inherited Alzheimer’s Network (DIAN). In this role, Dr. Paumier helps coordinate efforts and facilitates the day-to-day progress toward the core goals of the DIAN observational study (grant # U19AG032438).

 
Abstract: ...Read More 

In vivo metabolism of alpha-synuclein in human central nervous system

Katrina L. Paumier, Rebecca L. Miller, James G. Bollinger, Jennifer Jockel-Balsarotti, Chihiro Sato, Bruce W. Patterson, Timothy M. Miller, Randall J. Bateman and Paul T. Kotzbauer
Neurology, Washington University, Saint Louis, MO

Multiple approaches targeting alpha-synuclein accumulation are currently being pursued for the treatment of Parkinson Disease (PD). Biomarkers are needed to assess target engagement and pharmacodynamics at both the preclinical and clinical stages of development for alpha-synuclein (aSyn) targeted therapies. To address this need, we developed an immunoprecipitation tandem mass spectrometry (IP-LC/MS) method based on the Stable Isotope Labeling Kinetics (SILK) technique to quantitate aSyn and measure its turnover kinetics in human cerebrospinal fluid (CSF). SILK utilizes mass spectrometry to track the incorporation of a safe stable isotope amino acid tracer (13C6-leucine) into newly synthesized proteins. Production and clearance rates can be determined in CSF, as a proxy for brain, by quantifying the isotopically-labeled protein during pulse-chase labeling with the stable isotope tracer. Leveraging our previous experience with the development of SILK protocols in human participants, we first developed an optimized IP protocol by screening a panel of in-house and commercial antibodies under a set of commonly applied IP conditions. Then, we optimized a LC/MS method capable of quantifying the amount of 13C-labeled aSyn present in human CSF. With optimized assay parameters in hand, we defined a suitable labeling strategy for SILK analysis to determine the production and clearance rates of aSyn in CSF from a set of control human participants. Using this method, we determined that the physiological half-life of aSyn in the human CNS is approximately 8 days. Additional studies are underway to assess aSyn production and clearance rates in PD patients to determine whether altered aSyn metabolism occurs. Further, this technique will provide a valuable measure of target engagement and pharmacodynamics for future clinical studies targeting aSyn.

Supported by Biogen and Michael J. Fox Foundation.

Benefits:
1. SILK methods allow for robust characterization of aSyn half-life in humans
2. SILK can be utilized to assess altered aSyn metabolism in PD and other synucleinopathies (diagnostic)
3. SILK can provide a measure of target engagement for therapeutic trials

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2:45
Panel: Industry Partnering with Academia & CROs to Advance Research
Linda Hedley
Linda Hedley
President
Hedley Consulting
 
Linda Hedley
Linda Hedley
President
Hedley Consulting
 
About Speaker:

Linda was previously a Principle Research Scientist and the Research Manger, CNS Division of Roche Palo Alto LLC. She has thirty years of experience in pre-clinical pharmacology and drug development. Linda Hedley is the former Global CNS Technical Director at a CRO; Eurofins Panlabs/Cerep (formally MDS Pharma Services/Ricerca Bioscience) where she spent 6 years working with Business Development and Operations to conduct efficacy studies for Pharma and Biotech clients worldwide. Currently, Linda provides Business Development consulting services for CRO's and consults with biotech clients to implement custom study designs to determine the efficacy of novel compounds in CNS in vivo models. She has expertise in several therapeutic areas such as epilepsy, pain, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, MS, psychosis, anxiety, and depression.

Nadim Shohdy
Nadim Shohdy
Office of Therapeutics Alliances
NYU
 
Nadim Shohdy
Nadim Shohdy
Office of Therapeutics Alliances
NYU
 
About Speaker:

Dr. Shohdy is an Assistant Dean at NYU School of Medicine, and is both the co-founder and Director of its Office Therapeutics Alliances (OTA), a drug discovery accelerator that utilizes a novel “virtual biotech” approach to help advance NYU biomedical research to a far more validated stage to enable greater quality dealmaking with biopharma and investors. Since its inception in 2013, OTA has transformed the way NYU de-risks its therapeutic projects, currently has a pipeline of over 15 projects, and 7 of its projects have been successfully partnered with biopharma or investors to launch new startups. He received his PhD with Distinction in Microbiology from Columbia University, and completed his postdoctoral training at the Rockefeller University.

Robert  Scannevin
Robert Scannevin
Vice President
Yumanity
 
Robert  Scannevin
Robert Scannevin
Vice President
Yumanity
 
About Speaker:

In early 2016 Dr. Scannevin joined Yumanity Therapeutics as the Vice President of Discovery Biology, where he now oversees all biology research. Yumanity utilizes a transformational discovery platform which can deliver new neurodegenerative disease-relevant targets and identify therapeutic agents that can ameliorate pathological cellular phenotypes. Dr. Scannevin’s career has spanned more than 16 years in neurology drug discovery research, and he has been a research leader at Wyeth (now Pfizer), Johnson & Johnson Pharmaceutical Research and Development (now Janssen), and Biogen.
Dr. Scannevin has focused on the discovery of disease modifying therapies for the treatment of amyotrophic lateral sclerosis (ALS), multiple sclerosis, Parkinson’s and Alzheimer’s diseases. Most of these efforts center around identifying molecular mechanisms that contribute to pathological states and developing approaches to combat these disease processes. In his time at Biogen, Dr. Scannevin was involved with the preclinical development of BIIB037 (aducanumab), the anti-amyloid beta antibody now in phase III clinical trials for the treatment of Alzheimer’s disease. Dr. Scannevin also was the Preclinical Research Lead for the development and marketing approval of Tecfidera (dimethyl fumarate), an oral drug for the treatment of multiple sclerosis.

Suzana  Petanceska
Suzana Petanceska
Senior Advisor for Strategic Development and Partnerships
National Institute on Aging, NIH
 
Suzana  Petanceska
Suzana Petanceska
Senior Advisor for Strategic Development and Partnerships
National Institute on Aging, NIH
 
About Speaker:

Dr. Petanceska is a senior advisor for strategic development and partnerships and a program director for systems biology and systems pharmacology at the Division of Neuroscience of the National Institute on Aging (NIA).  During her tenure at the NIA she has been overseeing and developing a number of research portfolios and innovative programs in basic and translational research for Alzheimer’s disease (AD).  Her recent program development efforts have been focused on developing systems biology and systems pharmacology capabilities for AD research and drug development within an open science framework.    Dr. Petanceska was instrumental for the development of NIA’s AD Translational Research Program and leads NIA’s open-science, systems biology programs for target and biomarker discovery: the AMP-AD Target Discovery and Preclinical Validation Project and the M2OVE-AD Consortium

 

Dr Petanceska holds a Ph.D. in Pharmacology from New York University.  Following her postdoctoral training at Rockefeller University and Cornell University, she established her independent research career at the Nathan Kline Institute in Orangeburg, N.Y., and joined the faculty of New York University Medical Center. Her research focused on the role of disrupted sterol metabolism in Alzheimer’s disease pathogenesis and on the mechanisms by which estrogens and cholesterol-lowering drugs exert neuroprotection.

Panel Moderator:
Lara  Mangravite
Lara Mangravite
President
Sage Bionetworks
 
Lara  Mangravite
Lara Mangravite
President
Sage Bionetworks
 
About Speaker:

Lara Mangravite, PhD is President of Sage Bionetworks. This organization is focused on the development and implementation of practices for large-scale collaborative biomedical research. Previously, Dr. Mangravite served as Director of the Systems Biology research group at Sage Bionetworks where she focused on the application of collaborative approaches to advance understanding of disease biology and treatment outcomes at a systems level with the overriding goal of improving clinical care. Dr. Mangravite obtained a BS in Physics from the Pennsylvania State University and a PhD in Pharmaceutical Chemistry from the University of California, San Francisco. She completed a postdoctoral fellowship in cardiovascular pharmacogenomics at the Children’s Hospital Oakland Research Institute.

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3:35
Afternoon Networking Break
1
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Round Table Discussions
Chairpersons Shown Below
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4:00
Round Table 1: Predictive Biomarkers in CNS diseases
Arti  Gaur
Arti Gaur
Assistant Professor of Neurology
Dartmouth Geisel School of Medicine
 
Arti  Gaur
Arti Gaur
Assistant Professor of Neurology
Dartmouth Geisel School of Medicine
 
About Speaker:

Dr. Gaur is an Assistant Professor of Neurology and Member of the Norris Cotton Cancer Center at the Geisel School of Medicine, Dartmouth. She received her BS from Rochester Institute of Technology, NY; an MS from University of Rochester, NY and her PhD from University of Cologne, Germany.

At Dartmouth, her group studies the molecular basis of neurological pathologies, specifically gliomas. Dr. Gaur is the Study Chair on an Alliance funded, multi-institutional, prospective clinical trial that is establishing a comprehensive panel of diagnostic and prognostic markers as well as biomarkers of tumor burden, treatment efficacy and toxicity. Furthermore in this trial her group is testing patient tumors ex vivo and establishing novel therapies to treat gliomas.  The other sites for this trial are University of Vermont Medical Center, Tufts Medical Center and Massachussets General Hospital.  Additionally, Dr. Gaur’s group is also developing innovative, in vivo wireless, nano scale devices, to measure multiple biomarkers of health that can predict and track the course of diseases, enable real-time evaluation of treatment efficacy and deliver targeted therapies in patients suffering from incurable and debilitating neurological disorders.

Round Table 2: Use of iPSCs to Understand Neurological Disease Pathogenesis and Discover New Therapies/Screen Drugs
Thomas  Kukar
Thomas Kukar
Assistant Professor
Emory University School of Medicine
 
Thomas  Kukar
Thomas Kukar
Assistant Professor
Emory University School of Medicine
 
About Speaker:

Thomas Kukar began his research career at the University of Florida where he obtained his BS in Microbiology and Cell Science and subsequently earned a PhD in Medicinal Chemistry and Pharmaceutical science. Dr. Kukar completed his post-doctoral training at the Mayo Clinic Jacksonville where he focused on the molecular pathogenesis and development of therapies for Alzheimer’s disease. He competed successfully for an NIH Pathways to Independence Award and started his lab in 2010 as Assistant Professor in the Department of Pharmacology and Neurology at Emory University in Atlanta, GA and joined the Center for Neurodegenerative Disease. He is a member of the Executive Steering Committee for the Emory Neuroscience graduate program and training faculty of the Molecular and Systems Pharmacology graduate program.

 

Dr. Kukar’s laboratory is broadly interested in deciphering the pathogenic mechanisms to enable development of novel therapies for neurodegenerative diseases. A major research focus has been studying dominantly-inherited genetic forms of disease and the contribution of the environment to Frontotemporal dementia, Amyotrophic Lateral Sclerosis, and Alzheimer’s disease. Recent work from the Kukar lab suggests that particular mutations linked to neurodegeneration (i.e. PGRN/GRN, C9ORF72, Trem2, GBA, LRRK2) are pathogenic through dysregulation of the lysosomal-inflammatory axis, opening up new avenues for therapeutic intervention in the clinic. 

 

Round Table 3: New Approaches for Target Validation in CNS Diseases
Katrina Paumier
Katrina Paumier
Assistant Professor of Neurology
Washington University School of Medicine
 
Katrina Paumier
Katrina Paumier
Assistant Professor of Neurology
Washington University School of Medicine
 
About Speaker:

Dr. Paumier received her undergraduate training in psychology at the University of Illinois Champaign-Urbana and her PhD in neuroscience from the University of Cincinnati. She then completed her postdoctoral training in the laboratories of Dr. John Dunlop and Dr. Warren Hirst at Pfizer Inc, where she focused on autophagy enhancers for Parkinson’s disease. She was previously an Assistant Professor on the neuroscience faculty at Michigan State University where she focused on treatment strategies for Parkinson’s disease. Dr. Paumier joined the Bateman team in 2015 and is actively involved in several research activities related to misfolded proteins involved in neurodegeneration including alpha-synuclein and amyloid-beta. At present, Dr. Paumier contributes to several studies including: 1) Development of a stable isotope labeling kinetic (SILK) method to measure the turnover rate of alpha-synuclein in human CSF; 2) Investigation of genetic protective factors against Alzheimer's disease; 3) Examination of effects of ApoE4 on beta-amyloid clearance in the human central nervous system. She also serves as the Administrative Core Leader and Deputy Director of the Dominantly Inherited Alzheimer’s Network (DIAN). In this role, Dr. Paumier helps coordinate efforts and facilitates the day-to-day progress toward the core goals of the DIAN observational study (grant # U19AG032438).

Round Table 4: Are Immunomodulatory Interventions Ready for Prime Time?
Malu Tansey
Malu Tansey
Professor, Physiology
Emory University
 
Malu Tansey
Malu Tansey
Professor, Physiology
Emory University
 
About Speaker:
Round Table 5: New Methods of Neuroimaging and Analysis
Sudeepti Southekal
Sudeepti Southekal
Principal Imaging Scientist
Avid Radiopharmaceuticals
 
Sudeepti Southekal
Sudeepti Southekal
Principal Imaging Scientist
Avid Radiopharmaceuticals
 
About Speaker:

Dr. Sudeepti Southekal serves as Principal Imaging Scientist at Avid Radiopharmaceuticals, Inc., where she leads image acquisition and analysis efforts to support the clinical development of biomarkers for Alzheimer’s disease. She is currently researching novel techniques to maximize the clinical potential of tau PET using tracer Flortaucipir F 18.

Dr. Southekal has over 10 years of experience in the development, physics and clinical applications of medical imaging systems, specializing in quantitative PET. She has previously held roles at Brookhaven National Laboratory, Brigham and Women’s hospital, and GE Healthcare.

Dr. Southekal received her Ph.D. in Biomedical Engineering with a concentration in Medical Physics from Stony Brook University, NY. She has co-authored 15 peer-reviewed articles and over 40 abstracts and conference proceedings.

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5:15
Networking Reception & Poster Session
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Day - 2 Tuesday, September 12, 2017
 
7:15
Breakfast with Mentors from Academia & Industry
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Neurodegenerative Diseases Research & Development 2017
Next Frontier in Multiple Sclerosis to Limit or Reverse Neurodegeneration
Moderator: Thomas Kukar, Emory University School of Medicine
8:30
Regulation of CNS Inflammation and Neurodegeneration by Astrocytes
 
Francisco Quintana
Francisco Quintana
Associate Professor of Neurology
Harvard Medical School
About Speaker: Francisco J. Quintana, PhD is an Associate Professor of Neurology at the Center for Neurologic Diseases, at Brigham and Women’s Hospital, Harvard Medical School, and an Associate Member at the Broad Institute of Harvard and MIT. Dr. Quintana, ... Read Full Bio 
 
 
Francisco Quintana
Francisco Quintana
Associate Professor of Neurology
Harvard Medical School
 
About Speaker:

Francisco J. Quintana, PhD is an Associate Professor of Neurology at the Center for Neurologic Diseases, at Brigham and Women’s Hospital, Harvard Medical School, and an Associate Member at the Broad Institute of Harvard and MIT.

Dr. Quintana, a graduate of the University of Buenos Aires (1999, Argentina), obtained his PhD in immunology at the Weizmann Institute of Science (2004, Israel). He received postdoctoral training at the Weizmann Institute of Science and at Harvard Medical School. In 2009, Dr. Quintana joined the faculty of Harvard Medical School.

Dr. Quintana’s research investigates signaling pathways that control the immune response and neurodegeneration, with the ultimate goal of identifying novel therapeutic targets and biomarkers for immune-mediated disorders. Dr. Quintana has published over 140 peer reviewed articles and book chapters. In addition, Dr. Quintana’s research has resulted in multiple patents which have been the foundation of three companies: ImmunArray Ltd, Alma Bio Therapeutics and AnTolRx Inc.

Dr. Quintana is the recipient of the Lady Anne Chain Prize for Academic Excellence and Scientific Achievements, the Junior Investigator Award from the National Multiple Sclerosis Society, the Pathway to Independence Award of the National Institute of Allergy and Infectious Diseases, the Award for Outstanding Research Achievement form Nature Biotechnology and the Tecan Award for Innovation, the Harry Weaver Award from the National Multiple Sclerosis Society.

 
Abstract: Astrocytes play important roles in th...Read More 

Astrocytes play important roles in the central nervous system (CNS) during health and disease. Thus, the identification of factors that regulate astrocyte activity may shed light on CNS physiology and guide new therapies for human neurologic disorders. We found that glycosphingolipid metabolism promotes pathogenic astrocyte activities up-regulated in the CNS during experimental autoimmune encephalomyelitis (EAE). Lactosylceramide (LacCer) synthesized by β-1,4-galactosyltransferase 6 (B4GALT6) in astrocytes acts in an autocrine manner to trigger transcriptional programs that promote the recruitment and activation of CNS-infiltrating monocytes and microglia, and neurodegeneration.  We also detected increased B4GALT6 expression and LacCer levels in CNS MS lesions.  The inhibition of glycosphingolipid metabolism suppressed local CNS innate immunity and neurodegeneration in EAE, and interfered with the activation of human astrocytes in vitro. Conversely, we found that type I interferon (IFN-I) signaling in astrocytes limits neurodegeneration through a mechanism mediated by the ligand-activated transcription factor aryl hydrocarbon receptor (AhR) and suppressor of cytokine signaling 2 (SOCS2). Moreover, we found that AhR agonists derived from dietary tryptophan by the metabolism of the commensal flora and the host act on astrocytes to limit CNS inflammation. AhR agonists, however, were decreased in MS patients. Taken together, these findings identify pathways that regulate astrocyte activities in the context of CNS inflammation and identify new targets for therapeutic intervention in MS and other neurologic diseases.

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CNS Partnering & Deal-Making 2017
CNS Perspectives from Venture Capital & Public Markets
Moderator: Parag Mehta, Aveta Biomics
8:30
CNS in the Eyes of Partners and Institutions
 
Harry Tracy
Harry Tracy
President
NI Research
About Speaker: Harry Tracy PhD is the founder and President of NI Research. Following his training at the University of Miami and Massachusetts General Hospital, and twenty-five years of clinical expertise in a variety of inpatient and outpatient settings, Tracy la... Read Full Bio 
 
 
Harry Tracy
Harry Tracy
President
NI Research
 
About Speaker:

Harry Tracy PhD is the founder and President of NI Research. Following his training at the University of Miami and Massachusetts General Hospital, and twenty-five years of clinical expertise in a variety of inpatient and outpatient settings, Tracy launched NIR and NeuroPerspective (formerly NeuroInvestment) in 1995. NeuroPerspective has set the standard for in-depth, independent, and frank appraisals of the companies and programs advancing new treatments and technologies in neurotherapeutics. NIR has also published NeuroLicensing since 2007. NIR formed its strategic consultation arm ('Second Opinion') in 2000, providing services to companies ranging from the largest major pharma companies to early-stage startups, featuring strategic portfolio reviews and licensing consultation.

 
Abstract: Evaluating the real-world performance...Read More 

Evaluating the real-world performance of the CNS sector can be a challenge in itself, given the herd mentality that often grips the CNS sector and those investing in it. Assessing the evaluations made by partners and investors  requires the consideration of where they are actually putting their resources, as opposed to where they say they are looking. This talk will look at CNS partnering and investing trends over the past several years; some common misperceptions about the CNS sector; and a review of clinical progress that belies many of those misconceptions.

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8:55
Mobilizing Neural Stem Cells for Remyelination by Targeting Gli1
 
James Salzer
James Salzer
Professor, Department of Neuroscience and Physiology
NYU School of Medicine
About Speaker: Dr. James Salzer is Professor of Neuroscience and Physiology and of Neurology at the NYU School of Medicine. He received his bachelor’s degree from Stanford University, an M.D., Ph.D. from Washington University, and completed a neurology residency ... Read Full Bio 
 
 
James Salzer
James Salzer
Professor, Department of Neuroscience and Physiology
NYU School of Medicine
 
About Speaker:

Dr. James Salzer is Professor of Neuroscience and Physiology and of Neurology at the NYU School of Medicine. He received his bachelor’s degree from Stanford University, an M.D., Ph.D. from Washington University, and completed a neurology residency at New York Hospital/Weill Cornell Medical College.  He has been on the faculty at NYU since 1984, serving as the Head of the Neurology Clinics at Bellevue Hospital, Director of the Medical Scientist Training Program, and Co-Director of the NYU Langone Multiple Sclerosis Center of Excellence.  Dr. Salzer’s research focuses on myelinated axons – nerve fibers essential for the rapid conduction of electrical impulses in the nervous system.  Myelinated axons are the main target in a large number of neurological disorders, including multiple sclerosis.  His lab identified the key signal that promotes myelination in the PNS and elucidated the organization of axons that underlie their ability to rapidly conduct electrical impulses.  Current studies are focused on promoting myelin repair by endogenous stem cells in the adult CNS by targeting novel targets in the hedgehog signaling pathway.

 

 
Abstract: A major unmet goal in treating demyel...Read More 

A major unmet goal in treating demyelinating disorders, in particular Multiple Sclerosis (MS), is to enhance remyelination, which is typically limited.  Remyelination has the dual benefit of restoring normal nerve conduction and preventing the loss of axons – a significant source of morbidity in MS patients.  Remyelination requires expansion of stem/precursor cells, their recruitment to lesion sites, their differentiation into oligodendrocytes (OLs), and their formation of myelin sheaths around demyelinated axons. Both parenchymal oligodendrocyte progenitors (OPCs) and neural stem cells (NSCs) in the subventricular zone (SVZ) are known sources of remyelinating oligodendrocytes.  Their precise roles and contributions to remyelination and the factors that limit their effectiveness in repair are active research areas with important therapeutic implications. 

We have been investigating remyelination by NSCs in mouse models of demyelination.  We have identified a pool of Sonic hedgehog (Shh)-responsive adult NSCs that are a significant source of remyelinating cells.  These NSCs are enriched in the ventral SVZ and, with demyelination, are activated and migrate into lesion sites where they become remyelinating oligodendrocytes.  We have found recruitment of these NSCs into demyelinated lesions and their efficacy of remyelination is substantially enhanced by genetic ablation or pharmacological inhibition of Gli1 – a Shh-dependent transcription factor.  Pharmacological inhibition of Gli1 significantly increases remyelination in the adult CNS and improves functional recovery from inflammatory demyelination, i.e. experimental autoimmune encephalomyelitis.  Inhibition of Gli1 therefore represents a novel strategy to promote remyelination.  To advance this program, we have identified novel small molecule inhibitors of Gli1 that are currently being analyzed for their pharmacological properties and functional efficacy.

 

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8:55
Neuro-Innovator Land – Every Day is a New Opportunity…(A Wall Streeter’s Perspective)
 
Charles Duncan
Charles Duncan
Managing Director, Senior Analyst, Equity Research/Emerging Biopharma
Piper Jaffray
About Speaker: Charles Duncan, Ph.D., joined Piper Jaffray in 2012 as a managing director and senior research analyst focused on small- and mid-cap emerging growth biotechnology companies. Duncan brings more than 20 years of sell-side experience during which he h... Read Full Bio 
 
 
Charles Duncan
Charles Duncan
Managing Director, Senior Analyst, Equity Research/Emerging Biopharma
Piper Jaffray
 
About Speaker:

Charles Duncan, Ph.D., joined Piper Jaffray in 2012 as a managing director and senior research analyst focused on small- and mid-cap emerging growth biotechnology companies. Duncan brings more than 20 years of sell-side experience during which he has covered a broad range of biopharma companies, most recently serving as an analyst at JMP Securities since 2002. Previously, Duncan covered the sector at Dresdner Kleinwort Wasserstein, Vector Healthcare Group - Prudential Securities, Tucker Anthony Cleary Gull and Chatfield Dean & Co. Duncan has been recognized by industry sources, including the Financial Times and StarMine Analyst Awards, as being among the best analysts for his fundamental and timely analysis. Duncan began his career as a manager of clinical development at Global Drug Development, Inc., a pharmaceutical development consulting firm, and he also launched InfusionVision Medical, a
venture-backed start-up medical device company. He is a graduate of the University of Wisconsin-Madison and holds a doctorate in pharmaceutical sciences with a concentration in neuropharmacology from the University of Colorado.

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9:20
Myelin Repair and Neuroprotection POC assays for MS
 
Bruce Trapp
Bruce Trapp
Founder, Chairman of SAB and Chief Scientific Officer
Renovo Neural
About Speaker: University. Dr. Trapp received his Ph.D. from Loyola University Stritch School of Medicine in Chicago, IL.  He received postdoctoral training at the National Institutes of Health (NIH), Bethesda, MD and then was appointed Assistant and subsequently ... Read Full Bio 
 
 
Bruce Trapp
Bruce Trapp
Founder, Chairman of SAB and Chief Scientific Officer
Renovo Neural
 
About Speaker:

University. Dr. Trapp received his Ph.D. from Loyola University Stritch School of Medicine in Chicago, IL.  He received postdoctoral training at the National Institutes of Health (NIH), Bethesda, MD and then was appointed Assistant and subsequently Associate Professor of Neurology at the Johns Hopkins University School of Medicine in Baltimore.  He joined the Cleveland Clinic as Chairman of the Department of Neurosciences in 1994. He is the recipient of the Jordi Folch-Pi Award from the American Society of Neurochemistry, The Weil Award from the American Association of Neuropathologists, the Harry Weaver Neuroscience Scholar Award from the National Multiple Sclerosis Society (NMSS), the Jacob Javits Award in Neuroscience from the National Institute of Neurological Disorders and Stroke, the John Dystel Prize for MS Research from the American Academy of Neurology and the National Multiple Sclerosis Society, the Scientific Achievement Award in Basic Science and the Award for Excellence in Science from the Cleveland Clinic and Dr Trapp is a Fellow of the AAAS. Dr. Trapp’s research investigates the cause of neurological disability in multiple sclerosis patients, cellular mechanism of brain repair in neurodegenerative diseases, and the molecular biology of myelination in the central and peripheral nervous systems.  He is internationally known for his work on mechanisms of neurodegeneration and repair in multiple sclerosis and has published over 185 peer-reviewed articles and over 30 book chapters.  Dr Trapp sits on the advisory board of major biotech companies, the National Institutes of Health and the National Multiple Sclerosis Society and he is active in organizing national and international conferences related to Neurodegenerative diseases.

9:45
Extracellular RNA as Disease Marker for Multiple Sclerosis
 
Roopali  Gandhi
Roopali Gandhi
Assistant Professor in Neurology
Brigham and Women's Hospital
About Speaker: Dr. Roopali Gandhi is an Assistant Professor of Neurology at Harvard and Head of MS biomarkers at Brigham and Women’s Hospital.    Dr. Roopali Gandhi obtained her PhD. degree in Immunology from Jamia Milia Islamia and National Institute of... Read Full Bio 
 
 
Roopali  Gandhi
Roopali Gandhi
Assistant Professor in Neurology
Brigham and Women's Hospital
 
About Speaker:

Dr. Roopali Gandhi is an Assistant Professor of Neurology at Harvard and Head of MS biomarkers at Brigham and Women’s Hospital. 

 

Dr. Roopali Gandhi obtained her PhD. degree in Immunology from Jamia Milia Islamia and National Institute of Immunology (2005, India). She did her post doc fellowship at Harvard Medical School with Dr. Howard Weiner. Dr. Gandhi joined as faculty at Harvard in 2013.

Dr. Gandhi has more than 10 years experience in studying human immunology related to autoimmune diseases. She has made seminal contribution in identifying pathways related to regulatory immune cells induction and their function. Dr. Gandhi helped in developing several novel projects for identifying biomarkers for Multiple Sclerosis (MS). During her research with circulating miRNAs, she identified several miRNAs, linked with MS disease and its clinical parameters.  Currently, her laboratory is focused on identification of immune biomarkers in MS and understanding immune pathways linked to treatment effects. Her lab is funded by NIH and industry collaborations. She has published more than 30 peer reviewed papers and is reviewer of neurology journals and grants.

 
Abstract: Objective: To identify circulating mi...Read More 

Objective: To identify circulating microRNAs (miRNAs) linked to disease, disease
stage, and disability in multiple sclerosis (MS).
Methods: This study involved three phases: a discovery, an internal validation divided
into first and second validation in the CLIMB cohort, and an external validation using
samples from three other international MS cohorts. Serum miRNAs expression was
compared between MS patients and healthy controls and between disease subtypes;
relapsing remitting (RR) and secondary progressive (SP). Disability, as measured by
expanded disease severity scale (EDSS) was correlated with miRNA expression.
Results: Five miRNAs; hsa.miR484, hsa.miR140.5p, hsa.miR320a, hsa.miR486.5p,
and hsa.miR320c showed a significant difference between MS patients and healthy
individuals, among these miR484 remained significant after accounting for multiple
comparisons (p=0.01). When comparing RRMS to HCs, hsa.miR484 showed a
significant difference (p=0.004) between the groups after accounting for multiple group
comparisons. When SP and HC were compared, eight miRNAs were significantly
different, of these; six miRNAs remained significantly different after accounting for
multiple comparisons (hsa.miR484, hsa.miR140.5p, hsa.miR142.5p, hsa.miR320a,
hsa.miR320b, and hsa.miR320c). Disability correlation analysis with miRNA provided
four miRNAs hsa.miR320a, hsa.miR337.3p, hsa.miR199a.5p and hsa.miR142.5p that
correlated with EDSS.
Conclusions: We found several miRNAs that were differentially expressed in MS
patients in comparison to HC group and were associated with disability. These miRNAs
exhibit biological significance in MS pathophysiology and are promising candidates for
2 further validation and exploration of their potential use in developing diagnostic,
prognostic, and therapeutic tools for MS.

 Read Less
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9:20
Panel: CNS Perspectives from Venture Capital & Public Markets
Barbara  Tate
Barbara Tate
Venture Partner
Dementia Discovery Fund/SV Life Sciences
 
Barbara  Tate
Barbara Tate
Venture Partner
Dementia Discovery Fund/SV Life Sciences
 
About Speaker:

Dr. Barbara Tate is a neuroscientist who has worked in both large and small pharmaceutical companies. Prior to joining DDF, she was Vice President and Head of Biology at Rodin Therapeutics, an Atlas Venture company, where she also consulted on other portfolio companies. Prior to Rodin, Barbara was Vice President for Research at Satori Pharmaceuticals, a biotech company developing a treatment for Alzheimer’s disease. At Pfizer, Barbara managed the neurodegenerative disease area group in Groton, CT. Prior to working in industry, Barbara was an academic researcher at Brown Medical School and Harvard Medical School. Barbara has a BA in Biology, a PhD in Physiology, and she was a postdoctoral fellow at Harvard Medical School.

Kevin  Bitterman
Kevin Bitterman
Partner
Atlas Venture
 
Kevin  Bitterman
Kevin Bitterman
Partner
Atlas Venture
 
About Speaker:

Kevin Bitterman focuses on creating and investing in companies that translate groundbreaking science into innovative medicines.

Kevin was the founding CEO of Editas Medicine (NASDAQ: EDIT), Morphic Therapeutic and Visterra, and co-founded Genocea Biosciences (NASDAQ: GNCA). He previously served as a director of InSeal Medical, Kala Pharmaceuticals, Neuronetics, Taris Biomedical (acquired by Allergan) and Vets First Choice among other ventures. Prior to joining Atlas Venture in 2017, Kevin was a partner at Polaris Partners, where he had been a member of the healthcare team since 2004.

Kevin is active in the local life science and healthcare start-up community, serving on the Scientific Advisory Board of the Massachusetts Life Sciences Center (MLSC) and as Board Chair of the New England Venture Capital Association (NEVCA).  He received a BA in biology, summa cum laude, from Rutgers University before completing his PhD in genetics at Harvard Medical School.  He has published numerous scientific articles and is an inventor on several issued patents.

Outside of work Kevin enjoys spending time with his wife and two young daughters.  He also enjoys trail running and whiskey, though generally not at the same time.

Kiran  Reddy
Kiran Reddy
Partner
Clarus Ventures
 
Kiran  Reddy
Kiran Reddy
Partner
Clarus Ventures
 
About Speaker:

Kiran Reddy is the President & CEO of a Praxis Precision Medicines, a recently launched biotech company focused on precision medicine in genetically defined epilepsy and autism disorders. He is also a Venture Partner at Clarus Ventures where he focuses on new company formation, venture investments, and risk sharing partnerships with pharmaceutical companies.

Prior to Clarus, Kiran was at Biogen and led the CAPITA team within Corporate Development where he focused on sourcing new technologies and product opportunities to support the Company's growth via acquisitions, partnerships, and equity investments.

Prior to Biogen, Kiran was an Associate Partner at Third Rock Ventures. He supported and managed various portfolio companies in addition to focusing on new company formation and new investments.

Kiran was a key member of the founding team at SAGE Therapeutics, and served as its CBO and CMO through its IPO (NASDAQ: SAGE). He is also the co-inventor of SAGE-547 the Phase 3 program for an orphan epilepsy disorder and postpartum depression. SAGE has a market capitalization of ~$3.0B USD with greater than 250 employees, and named 2016 “Best places to work” in Cambridge, Massachusetts.

In addition, while at Third Rock, Kiran was part of the team that launched Foundation Medicine [NASDAQ: FMI] that has a market capitalization of greater than $1.0B USD and greater than 275 employees.

Before Third Rock Ventures, Kiran was a management consultant at the Lewin Group within in the biotechnology and pharmaceutical practice, and advised clients on clinical development and commercial strategy. Kiran holds MD and MBA degrees from Georgetown University. He completed his internship in medicine and his neurology residency at Harvard / Massachusetts General Hospital and is a board certified neurologist. Kiran was previously a Howard Hughes science fellow, and has authored several peer-reviewed scientific papers in the field of  epilepsy, neuroimmunology and neurodegenerative diseases. 

 

Iva Toudjarska
Iva Toudjarska
Principal Consultant
Halloran Consulting Group
 
Iva Toudjarska
Iva Toudjarska
Principal Consultant
Halloran Consulting Group
 
About Speaker:

Iva Toudjarska, PhD, MBA, is a principal Consultant at Halloran Consulting Group, Boston, MA. Iva joined Halloran with more than 15 years of experience, she has served biopharmaceutical companies across all phases of drug discovery, development and commercialization.  Iva’s expertise spans across diverse range of therapeutic areas with focus on neurodegenerative diseases among several others. At Halloran Iva spearheads development strategy engagements as well as due diligence, indication assessment and product and portfolio prioritization work, integrating scientific, clinical, regulatory and early commercial insights.

 

Prior to joining Halloran, Iva spent 4 years as a consultant to start-ups and pharma clients. As part of Putnam Associates’ team, she informed strategic insights and decisions pertaining to new product development, portfolio prioritization, clinical development, and commercial opportunity assessments for large pharma clients. She has informed launch of new therapeutics, as well as strategic brand planning and product positioning. Previously, she was a founding employee at Alnylam Pharmaceuticals, the leader in RNAi therapeutics. During her nine year tenure at Alnylam, as part of a multi-disciplinary team she advanced several programs to clinic, most notably ALN-RSV (a virology program) and ALN-VSP (an oncology program). She led the evaluation and initiation of two hematology programs ALN-AT3 for the treatment of hemophilia (currently in clinic) and ALN-TMP for the treatment of beta-thalassemia. 

Christine Brennan
Christine Brennan
Partner
MRL Ventures Fund
 
Christine Brennan
Christine Brennan
Partner
MRL Ventures Fund
 
About Speaker:

Dr. Brennan joined MRL Ventures Fund in June 2017. Prior to joining MRL Ventures Fund she spent 3 years as Principal at Novartis Venture Fund (NVF). Prior to NVF, she spent 3 years at Vitae Pharmaceuticals as Chief Business Officer responsible for strategy & business development. Prior to Vitae, she spent 5 years at Novartis Institutes for BioMedical Research in Strategic Alliances, most recently as Executive Director and Head of Strategy & Operations. Prior to Novartis, she held positions in business development and marketing at the biopharmaceutical companies, EnVivo Pharmaceuticals (now FORUM Pharmaceuticals), Biovail (now Valeant Pharmaceuticals) and Cogent Neuroscience. In addition, she was Director at Fidelity Biosciences Group (now F-Prime Capital Partners), a venture capital company. She received her Ph.D. in neuroscience from Dartmouth Medical School in 1995 and completed a postdoctoral fellowship at the National Institutes of Health in 1999.

Yaron  Werber
Yaron Werber
Chief Financial Officer and Chief Business Officer
Ovid Therapeutics
 
Yaron  Werber
Yaron Werber
Chief Financial Officer and Chief Business Officer
Ovid Therapeutics
 
About Speaker:

Yaron Werber, M.D., MBA, has served as our chief business officer since July 2016, as our chief financial officer since June 2015, as our secretary since July 2015 and as our treasurer since April 2017. Prior to joining us, Dr. Werber worked at Citigroup Global Markets Inc. from March 2004 to June 2015, where he most recently served as a managing director, starting December 2011, and the head of U.S. healthcare and biotech equity research teams. Previously, Dr. Werber was a senior biotech analyst and vice president at SG Cowen Securities Corporation. He began his career in academic research and was director of business development at NotifyMD, Inc., an e-health company. Dr. Werber earned his B.S. in Biology from Tufts University and a combined M.D./MBA degree from Tufts University School of Medicine.

Panel Moderator:
Charles Duncan
Charles Duncan
Managing Director, Senior Analyst, Equity Research/Emerging Biopharma
Piper Jaffray
 
Charles Duncan
Charles Duncan
Managing Director, Senior Analyst, Equity Research/Emerging Biopharma
Piper Jaffray
 
About Speaker:

Charles Duncan, Ph.D., joined Piper Jaffray in 2012 as a managing director and senior research analyst focused on small- and mid-cap emerging growth biotechnology companies. Duncan brings more than 20 years of sell-side experience during which he has covered a broad range of biopharma companies, most recently serving as an analyst at JMP Securities since 2002. Previously, Duncan covered the sector at Dresdner Kleinwort Wasserstein, Vector Healthcare Group - Prudential Securities, Tucker Anthony Cleary Gull and Chatfield Dean & Co. Duncan has been recognized by industry sources, including the Financial Times and StarMine Analyst Awards, as being among the best analysts for his fundamental and timely analysis. Duncan began his career as a manager of clinical development at Global Drug Development, Inc., a pharmaceutical development consulting firm, and he also launched InfusionVision Medical, a
venture-backed start-up medical device company. He is a graduate of the University of Wisconsin-Madison and holds a doctorate in pharmaceutical sciences with a concentration in neuropharmacology from the University of Colorado.

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10:10
Morning Networking Break
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Neurodegenerative Diseases Research & Development 2017
Therapeutics for Rare & Orphan CNS Disorders
Moderator: Cheryl Wellington, University of British Columbia
10:40
Innovating Therapeutics for Neuromyelitis Optica
 
Michael Yeaman
Michael Yeaman
Professor of Medicine, David Geffen School of Medicine
UCLA
About Speaker: Michael arrived in Roswell, New Mexico in 1963 – a land of mystery set amid Santa Fe to the North and Mayan pyramids to the South. Elements that bring meaning to this Land of Enchantment have indelibly etched his extraordinary successes in medicine... Read Full Bio 
 
 
Michael Yeaman
Michael Yeaman
Professor of Medicine, David Geffen School of Medicine
UCLA
 
About Speaker:

Michael arrived in Roswell, New Mexico in 1963 – a land of mystery set amid Santa Fe to the North and Mayan pyramids to the South. Elements that bring meaning to this Land of Enchantment have indelibly etched his extraordinary successes in medicine and art. Michael was raised in Southern California prior to earning undergraduate, doctoral and post-doctoral degrees in medical science from the University of New Mexico School of Medicine and the UCLA School of Medicine. In 1990, he returned to Los Angeles to conduct NIH and AHA Fellowships in Infectious Diseases and Molecular Immunology at UCLA and Harbor-UCLA Medical Center. In 1992, he was appointed to the Faculty of the UCLA School of Medicine – and by age 40 became among the youngest to earn the rank of full Professor of Medicine at UCLA. In 2005 he was named the Vice Chair of Medicine, and in 2010 was appointed Chief, Division of Molecular Medicine at Harbor-UCLA Medical Center.

Over three decades, Michael has pioneered medical solutions at the interface between infection and immunity. He led a team that discovered a new universal code in immune defense molecules. This breakthrough – published by the National Academy of Sciences – launched an entirely novel class of antibiotic compounds called kinocidins. He then discovered how the kinocidin molecular code accelerates adaptation of the human immune system to fend off even the most resistant pathogens. This advance was published by Nature. Based on these discoveries, Michael invented context-activated protides, or “smart antibiotics", which target only microbes that cause disease. In 2003, he discovered the structural relatedness in bacteria and fungi that enabled development of the first cross-kingdom vaccine against Candida and Staphylococcus, including MRSA. This vaccine has now completed Phase I and Phase II clinical trials. He and colleagues founded NovaDigm Therapeutics, Inc. to develop this and other next-generation vaccines. Most recently, he invented an exciting new class of anti-infective agents that exploit ancient programmed cell death pathways in pathogens. His groundbreaking work has received continuous funding from the National Institutes of Health and Department of Defense since 1995. Today, he is Principal Investigator and Director of the only NIH Systems Immunology program focused on MRSA infection and immunity, and PI of an NIH Innovation Award to catalyze breakthroughs in treating MRSA infection. Michael has received numerous honors & awards, including the Weitzman Memorial Research Award (Harbor-UCLA Medical Center), Alexander Research Award (American Heart Association), the National Research Service Award (National Institutes of Health) and Excellence in Teaching Awards from multiple institutions. He has published over 200 medical & scientific papers and related works, and holds 18 issued patents and many patents pending. He teaches medical students and Fellows at the Geffen School of Medicine at UCLA and Harbor-UCLA Medical Center, lectures internationally, is an appointed NIH expert in infectious diseases and immunology, and serves on editorial boards of preeminent journals. These examples illustrate his goal to discover, invent and develop innovative anti-infective agents and immunotherapeutic strategies to meet the challenge of antibiotic-resistant infections.

 
Abstract: Neuromyelitis optica spectrum disorde...Read More 

Neuromyelitis optica spectrum disorder (NMOSD) is a rare and life-threatening autoimmune disease which appears to have at its pathogenic origin a loss of immune tolerance to the water channel aquaporin-4 (AQP4) enriched on astrocytes or myelin oligodendrocyte glycoprotein (MOG) supporting neurons.  Resulting autoantibodies to AQP4 or MOG can initiate a cascade of autoimmune mechanisms including complement fixation and recruitment of inflammatory leukocytes.  In turn, these processes yield a characteristic pattern of astrocyte dysfunction and neuronal demyelination that most predominantly affect the spinal cord and optic nerves.  Although a variety of empirical therapies are currently used in treating NMOSD, none has been proven effective in prospective, adequately powered, randomized trials.  Moreover, the majority of current therapies subject patients to long-term immunosuppression that increases the risks of serious infections and development of cancers. Through an international collaboration catalyzed by the Guthy-Jackson Charitable Foundation, scientific advances in the last decade have enabled multiple clinical trials targeting mechanisms integral to NMOSD.  These trials are evaluating agents that target B cells (CD19), complement protein 5 (C5) or the interleukin-6 receptor (IL-6R) as logical therapeutic strategies.  Furthermore, specific mechanisms are the targets of next-generation technologies intended to modify only the pathogenic mechanisms in NMOSD, sparing host defenses against infection and cancer.  Finally, having known dominant autoantigen(s) makes NMOSD an ideal model for development of tolerogenic therapies or cures in the advent of the era of immune tolerization.  Lessons learned in meeting the challenge of solving NMOSD are likely to enhance and accelerate success of treatments or cures for other diseases of immune dysfunction as well.

 Read Less
CNS Partnering & Deal-Making 2017
Technology Transfer & Licensing: Challenges & Opportunities
Moderator: Michael McGurk, DLA Piper
10:40
Opportunities and Challenges of Licensing CNS Technologies from Academic Medical Centers
 
David Glass
David Glass
Technology Transfer Consultant
University of Massachusetts Dartmouth & D. Glass Associates, Inc.
About Speaker: David Glass is an independent consultant with over thirty years’ experience in biotechnology and related life sciences fields, with much of that time devoted to patents, licensing and technology transfer. Dr. Glass has served as associate director ... Read Full Bio 
 
 
David Glass
David Glass
Technology Transfer Consultant
University of Massachusetts Dartmouth & D. Glass Associates, Inc.
 
About Speaker:

David Glass is an independent consultant with over thirty years’ experience in biotechnology and related life sciences fields, with much of that time devoted to patents, licensing and technology transfer. Dr. Glass has served as associate director of the tech transfer office at Massachusetts General Hospital, and has also managed technology transfer as a consultant or part-time employee at other academic institutions including McLean Hospital, Joslin Diabetes Center, and Baystate Medical Center. Since 2014, he has directed tech transfer as a consultant at the University of Massachusetts at Dartmouth, and he recently completed a one-year assignment as interim licensing manager at Partners HealthCare Innovation, managing and licensing neuroscience and psychiatric technologies from McLean Hospital and the MGH Psychiatry Department. Dr. Glass has also consulted for several other academic institutions and life sciences companies on various aspects of technology licensing and academic-industry partnering., He is a longstanding member of LES and AUTM, and was awarded the Certified Licensing Professional certification in July 2008 (recertified July 2011, expired June 2014) and the Registered Technology Transfer Professional certification in November 2013 (active). He holds a B.S. from Cornell University and a Ph.D. from Princeton University.

 
Abstract: Cutting edge, commercially and clinic...Read More 

Cutting edge, commercially and clinically relevant research on a wide range of CNS diseases and disorders takes place at medical schools and academic medical centers all over the world. Much of this research would be of great value to pharmaceutical and biotech companies seeking to develop beneficial products to diagnose, treat or prevent debilitating neurological and psychiatric disorders, but successfully transferring such technologies to industry remains challenging. Some of the obstacles faced by the licensing and innovation officers of academic medical centers are common to all technology transfer offices, in that much of the research is early-stage, exploratory research, which rarely progresses to the level of validation and proof of concept needed to survive the rigorous vetting process employed by most biopharma companies. But other obstacles are unique to the CNS sector, including the lack of animal models to effectively model many human CNS disorders, the difficulties in carrying out clinical trials and assessing efficacy using subjective rating scales or other criteria, and the changing levels of interest shown by big pharma in CNS diseases, particularly many psychiatric disorders.

This presentation will describe some of these challenges and how they can be overcome. The presentation will be illustrated by case examples of technologies successfully commercialized by Partners HealthCare Innovation, the office tasked with commercializing inventions and research from Massachusetts General Hospital, Brigham & Women’s Hospital, and McLean Hospital, and would include discussion of licensing deals to established companies as well as the formation of spin-off companies.

Benefits:
• Learn about the research activities taking place in the CNS space at the leading Boston-area hospitals of Partners HealthCare
• Learn about the challenges faced in successfully licensing CNS research developments to biopharma companies.
• Understand the challenges of start-up company formation in the CNS space.
• Hear what innovative strategies are being employed to bring academic CNS research to commercial utility.

 Read Less
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11:05
Splice-switching Antisense Oligonucleotides for the Treatment of Neurological Disease
 
Michelle Hastings
Michelle Hastings
Associate Professor of Cell Biology and Anatomy
Rosalind Franklin University/Chicago Medical School
About Speaker: Dr. Michelle Hastings, Ph.D., is an Associate Professor of Cell Biology and Anatomy at The Chicago Medical School at Rosalind Franklin University of Medicine and Science. Dr. Hastings earned an undergraduate degree in biology from St. Olaf Colle... Read Full Bio 
 
 
Michelle Hastings
Michelle Hastings
Associate Professor of Cell Biology and Anatomy
Rosalind Franklin University/Chicago Medical School
 
About Speaker:

Dr. Michelle Hastings, Ph.D., is an Associate Professor of Cell Biology and Anatomy at The Chicago Medical School at Rosalind Franklin University of Medicine and Science. Dr. Hastings earned an undergraduate degree in biology from St. Olaf College and went on to receive a Ph.D. in biology from Marquette University. She was a postdoctoral and senior fellow at Cold Spring Harbor Laboratory before joining the faculty at the Chicago Medical School in 2007. Dr. Hastings’ work focuses on the molecular mechanisms of RNA processing and how defects in this process can cause diseases such as spinal muscular atrophy (SMA), Usher syndrome, and Alzheimer’s and Parkinson’s disease. Her studies also aim to develop therapeutic approaches to treat these disorders using antisense technology. Her work describing an antisense molecule that rescues hearing and vestibular dysfunction in Usher type 1C was one of the first studies to demonstrate the feasibility of recovering hearing and vestibular function in an animal model of a human disease involving congenital neurosensory hearing loss.

 
Abstract: Splice-switching oligonucleotides (SS...Read More 

Splice-switching oligonucleotides (SSOs) have recently achieved clinical success in the treatment of neurodegenerative disease. We have used a similar approach to develop therapeutic SSOs that treat neurosensory hearing loss, vestibular deficits and retinitis pigmentosa in a mouse model of Usher syndrome Type 1C. Building on this success, we are exploring SSO-based approaches for the treatment of other rare diseases including Juvenile Neuronal Ceroid Lipofuscinosis (JNCL). JNCL is a fatal, pediatric, neurodegenerative, lysosomal storage disease that is caused most frequently by deletion of exons 7 and 8 in CLN3, resulting in a reading frameshift. We have designed SSOs that induce splicing changes that restore the CLN3Δex7/8 mRNA reading frame. We find that this frame-correcting approach partially reduces histopathological and phenotypic features of JNCL in Cln3Δex7/8 transgenic mice. Our successful treatments of Usher syndrome and JNCL in animal models are further demonstration of the potential of SSOs as treatments for neurological disease.

 Read Less
11:05
De-risking Academic Therapeutic Assets to Enable Greater Partnerships with Industry
 
Nadim Shohdy
Nadim Shohdy
Office of Therapeutics Alliances
NYU
About Speaker: Dr. Shohdy is an Assistant Dean at NYU School of Medicine, and is both the co-founder and Director of its Office Therapeutics Alliances (OTA), a drug discovery accelerator that utilizes a novel “virtual biotech” approach to help advance NYU biome... Read Full Bio 
 
 
Nadim Shohdy
Nadim Shohdy
Office of Therapeutics Alliances
NYU
 
About Speaker:

Dr. Shohdy is an Assistant Dean at NYU School of Medicine, and is both the co-founder and Director of its Office Therapeutics Alliances (OTA), a drug discovery accelerator that utilizes a novel “virtual biotech” approach to help advance NYU biomedical research to a far more validated stage to enable greater quality dealmaking with biopharma and investors. Since its inception in 2013, OTA has transformed the way NYU de-risks its therapeutic projects, currently has a pipeline of over 15 projects, and 7 of its projects have been successfully partnered with biopharma or investors to launch new startups. He received his PhD with Distinction in Microbiology from Columbia University, and completed his postdoctoral training at the Rockefeller University.

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11:30
Therapeutic Strategies to Treat Frontotemporal Dementia Caused by Progranulin Haploinsufficiency
 
Thomas  Kukar
Thomas Kukar
Assistant Professor
Emory University School of Medicine
About Speaker: Thomas Kukar began his research career at the University of Florida where he obtained his BS in Microbiology and Cell Science and subsequently earned a PhD in Medicinal Chemistry and Pharmaceutical science. Dr. Kukar completed his post-doctoral train... Read Full Bio 
 
 
Thomas  Kukar
Thomas Kukar
Assistant Professor
Emory University School of Medicine
 
About Speaker:

Thomas Kukar began his research career at the University of Florida where he obtained his BS in Microbiology and Cell Science and subsequently earned a PhD in Medicinal Chemistry and Pharmaceutical science. Dr. Kukar completed his post-doctoral training at the Mayo Clinic Jacksonville where he focused on the molecular pathogenesis and development of therapies for Alzheimer’s disease. He competed successfully for an NIH Pathways to Independence Award and started his lab in 2010 as Assistant Professor in the Department of Pharmacology and Neurology at Emory University in Atlanta, GA and joined the Center for Neurodegenerative Disease. He is a member of the Executive Steering Committee for the Emory Neuroscience graduate program and training faculty of the Molecular and Systems Pharmacology graduate program.

 

Dr. Kukar’s laboratory is broadly interested in deciphering the pathogenic mechanisms to enable development of novel therapies for neurodegenerative diseases. A major research focus has been studying dominantly-inherited genetic forms of disease and the contribution of the environment to Frontotemporal dementia, Amyotrophic Lateral Sclerosis, and Alzheimer’s disease. Recent work from the Kukar lab suggests that particular mutations linked to neurodegeneration (i.e. PGRN/GRN, C9ORF72, Trem2, GBA, LRRK2) are pathogenic through dysregulation of the lysosomal-inflammatory axis, opening up new avenues for therapeutic intervention in the clinic. 

 

 
Abstract: Pathogenic mutations in the ...Read More 

Pathogenic mutations in the GRN gene are one of the most common causes of Frontotemporal Dementia (FTD) and cause a loss of progranulin (PGRN) function by decreasing PGRN mRNA and protein by ~50%. FTD is a rare neurodegenerative disease characterized by atrophy of the frontal and temporal lobes of the brain. PGRN is a secreted glycoprotein composed of 7.5 domains that is cleaved into 6kDa granulin proteins (GRNs 1-7) through a poorly defined pathway. Furthermore, carriers of two pathogenic GRNmutations fail to make PGRN and develop neuronal ceroid lipofuscinosis (NCL), a neurodegenerative lysosomal storage disease. Although FTD and NCL have different ages of onset and clinical symptoms there is increasing evidence that decreased levels of PGRN in both diseases cause pathogenesis through a shared pathway: the lysosome. Specifically, multiple lines of converging evidence suggest that decreased levels of PGRN/GRNs induce lysosomal dysfunction which leads to neuroinflammation and neurodegeneration. In particular, Grn knockout (KO) mice have markers of lysosome dysfunction (enlarged lysosomes, accumulation of lysosomal proteins) and NCL pathology (accumulation of lipofuscin and ubiquitinated proteins). Heterozygote Grn KO mice also accumulate lysosomal proteins abnormally with increasing age. More importantly, multiple cell types and tissues isolated from FTD-GRN patients accumulate markers of NCL pathology and lysosome dysfunction. Taken together, these data demonstrate that PGRN is critical for lysosomal homeostasis, but the molecular link between PGRN, GRNs, and the lysosome has yet to be elucidated.

Using newly developed and characterized biochemical and immunocytochemical tools, we find that PGRN is rapidly processed into stable, mature GRNs in the endolysosomal pathway, suggesting GRNs have a function within the lysosome (Holler et al,eNeuro, 2017). Further, at least two GRNs (GRN-2 and GRN-4) are haploinsufficient in FTD-GRN patient derived fibroblasts and brain tissue. Finally, administration of a human recombinant GRN, which is internalized and directed to the lysosome, rescues lysosomal defects in Grn KO mouse fibroblasts, providing additional evidence that granulins have a lysosomal function and are protective. We hypothesize that GRNs are the functional unit(s) of PGRN and are required to perform critical lysosomal functions.

 

 Read Less
11:55
Novel Wireless Therapeutic Strategies to Treat Gliomas
 
Arti  Gaur
Arti Gaur
Assistant Professor of Neurology
Dartmouth Geisel School of Medicine
About Speaker: Dr. Gaur is an Assistant Professor of Neurology and Member of the Norris Cotton Cancer Center at the Geisel School of Medicine, Dartmouth. She received her BS from Rochester Institute of Technology, NY; an MS from University of Rochester, NY and her ... Read Full Bio 
 
 
Arti  Gaur
Arti Gaur
Assistant Professor of Neurology
Dartmouth Geisel School of Medicine
 
About Speaker:

Dr. Gaur is an Assistant Professor of Neurology and Member of the Norris Cotton Cancer Center at the Geisel School of Medicine, Dartmouth. She received her BS from Rochester Institute of Technology, NY; an MS from University of Rochester, NY and her PhD from University of Cologne, Germany.

At Dartmouth, her group studies the molecular basis of neurological pathologies, specifically gliomas. Dr. Gaur is the Study Chair on an Alliance funded, multi-institutional, prospective clinical trial that is establishing a comprehensive panel of diagnostic and prognostic markers as well as biomarkers of tumor burden, treatment efficacy and toxicity. Furthermore in this trial her group is testing patient tumors ex vivo and establishing novel therapies to treat gliomas.  The other sites for this trial are University of Vermont Medical Center, Tufts Medical Center and Massachussets General Hospital.  Additionally, Dr. Gaur’s group is also developing innovative, in vivo wireless, nano scale devices, to measure multiple biomarkers of health that can predict and track the course of diseases, enable real-time evaluation of treatment efficacy and deliver targeted therapies in patients suffering from incurable and debilitating neurological disorders.

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11:30
Panel Discussion: Technology Transfer and Licensing: Challenges and Opportunities
Mark  Allegretta
Mark Allegretta
Associate VP, Commercial Research
National Multiple Sclerosis Society
 
Mark  Allegretta
Mark Allegretta
Associate VP, Commercial Research
National Multiple Sclerosis Society
 
About Speaker:

Dr. Allegretta is the Associate Vice President of Commercial Research at the National MS Society, whose responsibilities include providing leadership and direction for the Society’s commercial research programs and portfolio, including partnerships developed through Fast Forward. He works to engage the academic and commercial research community to develop innovative solutions for MS treatments, and works with other members of the research team to assure integration and alignment of commercial research programs with the Society's overall research goals. He joined the Society in 2014 with 28 years of experience in biotechnology and pharmaceutical operations. Most recently, he was President, Chief Scientific Officer and Co-founder of BioMosaics Inc. in Burlington, Vermont, where he managed all aspects of cancer biomarker development, licensing deals and business partnerships. Prior to that, he worked in various positions at InterMune Pharmaceuticals and Connetics Corporation. Mark earned his Bachelor’s degree from Hartwick College and his PhD in Cellular and Molecular Biology from the University of Vermont. He was the recipient of a National MS Society postdoctoral fellowship at Stanford University.

David Glass
David Glass
Technology Transfer Consultant
University of Massachusetts Dartmouth & D. Glass Associates, Inc.
 
David Glass
David Glass
Technology Transfer Consultant
University of Massachusetts Dartmouth & D. Glass Associates, Inc.
 
About Speaker:

David Glass is an independent consultant with over thirty years’ experience in biotechnology and related life sciences fields, with much of that time devoted to patents, licensing and technology transfer. Dr. Glass has served as associate director of the tech transfer office at Massachusetts General Hospital, and has also managed technology transfer as a consultant or part-time employee at other academic institutions including McLean Hospital, Joslin Diabetes Center, and Baystate Medical Center. Since 2014, he has directed tech transfer as a consultant at the University of Massachusetts at Dartmouth, and he recently completed a one-year assignment as interim licensing manager at Partners HealthCare Innovation, managing and licensing neuroscience and psychiatric technologies from McLean Hospital and the MGH Psychiatry Department. Dr. Glass has also consulted for several other academic institutions and life sciences companies on various aspects of technology licensing and academic-industry partnering., He is a longstanding member of LES and AUTM, and was awarded the Certified Licensing Professional certification in July 2008 (recertified July 2011, expired June 2014) and the Registered Technology Transfer Professional certification in November 2013 (active). He holds a B.S. from Cornell University and a Ph.D. from Princeton University.

Shafique 	 Virani
Shafique Virani
Chief Executive Officer
BridgeBio
 
Shafique 	 Virani
Shafique Virani
Chief Executive Officer
BridgeBio
 
About Speaker:

Shafique Virani is currently CEO in Residence at BridgeBio, LLC based in Palo Alto, CA. Until June 2017, he was Vice President, Global Head of Neuroscience, Ophthalmology & Rare disease (NORD) Roche Partnering. Shafique’s responsibilities in this role encompassed all partnering activities within Roche’s NORD franchise, from academic collaborations to licensing and acquisitions. Shafique joined Roche in 2004 and served various roles within medical affairs, marketing and business development in the UK, USA and Switzerland until 2012, when he transitioned to South San Francisco. Shafique is trained as a neurosurgeon in Cambridge, UK and Boston, USA and holds the Fellowship of the Royal College of Surgeons of England.

Hans  Christinger
Hans Christinger
Vice President, Business Development & Alliance Management
Ovid Therapeutics
 
Hans  Christinger
Hans Christinger
Vice President, Business Development & Alliance Management
Ovid Therapeutics
 
About Speaker:

Hans Christinger, Vice President Business Development & Alliance Management at Ovid Therapeutics. Hans is responsible for Ovid business development activities to expand the company’s portfolio of Bold Medicines for rare neurological disorders through strategic partnerships. Additionally, Hans is responsible for the alliance management of Ovid’s relationships including Takeda and Lundbeck.

Hans has more than 25 years of bio-pharmaceutical experience, primarily focused on partnering, licensing (in- and out), and acquisitions as well as divestments. Hans has been based both in the US and abroad. He has led and executed numerous transactions at Ophthotech, Abbott, Roche and Vertex. Hans has established and led teams enabling global, regional and local deals across more than 30 countries in emerging and developed markets. He is currently serving on several advisory boards to early-staged biotechnology companies.

Hans began his career as a bench scientist at Genentech where he authored 15 structural biology related peer-reviewed papers. He earned a Master of Business Administration from Babson College, a Master of Science in Chemistry from San Francisco State University and a Bachelor of Science in Biochemistry from the University of Vermont.

Panel Moderator:
Michael  McGurk
Michael McGurk
Partner
DLA Piper
 
Michael  McGurk
Michael McGurk
Partner
DLA Piper
 
About Speaker:

Mike McGurk has more than 30 years of experience in virtually all
aspects of patent law. Working with clients ranging from small startups
to large Fortune 100 companies, he provides counsel in due
diligence matters, global portfolio development and adversarial
proceedings, including litigation, post-grant reviews, interferences
and re-examinations. Mike works primarily with pharmaceutical,
chemical, medical device and mechanical technology businesses.
Mike's practice is informed by his background as a patent examiner
in the chemical arts for the United States Patent and Trademark
Office (USPTO), as a litigator for ten years, and as an expert in due
diligence matters. He regularly provides strategic litigation
counseling support to clients across the nation, and has handled
numerous patent matters before the Patent Trial and Appeal Board
of the USPTO. His extensive due diligence experience includes prelitigation,
acquisition, in-license, freedom-to-operate product and/or process clearance, design around,
validity and patentability matters. Mike works with teams having the appropriate technical and legal abilities.

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12:20
Lunch & “Ready, Set, Grow – New Company Technology Pitch Competition”
1
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Ready, Set, Grow – New Company Technology Pitch Competition
Moderator: Ginger Johnson, Defined Health
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12:25
Reviewers & Judging Panelists:
Kevin  Bitterman
Kevin Bitterman
Partner
Atlas Venture
 
Kevin  Bitterman
Kevin Bitterman
Partner
Atlas Venture
 
About Speaker:

Kevin Bitterman focuses on creating and investing in companies that translate groundbreaking science into innovative medicines.

Kevin was the founding CEO of Editas Medicine (NASDAQ: EDIT), Morphic Therapeutic and Visterra, and co-founded Genocea Biosciences (NASDAQ: GNCA). He previously served as a director of InSeal Medical, Kala Pharmaceuticals, Neuronetics, Taris Biomedical (acquired by Allergan) and Vets First Choice among other ventures. Prior to joining Atlas Venture in 2017, Kevin was a partner at Polaris Partners, where he had been a member of the healthcare team since 2004.

Kevin is active in the local life science and healthcare start-up community, serving on the Scientific Advisory Board of the Massachusetts Life Sciences Center (MLSC) and as Board Chair of the New England Venture Capital Association (NEVCA).  He received a BA in biology, summa cum laude, from Rutgers University before completing his PhD in genetics at Harvard Medical School.  He has published numerous scientific articles and is an inventor on several issued patents.

Outside of work Kevin enjoys spending time with his wife and two young daughters.  He also enjoys trail running and whiskey, though generally not at the same time.

Barbara  Tate
Barbara Tate
Venture Partner
Dementia Discovery Fund/SV Life Sciences
 
Barbara  Tate
Barbara Tate
Venture Partner
Dementia Discovery Fund/SV Life Sciences
 
About Speaker:

Dr. Barbara Tate is a neuroscientist who has worked in both large and small pharmaceutical companies. Prior to joining DDF, she was Vice President and Head of Biology at Rodin Therapeutics, an Atlas Venture company, where she also consulted on other portfolio companies. Prior to Rodin, Barbara was Vice President for Research at Satori Pharmaceuticals, a biotech company developing a treatment for Alzheimer’s disease. At Pfizer, Barbara managed the neurodegenerative disease area group in Groton, CT. Prior to working in industry, Barbara was an academic researcher at Brown Medical School and Harvard Medical School. Barbara has a BA in Biology, a PhD in Physiology, and she was a postdoctoral fellow at Harvard Medical School.

Christine Brennan
Christine Brennan
Partner
MRL Ventures Fund
 
Christine Brennan
Christine Brennan
Partner
MRL Ventures Fund
 
About Speaker:

Dr. Brennan joined MRL Ventures Fund in June 2017. Prior to joining MRL Ventures Fund she spent 3 years as Principal at Novartis Venture Fund (NVF). Prior to NVF, she spent 3 years at Vitae Pharmaceuticals as Chief Business Officer responsible for strategy & business development. Prior to Vitae, she spent 5 years at Novartis Institutes for BioMedical Research in Strategic Alliances, most recently as Executive Director and Head of Strategy & Operations. Prior to Novartis, she held positions in business development and marketing at the biopharmaceutical companies, EnVivo Pharmaceuticals (now FORUM Pharmaceuticals), Biovail (now Valeant Pharmaceuticals) and Cogent Neuroscience. In addition, she was Director at Fidelity Biosciences Group (now F-Prime Capital Partners), a venture capital company. She received her Ph.D. in neuroscience from Dartmouth Medical School in 1995 and completed a postdoctoral fellowship at the National Institutes of Health in 1999.

Kiran  Reddy
Kiran Reddy
Partner
Clarus Ventures
 
Kiran  Reddy
Kiran Reddy
Partner
Clarus Ventures
 
About Speaker:

Kiran Reddy is the President & CEO of a Praxis Precision Medicines, a recently launched biotech company focused on precision medicine in genetically defined epilepsy and autism disorders. He is also a Venture Partner at Clarus Ventures where he focuses on new company formation, venture investments, and risk sharing partnerships with pharmaceutical companies.

Prior to Clarus, Kiran was at Biogen and led the CAPITA team within Corporate Development where he focused on sourcing new technologies and product opportunities to support the Company's growth via acquisitions, partnerships, and equity investments.

Prior to Biogen, Kiran was an Associate Partner at Third Rock Ventures. He supported and managed various portfolio companies in addition to focusing on new company formation and new investments.

Kiran was a key member of the founding team at SAGE Therapeutics, and served as its CBO and CMO through its IPO (NASDAQ: SAGE). He is also the co-inventor of SAGE-547 the Phase 3 program for an orphan epilepsy disorder and postpartum depression. SAGE has a market capitalization of ~$3.0B USD with greater than 250 employees, and named 2016 “Best places to work” in Cambridge, Massachusetts.

In addition, while at Third Rock, Kiran was part of the team that launched Foundation Medicine [NASDAQ: FMI] that has a market capitalization of greater than $1.0B USD and greater than 275 employees.

Before Third Rock Ventures, Kiran was a management consultant at the Lewin Group within in the biotechnology and pharmaceutical practice, and advised clients on clinical development and commercial strategy. Kiran holds MD and MBA degrees from Georgetown University. He completed his internship in medicine and his neurology residency at Harvard / Massachusetts General Hospital and is a board certified neurologist. Kiran was previously a Howard Hughes science fellow, and has authored several peer-reviewed scientific papers in the field of  epilepsy, neuroimmunology and neurodegenerative diseases. 

 

Ole  Isacson
Ole Isacson
Professor of Neurology
Harvard Medical School
 
Ole  Isacson
Ole Isacson
Professor of Neurology
Harvard Medical School
 
About Speaker:

Dr. Ole Isacson is Professor of Neurology (Neuroscience) at Harvard Medical School and is the Founding Director of the Neuroregeneration Research Institute at McLean Hospital. Since 1990, Prof. Isacson’s laboratory at Harvard has focused on the understanding and treatments of neurodegenerative disease, with particular emphasis on distinguishing critical mechanisms and treatments of neuronal vulnerability before the full onset of disease, or new restorative treatments using stem cell derived neurons after symptoms. Prof. Isacson is Principal Faculty of the Harvard Stem Cell Institute since it was founded in 2005. He is a member of the Scientific Advisory Board of the Michael J. Fox Foundation and member of the Executive Scientific Advisory Board (2014-2016). He has received several international prizes, research awards and lectureships. Prof. Isacson served as an advisory committee member at the FDA Center for Biologics Evaluation and Research (CBER) (2014-2016). He also served as SVP & the Chief Scientific Officer of Pfizer’s former Neuroscience R&D Unit (2016-2017). He is the author or co-author of over 350 scientific research articles and 3 books in his field. He was the Editor-in-Chief of Molecular and Cellular Neuroscience from 2010-2016, and currently serves on the editorial board. Prof. Isacson was elected fellow of the American Association for the Advancement of Science (AAAS) in 2013.

New Company Technology Pitch Presenters:
Joshua  Sestak
Joshua Sestak
President and Chief Scientific Officer
Orion BioScience
 
Joshua  Sestak
Joshua Sestak
President and Chief Scientific Officer
Orion BioScience
 
About Speaker:

Inventor of Orion technologies with industry formulations and regulatory experience. Recipient of Madison and Lila Self and Institute for Advancing Medical Innovation Graduate Fellowships and a 2013 Pipeline Fellow.

Kent  Werner
Kent Werner
Chief Executive Officer
Cogentis Therapeutics
 
Kent  Werner
Kent Werner
Chief Executive Officer
Cogentis Therapeutics
 
About Speaker:

Kent Werner is a board-certified neurologist and neuroscientist.  He holds Assistant Professor appointments in the Departments of Neurology at the Uniformed Services University and adjunct at Johns Hopkins University. He has 14 years of experience in biomedical research and seven years in clinical training. Kent received his MD and PhD in Cellular and Molecular Medicine in the department of Neuroscience in 2012 at the Johns Hopkins University under Dr. Solomon Snyder. His areas of research include  neurodegenerative conditions to include dementia and traumatic brain injury and their relationship to sleep.

Bill  Kohlbrenner
Bill Kohlbrenner
Chief Executive Officer
NeuroLucent
 
Bill  Kohlbrenner
Bill Kohlbrenner
Chief Executive Officer
NeuroLucent
 
About Speaker:

Dr. Kohlbrenner joined NeuroLucent as President and CEO in June 2017 after serving several months as an advisor.  His primary responsibility is helping to develop and implement a small-molecule drug discovery program aimed at bringing NeuroLucent’s early-stage AD program to the clinic.  He is also CSO of Life Science Nation (since 2014), which is an emerging company that provides a suite of tools and services to life sciences entrepreneurs seeking investment capital.  Prior to joining LSN, Bill was a business development director at AbbVie (formerly Abbott pharma) where he led a global scouting team with the mission of identifying and pursuing external research opportunities and early-stage assets around the globe.  Earlier in his career, Bill led drug discovery programs in the oncology, antiviral and antibacterial areas (AbbVie and Pfizer).  He has extensive experience managing collaborations, outsourcing and business development. 

Ambuj  Singh
Ambuj Singh
President & Chief Executive Officer
Acelot
 
Ambuj  Singh
Ambuj Singh
President & Chief Executive Officer
Acelot
 
About Speaker:

The founder of Acelot, Prof. Singh, has authored over 140 scientific papers in the areas of bioinformatics, data mining, databases, and graph algorithms. He holds a Ph.D. from the University of Texas at Austin and a Bachelor's degree from the Indian Institute of Technology. He is currently a professor in the departments of Computer Science and Biomolecular Science & Engineering at the University of California, Santa Barbara. He works on developing the basic data and graph analysis methods that form the core IP of the company.

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Keynote Panel Discussion
Moderator: Phyllis Barkman Ferrell, Eli Lilly
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3:20
Conference Concludes