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Inflammatory & Immunological Biomarkers

2017-04-282018-02-212018-01-27
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The 2018 agenda is currently being formed.

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BELOW IS THE AGENDA FROM 2017.

2018 Agenda
Day 1 - Monday, March 20th, 2017
7:00
Continental Breakfast & Registration
8:00
Opening Remarks
Overcoming Challenges of Clinical Validation & Translation
Moderator: Xuemei Zhao, Merck
9:00
Overcoming Challenges in Early Detection of Ovarian Cancer Trials
 
Steven Skates
Associate Professor, Medicine
Harvard Medical School
About Speaker: Steven Skates received his PhD in statistics from the University of Chicago. He joined the Internal Medicine Unit and Radiation Oncology at Massachusetts General Hospital and reviewed a study on the early detection of ovarian cancer using a CA125 cut... Read Full Bio 
 
 
Steven Skates
Associate Professor, Medicine
Harvard Medical School
 
About Speaker:

Steven Skates received his PhD in statistics from the University of Chicago. He joined the Internal Medicine Unit and Radiation Oncology at Massachusetts General Hospital and reviewed a study on the early detection of ovarian cancer using a CA125 cutoff for referral to ultrasound. Since then he has developed algorithms for the early detection of ovarian cancer leveraging the information in longitudinal CA125 values, implemented them in five early detection trials in the US and UK, and led an NCI Early Detection Research Network Biomarker Developmental Laboratory using genomic, proteomic, and longitudinal approaches for discovery and validation of early detection ovarian cancer markers.

 
Abstract: Because ovarian cancer has a low incidence of 1 in 2,500 per year in postmenopausal women, the number of women-years required for screening trials ...Read More 

Because ovarian cancer has a low incidence of 1 in 2,500 per year in postmenopausal women, the number of women-years required for screening trials is much larger than screening trials for cancers with much higher incidence including breast, colorectal, and prostate cancers. Either large cohorts or long-term trials or both qualities are needed. A sequence of trials may be needed to establish success in intermediate endpoints such as specificity, positive predictive value, sensitivity, before establishing a trial of the size required to have ovarian cancer mortality as the endpoint. Such a sequence is possible but takes 30 years to accomplish.

Trials in high risk women require a definition of increased risk due to inherited mutations. BRCA1/2 mutation carriers are the highest risk. The list of mutations associated with ovarian cancer risk has increased over the past decade. What the ideal target population is may be approached through enrolling women with a strong family history of ovarian and/or breast cancer, however this will require a larger size than if women with known relevant mutations are enrolled. The endpoint of sensitivity for early stage disease is likely the final endpoint since mortality will require a randomized trial and the ethics of offering no screening to high risk women who choose to retain their ovaries is questionable. With risk reducing salpingo-oophorectomy showing reduction of ovarian cancer risk to baseline levels, the role of screening for ovarian cancer in high risk women becomes significantly reduced.

The benefits of this presentation will be an appreciation of the size and time required to undertake research into early detection of ovarian cancer, the changing landscape over long term trials resulting in changing clinical roles for early detection, and the uncertainty of whether screening reduces mortality in high risk populations will always appear to remain.

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9:25
Translating Protein MS Assays into the Clinical Laboratory
 
Dobrin Nedelkov
Scientist, Molecular Biomarkers Laboratory
Biodesign Institute, Arizona State University
About Speaker: Dr. Dobrin Nedelkov is a Scientist and Group Leader of the Molecular Biomarkers Unit in the Biodesign Institute at Arizona State University. He is also a Founding President of the Institute for Population Proteomics. Dr. Nedelkov received his Ph.D... Read Full Bio 
 
 
Dobrin Nedelkov
Scientist, Molecular Biomarkers Laboratory
Biodesign Institute, Arizona State University
 
About Speaker:

Dr. Dobrin Nedelkov is a Scientist and Group Leader of the Molecular Biomarkers Unit in the Biodesign Institute at Arizona State University. He is also a Founding President of the Institute for Population Proteomics.
Dr. Nedelkov received his Ph.D. in Chemistry and Biochemistry from Arizona State University in 1997. After spending a year as a Postdoctoral Associate at Yale University, Dr. Nedelkov joined Intrinsic Bioprobes where over the span of 14 years he progressed through the ranks to ultimately become Scientific Director and CEO of the company, leading the early efforts in technology development for targeted proteomics and protein biomarkers. In 2013 he joined the Biodesign Institute at Arizona State University where he is leading research in proteoform biomarker discovery, validation, and translation.
Dr. Nedelkov has authored over 90 peer reviewed scientific articles, presented at over 60 scientific meetings, has 4 issued patents and several pending patent applications, and has served as a principal investigator on numerous NIH grants.

 
Abstract: Protein MS assays are assured to be the next-generation tests for precise and enabling measurement of clinical protein...Read More 

Protein MS assays are assured to be the next-generation tests for precise and enabling measurement of clinical protein biomarkers. Or are they? In the 30 years after the MALDI and ESI invention, only a dozen or so protein MS tests have been translated into clinical laboratories. Analytical performance requirements have been in place for some time, along with small molecules MS clinical tests precedents, so why haven’t more protein MS assays found their ways into clinical labs? Is there anything else missing? What about the clinical and economic drivers? If some of these key drivers have not been met yet, are we to proceed with the protein MS tests translation anyway, anticipating near-term clinical adoption? How do we then pick the biomarker targets for these tests? Many players have a stake in the clinical protein MS tests– from reagents and instruments manufacturers, to clinical labs and diagnostic companies. This presentation will focus on these key aspects of protein MS assays translation that may help us answer the ultimate question: Are clinical protein MS tests prophetic or just rhetoric?

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9:50
Morning Networking Break
10:30
Overcoming Challenges of Clinical Biomarker Development: Clinical Biomarker Assay Development/Validation and Clinical Biomarker Qualification
 
Xuemei Zhao
Senior Principal Scientist
Merck
About Speaker: Xuemei Zhao obtained her Ph.D. in Chemistry at Columbia University and did her postdoctoral training at Cold Spring Harbor Laboratory.  Afterwards, Xuemei joined the Proteomics Department at Merck Research Laboratories.  Her group was responsible f... Read Full Bio 
 
 
Xuemei Zhao
Senior Principal Scientist
Merck
 
About Speaker:

Xuemei Zhao obtained her Ph.D. in Chemistry at Columbia University and did her postdoctoral training at Cold Spring Harbor Laboratory.  Afterwards, Xuemei joined the Proteomics Department at Merck Research Laboratories.  Her group was responsible for biochemical sample preparation for LC-MS based proteomics profiling in biomarker discovery and new target identification.  Xuemei then moved on to focus on clinical biomarker development and implementation.  Currently, Xuemei leads the immunoassay group in Translational Molecular Biomarkers to support clinical programs across all disease areas at Merck.

 
Abstract: The utility of various types of clinical biomarkers is to aid decision-making in drug development. After a pre-clinica...Read More 

The utility of various types of clinical biomarkers is to aid decision-making in drug development. After a pre-clinical candidate is approved for development in clinical studies, biomarker activities are transitioned from discovery to clinical biomarker development and implementation. Clinical biomarker development includes clinical biomarker assay development, fit-for-purpose assay validation, and clinical biomarker qualification in clinical studies. After a clinical biomarker is qualified, it will then be implemented in clinical studies to aid decision-making. Overcoming challenges of clinical biomarker development will be discussed.

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Marica Grskovic
Associate Director, R&D
CareDx
About Speaker: Marica Grskovic, Ph.D. is Associate Director, R&D at CareDx, a California based molecular diagnostics company focused on the discovery, development and commercialization of clinically differentiated, high-value, non-invasive diagnostic surveillan... Read Full Bio 
 
 
Marica Grskovic
Associate Director, R&D
CareDx
 
About Speaker:

Marica Grskovic, Ph.D. is Associate Director, R&D at CareDx, a California based molecular diagnostics company focused on the discovery, development and commercialization of clinically differentiated, high-value, non-invasive diagnostic surveillance solutions for organ transplants. Since joining CareDx in 2012, she has been instrumental in leading the research and development of cell-free DNA, NGS-based diagnostic solutions for rejection surveillance and immunosupressoin monitoring in transplant patients.

Dr. Grskovic received her Ph. D. in Molecular Biology and Biochemistry from the European Molecular Biology Laboratory and University of Heidelberg, Germany.

 
Abstract: Organ transplant patients require lifelong immunosuppression that necessitates a finely tuned therapeutic strategy.  The threat of allograft reje...Read More 

Organ transplant patients require lifelong immunosuppression that necessitates a finely tuned therapeutic strategy.  The threat of allograft rejection from sub-optimal dosing must be balanced against increased risk of infections and cancer from excessive dosing. Immunosuppression drug regimens are mostly “one-size fits all” and this imprecision of care has been reported as the primary reason for increased infections and malignancies of these patients. A significant unmet medical need exists for clinical diagnostic tools to enable surveillance management of transplant patients and improve the long-term outcome of immunosuppressive therapy.

Cell-free DNA (cfDNA) has been described as a biomarker for prenatal testing, cancer, and organ transplantation, each of which present different clinical and technological challenges. cfDNA circulating in the plasma of transplant recipients represents a mixture of recipient cfDNA and residual nucleosome-protected genomic regions released from dying cells of the allograft (“transgenome”). The genomes of the organ donor and allograft recipient are distinguishable by sequencing total cfDNA from the plasma. A clinical-grade cfDNA  NextGen sequencing (NGS) assay was developed and rigorously analytically validated to monitor the levels of the “transgenome”, enabling assessment of the allograft status of transplant recipients. The NGS-based assay does not require testing of genetic material from the donor or recipient thereby simplifying the testing of transplants with cadaveric donors.  Longitudinal samples from heart, lung and kidney transplant patients had higher dd-cfDNA levels at biopsy-confirmed rejection which were reduced following adjustments to immunosuppressive therapy in clinical validation studies. Serial assessment of dd‑cfDNA provides a measure of both the amount and kinetics of dying allograft cells, information clinicians may use to inform clinical utility. 

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11:20
Colorectal Cancer Screening: Advances and Challenges
 
Anuraag Shrivastav
Associate Professor, Department of Biology
University of Winnipeg
About Speaker: Dr. Anuraag Shrivastav completed his PhD in 2002 from the Banaras Hindu University, India. He received postdoctoral fellowship from Canadian Institutes of Health Research and obtained postdoctoral training at the department of Pathology and Laborator... Read Full Bio 
 
 
Anuraag Shrivastav
Associate Professor, Department of Biology
University of Winnipeg
 
About Speaker:

Dr. Anuraag Shrivastav completed his PhD in 2002 from the Banaras Hindu University, India. He received postdoctoral fellowship from Canadian Institutes of Health Research and obtained postdoctoral training at the department of Pathology and Laboratory Medicine and Saskatchewan Cancer Agency, University of Saskatchewan, Canada. Dr Shrivastav is a faculty member at the University of Winnipeg and adjunct member at the University of Manitoba and CancerCare Manitoba. He has published over 40 peer-reviewed articles in International journals of repute. His laboratory is engaged in studying cellular signaling mechanisms that are intricately linked to homeostasis that control cell proliferation, survival and death in cancer cells. Currently, his research group is actively pursuing scientific research to identify novel markers for CRC screening and early detection. Dr. Shrivastav is also Principal, VastCon Inc – A University of Winnipeg spinoff dedicated to developing novel biomarkers based simple prognostic/diagnostic tests.

 
Abstract: Colorectal cancer (CRC) is the third leading cause of cancer deaths in North America and is therefore a significant public health problem. Most cas...Read More 

Colorectal cancer (CRC) is the third leading cause of cancer deaths in North America and is therefore a significant public health problem. Most cases of CRC arise from pre-malignant adenomatous polyps and the tumor grows slowly over many years, thus providing a long window of opportunity for detection and effective treatment at pre-malignant or early malignant stages. CRC is the most preventable yet least prevented form of cancer due to lack of an effective screening test. The most commonly recommended screening tests for CRC are fecal occult blood testing, sigmoidoscopy and colonoscopy. These screening tests have several limitations including limited efficacy, invasive nature of the test, and/or poor acceptability in the population. Colonoscopy remains the “gold standard” test that has unique feature of allowing removal of any polyps that are present. Colonoscopy is both invasive and a resource intensive test. Given the high mortality rates in CRC patients, there is an urgent need for a convenient, more accurate and low-cost screening test that could triage patients for more intensive procedures such as colonoscopy. This should improve patient compliance and clinical outcomes. Signaling molecules regulating cell activation, proliferation, differentiation and transformation are attractive molecular markers for screening and/or prognosis of cancers. We have discovered biomarkers in the peripheral blood of CRC patients, which may aid in developing an efficacious blood test for screening CRC

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11:45
Lunch Sponsored by Trial Runners
Emerging Technologies in Biomarker Development
Moderator: Sandip Patel, University of California, San Diego
1:15
Flow Cytometry at the Point of Care : Emerging Trends
 
Lisa Patti-Diaz
Principal Scientist
Bristol-Myers Squibb
About Speaker: Lisa Patti-Diaz, MT (ASCP) is a Prinicipal Scientist at Bristol Myers Squib with 17 years experience specializing in developing, validating, and managing flow cytometry assays for biomarker analysis in clinical trials.   Lisa has contributed to bio... Read Full Bio 
 
 
Lisa Patti-Diaz
Principal Scientist
Bristol-Myers Squibb
 
About Speaker:

Lisa Patti-Diaz, MT (ASCP) is a Prinicipal Scientist at Bristol Myers Squib with 17 years experience specializing in developing, validating, and managing flow cytometry assays for biomarker analysis in clinical trials.   Lisa has contributed to biomarker programs in multiple therapuetic areas including immuno-oncology, virology and immunoscience.  Most recently, Lisa has been exploring new technologies that could enable clinical sites to process cells and perform diagnostic flow cytometry-based assays, locally.   Prior to joining BMS, Lisa’s background includes 15 years in clinical laboratory medicine, including oversight of CAP/CLIA certification requirements and educating clinical laboratory scientists.

 
Abstract: Flow cytomety has a rich history of being used for exploratory research to identify and monitor cell subsets and protein/receptor expression levels...Read More 

Flow cytomety has a rich history of being used for exploratory research to identify and monitor cell subsets and protein/receptor expression levels as biomarkers in disease and with pharmaceutical intervention.   As an assay platform, flow cytometry has taken divergent paths of evolution.  Most obvious is the development of multi-laser instruments with high complexity, multi-color fluorescent detectors which can give researchers deeper insight into periphery and patient immune status.  This level of flexibility and complexity is invaluable for discovery and research applications, however, it has been prohibitive to utilizing flow cytometry for point-of-care, diagnostic testing.   Behind the color explosion, there have been advances to simplify smaller flow cytometry-based systems to enable point-of-care sample analysis with timely delivery of results that can be actionable in the clinic.  Compact solid state lasers, micro-fabrication, and microfluidics are innovations incorporated in these alternative portable cytometers.  This presentation will describe some of the systems presently available and also take a glimpse into the potential future direction of diagnostic cytometry.

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1:40
Immunosignatures Applied to Early Disease Detection, High Resolution of Disease, Prognosis and Target Identification
 
Stephen Albert Johnston
Director, Center for Innovations in Medicine; Professor, School of Life Sciences, Arizona State University
CEO, Calviri
About Speaker: Dr. Johnston directs the Center for Innovations in Medicine (CIM) at the Biodesign Institute. CIM is unique in its focus on inventing disruptive technologies in biomedicine. The mission is to contribute to the transformation of medicine through techn... Read Full Bio 
 
 
Stephen Albert Johnston
Director, Center for Innovations in Medicine; Professor, School of Life Sciences, Arizona State University
CEO, Calviri
 
About Speaker:

Dr. Johnston directs the Center for Innovations in Medicine (CIM) at the Biodesign Institute. CIM is unique in its focus on inventing disruptive technologies in biomedicine. The mission is to contribute to the transformation of medicine through technologies that allow prevention, early detection and new therapeutic treatment of disease. Toward this goal CIM is focusing on developing three of his inventions. One project is to create a universal, preventative cancer vaccine. A second is to develop a system for continuous, comprehensive, cheap health monitoring. The third is based on an invention for making new therapeutics and targeted anti-infectives.
Johnston largely focuses on invention. He was inventor/ co-inventor of pathogen derived resistance, mitochondrial transformation, TEV protease system, the gene gun, gene immunization, expression library immunization, linear expression elements, synbodies and immunosignaturing. He was professor and director of the Center for Biomedical Inventions at UT-Southwestern Medical Center and Professor of Biology and Biomedical Engineering at Duke University before moving to ASU.

 
Abstract: Immunosignatures are a simple, broadly applicable method to profile health status. The technique is based on using pep...Read More 

Immunosignatures are a simple, broadly applicable method to profile health status. The technique is based on using peptide chips to unbiasedly signature the antibody composition in an individual. The technology is being commercialized by HealthTell, Inc. I will provide examples of the application of IMS to detect disease early, resolve difficult to diagnose diseases, provide prognostic information for therapy and to even to identify potential therapeutic targets.

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2:05
Bacterial Nucleases as Signal-Amplifying Biomarkers for Infectious Disease Diagnostics
 
James McNamara
Associate Professor, Department of Internal Medicine
University of Iowa
About Speaker: Dr. McNamara studied Chemical Engineering at the University of Virginia (B.S., 1992), Neurobiology at Duke University (Ph.D., 2003) and then completed a postdoc at Duke where he developed RNA based therapeutic approaches for cancer. He joined the Dep... Read Full Bio 
 
 
James McNamara
Associate Professor, Department of Internal Medicine
University of Iowa
 
About Speaker:

Dr. McNamara studied Chemical Engineering at the University of Virginia (B.S., 1992), Neurobiology at Duke University (Ph.D., 2003) and then completed a postdoc at Duke where he developed RNA based therapeutic approaches for cancer. He joined the Department of Internal Medicine at the University of Iowa in 2007. Dr. McNamara’s research is focused on developing rapid clinical diagnostic assays for bacterial infectious diseases based on selective detection of pathogen-derived nuclease activities.

 
Abstract: Culture-based assays are currently the primary means for diagnosing many bacterial infections. These assays are time-c...Read More 

Culture-based assays are currently the primary means for diagnosing many bacterial infections. These assays are time-consuming, typically taking a day or longer. Here, I will describe a platform technology that exploits the unique properties of various bacterial nucleases to rapidly detect them, thus revealing the presence of the bacterial pathogens that generate them. This platform uses quenched fluorescent oligonucleotide probes to enable the rapid detection of specific bacterial nuclease targets. My group has developed several applications of this technology, including rapid in vitro diagnostic assays for common infections and a noninvasive optical imaging approach for focal infections. The promising results of this work suggest that this nuclease-detecting approach could enable the more effective management of various problematic infections.

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2:30
 
Sandip Patel
Assistant Professor, Cancer Immunotherapy Program
University of California, San Diego
About Speaker: Sandip Patel, MD. Assistant Professor, Medical Oncology/Hematology, UC San Diego Moores Cancer Center Dr. Sandip Patel, MD is a medical oncologist focused on early development of novel immunotherapy, in particular early phase clinical trials of ... Read Full Bio 
 
 
Sandip Patel
Assistant Professor, Cancer Immunotherapy Program
University of California, San Diego
 
About Speaker:

Sandip Patel, MD. Assistant Professor, Medical Oncology/Hematology, UC San Diego Moores Cancer Center

Dr. Sandip Patel, MD is a medical oncologist focused on early development of novel immunotherapy, in particular early phase clinical trials of cancer immunotherapy and thoracic oncology immunotherapy trials. His research focus is on predictive biomarkers for immunotherapeutic response and generation of personalized cancer immunotherapy regimens.

He is Assistant Director of the Clinical Trials Office at UCSD Moores Cancer Center and a member of the Cancer Immunotherapy, Experimental Therapeutics (Phase 1), and Thoracic Oncology Programs. Dr. Patel earned his medical degree at Baylor College of Medicine, while performing research at MD Anderson Cancer Center. He completed a residency in Internal Medicine at UCLA Medical Center. He completed a fellowship in Medical Oncology and Hematology at Duke University Medical Center. He is triple board-certified in internal medicine, medical oncology, and hematology.

 
Abstract: Predictive biomarkers for immunotherapeutic response in cancer, with a focus on novel biomarker assays in tumor and blood. • Nuances...Read More 
  • Predictive biomarkers for immunotherapeutic response in cancer, with a focus on novel biomarker assays in tumor and blood.
    • Nuances in the development of PD-L1 IHC with a focus on alternative predictive biomarkers that may better determine patient response to
    immune checkpoint modulation.
    • Next generation cancer immunotherapeutics currently under development, including cell-based approaches dependent on personalized target discovery
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2:55
Afternoon Networking Break
Big Data Analytics & Bioinformatics for Biomarkers Development
Moderator: Bin Li, Takeda Data Science Institute
3:25
Computational Biology Considerations for Translational Biomarker Research
 
Bin Li
Director of Computational Biology
Takeda Data Science Institute
About Speaker: Dr. Bin Li leads a computational biology team at Data Science Institute, Takeda Pharmaceutics. His team provides computational supports on translational medicine research for various Takeda compounds. They also develop methods, build predictive model... Read Full Bio 
 
 
Bin Li
Director of Computational Biology
Takeda Data Science Institute
 
About Speaker:

Dr. Bin Li leads a computational biology team at Data Science Institute, Takeda Pharmaceutics. His team provides computational supports on translational medicine research for various Takeda compounds. They also develop methods, build predictive models for biomarker and patient stratification needs, as well as build a cloud-based translational medicine data repository. His team is also responsible on method evaluation and pipeline building for various NGS platforms, including WES, RNA-seq, and targeted NGS panels.

 
Abstract: Goal: to incorporate computational biology considerations into predictive biomarker framework, for identifying transla...Read More 

Goal: to incorporate computational biology considerations into predictive biomarker framework, for identifying translational biomarkers for patient stratification and disease indication selections

What we want to gain from building the predictive models:

  • Build a predictive model on cell line data, which can be used for patients
  • Model derived signature genes reflects a drug’s MOA
  • Each drug’s predictive model should be drug specific
  • Using the predictive model for cancer indication selections
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3:50
Revealing Deep Diversity in the Human Proteome
 
Nuno Bandeira
Associate Professor, Computer Science and Engineering; Executive Director, Center for Computational Mass Spectrometry
University of California, San Diego
About Speaker: Nuno Bandeira received his B.S. in Computer Science (1997); New University of Lisbon, Portugal; M.Sc. in Applied Artificial Intelligence (2001); New University of Lisbon, Portugal; Ph. D. in Computer Science and Bioinformatics (2007); University of C... Read Full Bio 
 
 
Nuno Bandeira
Associate Professor, Computer Science and Engineering; Executive Director, Center for Computational Mass Spectrometry
University of California, San Diego
 
About Speaker:

Nuno Bandeira received his B.S. in Computer Science (1997); New University of Lisbon, Portugal; M.Sc. in Applied Artificial Intelligence (2001); New University of Lisbon, Portugal; Ph. D. in Computer Science and Bioinformatics (2007); University of California, San Diego.  Awards include 2006 Human Proteome Organization (HUPO) Young Investigator Award, 2007 Ph.D. Dissertation Award (CSE/UCSD), 2010 Genome Technology’s Tomorrow’s PI, 2012 Molecular BioSystems’s Emering Investigator and 2013 Sloan Research Fellowship. 

 

Nuno Bandeira, Ph.D. is an Associate Professor of Computer Science and Engineering at the Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California, San Diego is also the founding and current Executive Director of the NIH/NIGMS Center for Computational Mass Spectrometry at UCSD. His lab’s research focuses on big data algorithms and systems for worldwide interpretation of proteomics and metabolomics mass spectrometry data, including data from endogenous and digested peptides, discovery and localization of post-translational modifications, protein-protein interactions, sequencing of non-linear peptides with unknown amino acids and characterization of microbial, marine, reptile and plant natural products. As Executive Director of UCSD Center for Computational Mass Spectrometry, his research further extends to distributed algorithms for large scale data analysis (ProteoSAFe), sharing (MassIVE) and crowdsourced, community-wide interpretation (GnPS) of all publicly available mass spectrometry data.

 
Abstract: The long road to biomarker discovery starts with being able to determine that a specific molecule is present in a sample of interest – a task tha...Read More 

The long road to biomarker discovery starts with being able to determine that a specific molecule is present in a sample of interest – a task that is currently poorly addressed when it comes to the identification of polymorphic or post-translationally modified proteins or endogenous peptides.

Building on the growing availability of public mass spectrometry (MS) data, we implemented large scale searches of over 15 terabytes of data from a range of human samples analyzed with various types of instruments, thus more than doubling the volume of annotations in public human data and increasing the number of peptide spectrum matches by several fold.

Beyond identification of common, mostly-unmodified peptides, we show how spectral alignment algorithms can improve the confidence of peptide identifications by several orders of magnitude and use these to reveal dozens of unexpected putative modifications supported by multiple highly-correlated spectra. These further show that protein regions can be observed in over 50 different variants with various combinations of post-translational modifications and cleavage events, thus suggesting that current coverage of proteome diversity (at ~1.3 variants per protein region) is far below what can be observed in experimental data.

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4:15
Unbiased Mass-Spectrometry-Based Metabolomics Profiling in Over 1,000 Patients Reveals Specific Abnormalities in Biochemical Pathways in Patients with Atherosclerosis, as Defined by Cardiovascular CT-Based Precision Phenotyping in the Multi-Center GLOBAL Clinical Study
 
Szilard Voros
Founder & Chief Executive Officer
Global Genomics Group
About Speaker: Dr. Szilard Voros, MD, FACC, FSCCT, FAHA is a cardiologist, geneticist and entrepreneur. He is Founder and CEO of Global Genomics Group (“G3”), a precision-medicine-based biotechnology company, developing and commercializing diagnostic biomarkers... Read Full Bio 
 
 
Szilard Voros
Founder & Chief Executive Officer
Global Genomics Group
 
About Speaker:

Dr. Szilard Voros, MD, FACC, FSCCT, FAHA is a cardiologist, geneticist and entrepreneur. He is Founder and CEO of Global Genomics Group (“G3”), a precision-medicine-based biotechnology company, developing and commercializing diagnostic biomarkers and novel drug targets for common diseases. G3’s platform is based on the largest-ever program using DNA and RNA sequencing, proteomics, metabolomics, lipidomics and other platforms. G3 has developed a diagnostic blood test for cardiovascular disease and has identified several novel drug targets. Before G3, Voros was Chief Scientific Officer, Chief of Cardiovascular Prevention and Medical Director of Cardiovascular Imaging at Piedmont Heart Institute. He has published extensively in major scientific journals including Nature Reviews, New England Journal of Medicine, Annals of Internal Medicine, Journal of the American College of Cardiology and Circulation: Cardiovascular Genetics, etc.

 
Abstract: Introduction: While the contribution of lipids and lipoproteins to atherosclerosis is well-described, little is known about specific derangements i...Read More 

Introduction: While the contribution of lipids and lipoproteins to atherosclerosis is well-described, little is known about specific derangements in biochemical metabolic pathways that exist in such patients.
Methods: We performed a nested case:control study in the broader GLOBAL multi-center clinical study (NCT01738828) in a total of 1,096 subjects (age 59.9±0.55; 49% male) in three successive pre-defined cohorts. Since it is known that “time from last meal” affects metabolic profiles, we assessed differential metabolomic profiles two different ways: 1. Fasting and non-fasting subjects combined and 2. Fasting subjects alone; fasting was defined as fasting for 8 hours or more. Validation was performed in a 3rd, independent set of fasting subjects. Non-targeted mass spectrometry (MS) analysis was performed by GC-MS and UPLC-MS/MS (Metabolon, Inc.; Raleigh, NC). Non-contrast and contrast cardiac CT was performed at each participating clinical site based on study protocol and all images were analyzed by independent core laboratory with consensus reads. “Case” was defined as ANY plaque on non-contrast/contrast CT; all others were controls. Univariate analysis with hierarchal clustering and multivariable analysis with gradient boosting was used for analysis; q-value of 0.05 or less (adjusted for false discovery rate) was significant.
Results: MS analysis quantified 1,088 analytes and of these, 83 and 34 were nominally associated with case:control status in the Discovery 1 and 2 Cohorts, respectively. Gradient boosting identified the 8 analytes that were strongest predictors, representing different biochemical pathways (Table).
Conclusions: Comprehensive metabolic profiling in the largest metabolomics study of atherosclerosis to date using cardiac CT as precision phenotyping tool identified simultaneous derangements in amino acid, pentose, nucleotide, dipeptide and anti-oxidant pathways; these provide mechanistic insights and provide potential diagnostic tools for human coronary atherosclerosis.

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4:40
From the Bench to the Cluster: Enhancing the Signal to Noise in Placental ExRNA Biomarker Discovery
 
Peter De Hoff
Research Associate, Reproductive Medicine
University of California, San Diego
About Speaker: Research Associate, Sanford Consortium - Placental Dysfunction Biomarker Discovery Program Scientist II, Synthetic Genomics Inc. - Tools Development in Algal Biofuels Program Postdoc, Salk Institute for Biological Studies - Evolution of Mating Sy... Read Full Bio 
 
 
Peter De Hoff
Research Associate, Reproductive Medicine
University of California, San Diego
 
About Speaker:

Research Associate, Sanford Consortium - Placental Dysfunction Biomarker Discovery Program
Scientist II, Synthetic Genomics Inc. - Tools Development in Algal Biofuels Program
Postdoc, Salk Institute for Biological Studies - Evolution of Mating Systems in Green Algae
PhD, UCLA - Gene Discovery in Legume Rhizobium Symbiosis

5:05
Networking Reception & Poster Session
Day - 2 Tuesday, March 21st, 2017
7:30
Breakfast
Biomarkers of Early Progressive Inflammatory & Immunological Diseases
Moderator: Stephen Pennington, University College Dublin
 
Geert Schmid-Schonbein
Distinguished Professor and Chairman, Department of Bioengineering
University of California, San Diego
About Speaker: Geert W. Schmid-Schönbein is Distinguished Professor and Chairman of the Department of Bioengineering at the University of California San Diego.  He teaches bioengineering of living tissues and cell and molecular mechanics in heath and disease. His... Read Full Bio 
 
 
Geert Schmid-Schonbein
Distinguished Professor and Chairman, Department of Bioengineering
University of California, San Diego
 
About Speaker:

Geert W. Schmid-Schönbein is Distinguished Professor and Chairman of the Department of Bioengineering at the University of California San Diego.  He teaches bioengineering of living tissues and cell and molecular mechanics in heath and disease. His research interest is in molecular/cell mechanics and bioengineering analysis of the microcirculation in disease. His group discovered a fundamental mechanism for cell dysfunctions and inflammation due to “Auto-digestion”.

He is Founding Member of AIMBE, former President of the Biomedical Engineering Society, the Microcirculatory Society and the North American Society of Biorheology, Fellow of the American Heart Association, the Biomedical Engineering Society, the Physiological Society, and the International Federation for Medical and Biological Engineering. He is Past Chair of the World Council for Biomechanics and Member of the US National Academy of Engineering. 

Geert W. Schmid-Schönbein is Distinguished Professor and Chairman of the Department of Bioengineering at the University of California San Diego. He teaches bioengineering of living tissues and cell and molecular mechanics in heath and disease. His research interest is in molecular/cell mechanics and bioengineering analysis of the microcirculation in disease. His group discovered a fundamental mechanism for cell dysfunctions and inflammation due to “Auto-digestion”. He is Founding Member of AIMBE, former President of the Biomedical Engineering Society, the Microcirculatory Society and the North American Society of Biorheology, Fellow of the American Heart Association, the Biomedical Engineering Society, the Physiological Society, and the International Federation for Medical and Biological Engineering. He is Past Chair of the World Council for Biomechanics and Member of the US National Academy of Engineering.

 

 

 

 
Abstract: Our recent evidence shows that the central involvement of the intestine in many acute and chronic diseases (“gas...Read More 

Our recent evidence shows that the central involvement of the intestine in many acute and chronic diseases (“gastrointestinal co-morbidities”) is due to the powerful pancreatic digestive enzymes. While required in life for normal digestion they need to be compartmentalized inside the lumen of the intestine. But our evidence in acute forms of experimental shock indicates that the digestive enzymes escape out of the lumen of the intestine into the systemic circulation at concentrations sufficient to start autodigestion. The digestive enzymes cause extensive cell and organ dysfunctions and death. Enteral blockade of digestive enzymes in the intestine serves to reduce autodigestion, organ dysfunctions and mortality in shock. In chronic metabolic disease unchecked proteases may also be present in the systemic circulation at lower concentrations, still causing cell dysfunctions. For example, unchecked extracellular protease activity causes cleavage of the beta-2 adrenergic receptor in arterioles, contraction and elevation of central blood pressure. The protease activity also causes cleavage of the insulin receptor with insulin resistance and cleavage of many other receptors causing co-morbidities. These results indicate that autodigestion may be a fundamental mechanism for cell and organ dysfunction, disease and death. The digestive enzymes in the pancreas and intestine not only serve digestion of food but may also leak into the circulation and lead to autodigestion.

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Stephen Pennington
Professor of Proteomics
University College Dublin
About Speaker: Steve graduated from Imperial College of Science and Technology, University of London with a joint honours degree in Chemistry and Biochemistry before completing a PhD in Biochemistry at the University of Cambridge. During his PhD he was awarded an E... Read Full Bio 
 
 
Stephen Pennington
Professor of Proteomics
University College Dublin
 
About Speaker:

Steve graduated from Imperial College of Science and Technology, University of London with a joint honours degree in Chemistry and Biochemistry before completing a PhD in Biochemistry at the University of Cambridge. During his PhD he was awarded an Elmore Medical Research Fellowship and it was during this fellowship that his interests in the regulation of the mammalian cell cycle began. He was Wellcome Lecturer in the University of Liverpool before moving to Dublin where he is currently Professor of Proteomics in UCD. His research team use a range of proteomics platforms to explore disesase mechanisms and identify biomarkers in oncology and inflammatory disease. Stephen has been awarded a Beit Memorial Fellowship and received a Sir Henry Wellcome Commemorative Award for Innovative Research. He serves on the editorial boards of several journals, and is currenty vice-president of the British Society for Proteome Research, a general council member of the European Proteomics Association and lead organizer of the forthcoming annual congress of the Human Proteome Organisation - HUPO2017 (www.hupo2017.ie).

 
Abstract: The problem: For many diseases, deciding which patients to treat, when to treat them and what treatment to use r...Read More 
  • The problem: For many diseases, deciding which patients to treat, when to treat them and what treatment to use remains challenging. Despite much promise the use of proteomics for new protein biomarker discovery has not yielded many clinically used biomarker tests – why is this?
  • Start at the end: A pragmatic ‘real world’ and patient-centric strategy for the discovery, development and delivery of biomarkers of potential clinical utility will be introduced.
  • Solutions:The application of this strategy for tests to support clinical decisions in prostate cancer and psoriatic arthritis will be described.
  • Scaled for delivery: Potential platform approaches for the delivery of multiplexed protein biomarker tests will be explored.
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Yang Dai
Assistant Professor, Immunology & Microbial Science
The Scripps Research Institute
About Speaker: 2002, received a Ph.D. degree in Immunology from Memorial University of Newfoundland, St John’s, Canada. 2002-2008, postdoctoral fellow in Dr. Eli Sercarz’s lab in Torrey Pines Institute for Molecular Studies, training in immune regulation and T-... Read Full Bio 
 
 
Yang Dai
Assistant Professor, Immunology & Microbial Science
The Scripps Research Institute
 
About Speaker:

2002, received a Ph.D. degree in Immunology from Memorial University of Newfoundland, St John’s, Canada. 2002-2008, postdoctoral fellow in Dr. Eli Sercarz’s lab in Torrey Pines Institute for Molecular Studies, training in immune regulation and T-cell-mediated autoimmune disease. 2008, started independent research career, and studied secreted microvesicles or exosomes in triggering autoimmune responses and type 1 diabetes in non-obese diabetic mice. His lab found that exosomes are potent immunogen and carry unique autoantigens to stimulate autoreactive B and T cells. The findings have been published in Journal of Immunology, European Journal of Immunology and Diabetes. Recent progress in studying candidate autoantigens expressed in the exosomes demonstrated that endogenous retrovirus antigens are enriched in the exosomes and contribute to the autoimmune responses to pancreatic islets.

 
Abstract: Autoimmune-mediated destruction of pancreatic islets occurs long before the onset of type 1 diabetes (T1D). It is cruc...Read More 

Autoimmune-mediated destruction of pancreatic islets occurs long before the onset of type 1 diabetes (T1D). It is crucial to identify those highly susceptible and pre-diabetic individuals for disease prevention. However, current early diagnostic markers such as autoantibodies are not very specific and also difficult to monitor. We studied a new source of autoantigens, exosomes, which are nano-size vesicles secreted by cells, including islet cells. We found that exosomes are unique immunogen; it contains specific autoantigens that can be recognized by the autoantibodies and autoreactive T cells causing T1D. One candidate antigen is endogenous retrovirus (ERV) protein. I will present data collected from pre-diabetic non-obese diabetic (NOD) mice to support that ERV antigens and their cognate immune cells may be useful early diagnositic markers and could be also targeted for tolerance induction to prevent T1D.

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Martin Schwickart
Scientist II
MedImmune
About Speaker: Martin Schwickart, Ph.D. is currently Scientist II in the Department of Clinical Immunology and Bioanalysis at Medimmune in Mountain View, California. Martin leads assay development and data analysis of cellular and soluble biomarkers. He also develo... Read Full Bio 
 
 
Martin Schwickart
Scientist II
MedImmune
 
About Speaker:

Martin Schwickart, Ph.D. is currently Scientist II in the Department of Clinical Immunology and Bioanalysis at Medimmune in Mountain View, California. Martin leads assay development and data analysis of cellular and soluble biomarkers. He also develops bioanalytical plans including PK, ADA and Nab support. Martin performed his post-doctoral studies at Genentech and his doctoral studies at the Max-Planck Institute of Molecular Cell Biology and Genetics.

 
Abstract: Background/Purpose:...Read More 

Background/Purpose:  Recent work has demonstrated that a subset of RA patients has autoantibodies to peptidyl arginine deiminase 4 (PAD4) and anti-PAD3. PAD4 is a citrullinating enzyme involved in NETosis and the formation of autoantigens targeted by anti-citrullinated protein antibodies (ACPA). Autoantibodies to PAD2 were reported to be associated with protection against radiographic progression in patients with rheumatic arthritis. We explored whether anti-PAD4, anti-PAD3, and anti-PAD2 autoantibodies may be associated with disease severity or with clinical response to mavrilimumab.

Methods:  Novel assays to detect autoantibodies reactive with PAD4, PAD3 or PAD2 were developed and used to measure anti-PAD4, PAD3 and PAD2 levels in 288 patients enrolled in the phase 2a study CP219 and anti-PAD4 and PAD3 levels in 323 patients enrolled in the phase 2b study 1071. All patients in the study met the ACR criteria for a diagnosis of RA.

Results:  Overall, 35% of the RA patients tested positive for anti-PAD4 antibodies and 20% tested positive for anti-PAD3 antibodies; healthy volunteers were negative for both autoantibodies. We only detected very low levels of anti-PAD2 antibodies in RA patients and the incidence of anti-PAD2 positivity was similar to healthy controls. In the phase 2b study 1071 in DMARD-IR patients, the patients who tested positive for anti-PAD4 were enriched for the presence of ACPA (OR = 54.7, 95% CI = 7.5, 400.0), had higher baseline joint erosion (mean difference = 9.9, 95% CI = 2.6, 17.2) and higher modified total Sharp scores (mean difference = 16.2, 95% C.I. = 3.9, 28.5) than patients who tested negative. There was a significant treatment-biomarker effect across clinical endpoints ACR20, ACR50, ACR70 and change in DAS28-CRP. Patients treated with the 150 mg dose of mavrilimumab, the highest dose evaluated, and who tested negative for anti-PAD4 (68% of patients), had significantly greater benefit from mavrilimumab compared to active placebo (36.7% difference from placebo; odds ratio for ACR50 response = 17.61, 95% CI =3.86, 80.29) relative to patients who tested positive for anti-PAD4 (8.7% difference from placebo; odds ratio for ACR50 response = 1.46, 95% CI = 0.47, 4.56; P = 0.01 for treatment-biomarker interaction). Similar trends were observed for an association between greater clinical responses to mavrilimumab relative to placebo in anti-PAD4 negative patients in the earlier phase 2a trial CP219. Finally, this effect is specific to anti-PAD4 antibodies as no significant association between response and presence of antibodies to PAD3 was observed.

Conclusion:  Autoantibodies to PAD4 are a promising novel predictive biomarker for the targeted GM-CSF receptor inhibitor mavrilimumab, and further validation of this biomarker in future clinical studies is warranted.

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10:10
Morning Networking Break
Biomarkers & Surrogate Endpoints for Clinical Trials in Inflammatory Diseases
Moderator: Stephen Pennington, University College Dublin
 
Frédéric Baribaud
Associate Scientific Director
Janssen
About Speaker: Fred is an Associate Director in the Systems Pharmacology & Biomarkers department at Janssen R&D which he joined in 2006. He has been working in various inflammatory diseases working on patient stratification to enable individualized therapeu... Read Full Bio 
 
 
Frédéric Baribaud
Associate Scientific Director
Janssen
 
About Speaker:

Fred is an Associate Director in the Systems Pharmacology & Biomarkers department at Janssen R&D which he joined in 2006. He has been working in various inflammatory diseases working on patient stratification to enable individualized therapeutic treatments.
Fred holds a BS and a MS in Biochemistry from the University of Geneva, Geneva, Switzerland and a PhD in Biology from the University of Lausanne, Lausanne, Switzerland. Fred is a former UPENN postdoctoral fellow where he worked on HIV entry and a former employee of Incyte Inc, were he worked on target validation and was a discovery compound team co-lead for SMI development.

 
Abstract: IBD is a clinically heterogeneous condition with numerous interaction factors, including genetics, the microbiome, and...Read More 

IBD is a clinically heterogeneous condition with numerous interaction factors, including genetics, the microbiome, and the environment. Identifying the biological and phenotypic changes during the transition from health to disease can only be achieved through longitudinal monitoring of multiple molecular and phenotypic dimensions. The absence of scientific approaches for characterizing disease biology and disease severity itself hampers their application in IBD.

We propose using our extensive molecular data to define IBD molecular severity scores across multiple molecular dimensions, including transcriptomics, genetics, microbiome, and proteomics. Using normal controls, we will define a gradient of molecular disease severity from health to severe IBD and then classifying IBD patients into subpopulations that capture differences in disease phenotype and progression. This approach can then be used to contextualize the molecular changes observed in pre-disease cohorts and can be assessed for predictability of progression to IBD.

An IBD activity score (IBD_rActS) developed from colon tissue transcriptomics reveals stark molecular differences between overt IBD and clinical remission. This score also identifies clinical responders and non-responders before anti-TNF treatment with 100% specificity and 78.8% sensitivity validated in an independent dataset with 87.5% specificity and 85.7% sensitivity, respectively suggesting a robust relationship between the score and patient outcomes. Furthermore, analysis of serum proteomics data has allowed identifying candidate peripheral markers reflective of IBD_rActS.

By combining IBD_rActS with similar scores capturing molecular dysregulation in the gut microbiome, genetics, and peripheral proteomics and with technologies that enable dense longitudinal assessments, we will enable precise molecular monitoring of disease progression. We hypothesize that asymptomatic individuals at-risk for developing IBD will have already progressed down one or multiple molecular dimensions toward the molecular dysregulation observed in symptomatic disease. This approach could be used to identify patients for IBD interception trials and monitor the molecular efficacy of interception therapies.

Benefits:
– Elaboration of new molecular endpoints complementing to enable quicker decision making
– Granular and precise definition of disease severity and activity for patient stratification

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Thomas Kleen
Executive Vice President, Immune Monitoring
Epiontis
About Speaker: Dr. Kleen is currently Executive VP Immune Monitoring at Epiontis.  Prior he held the position of Director, Business and Technology Development and the function of Director, Assay Development and R&D at CTL, USA He received his Ph.D. in Biology ... Read Full Bio 
 
 
Thomas Kleen
Executive Vice President, Immune Monitoring
Epiontis
 
About Speaker:

Dr. Kleen is currently Executive VP Immune Monitoring at Epiontis.  Prior he held the position of Director, Business and Technology Development and the function of Director, Assay Development and R&D at CTL, USA He received his Ph.D. in Biology with specialty in Immunology and Virology from the Bayerische Julius-Maximilians-University Würzburg, Bavaria, Germany.  During his studies at Case Western Reserve University in Cleveland, Ohio, he investigated conditions of the human immune system affected by infectious diseases, and in particular the detailed monitoring of immune cell function in patients with human immune deficiency virus (HIV) infections.  He further conducted research on the facilitation of umbilical cord blood stem cell transplantation for immune reconstitution of adult patients with life-threatening hematologic al disorders and malignancies after treatment with full myelo-ablative regimens (http://www.linkedin.com/in/thomasokleen).

His expertise is in the areas of immunology, vaccine and biologic evaluation, immunogenicity and oncology.  One focus is monitoring of Cell Mediated Immunity (CMI) and Bio-markers during immunotherapy, vaccine development and detection of adverse immune reactions during drug development.  He spends significant time educating pharmaceutical entities and governmental agencies about validated, standardized, GLP compliant technologies, which can be deployed today during pre-clinical and clinical trials, for sample logistics and immune monitoring.
Since 2009 Dr. Kleen is member of the SITC-FDA Task Force on Immunotherapy Biomarkers, which focuses on the unique issues related to immunologic monitoring assays as well as novel methodologies for assessing the immune landscape in cancer.

 
Abstract: The responsiveness of the immune system is a prominent medical parameter with high relevance in oncology, autoimmunity...Read More 

The responsiveness of the immune system is a prominent medical parameter with high relevance in oncology, autoimmunity and infectious diseases. This responsiveness often described by cell counts and ratios of different leukocyte subpopulations. Current standard methods – flow cytometry in peripheral blood, immunohistochemistry (IHC) in solid tissue – have a limited ability for exact cell quantification owing to arbitrary/subjective gating protocols for FACS and lacking precision for IHC. In addition, stability of blood samples is a big issue especially in samples from clinical trials.

The discovery of cell type specific epigenetic markers allows precise and robust quantitation of immune cells in all human samples. The tests are based on quantitative PCR targeting genomic DNA. Therefore, readout is stable and samples can be frozen and easily shipped. This allows monitoring of patients in multicenter studies, retrospective studies, comparison of results between different studies, and routine monitoring.

Application of epigenetic assays for Treg, Th17, Tfh, overall T-cells, Granulocytes; Monocytes, NK, overall/naïve CD8, CD4 and B cells will be presented in cancer and immune disease patients. The results reveal the same trends as measurements with alternative methods, while showing higher precision. Furthermore, the tests can be applied on both blood and tissue allowing measurements of circulating and tissue-infiltrating immune cells. An addition technical benefit is the small sample amount requirement of epigenetic assays. Tissue amounts down to 1mg and blood volumes of 75µl are sufficient and permit processing of biopsy material and small blood samples from pediatric studies.

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Round Table Discussions

11:45
Round Table 1: Non-protein Biomarkers
11:45
Round Table 2: What Can We Learn from Oncology, Cardiovascular & Infectious Diseases Field That Can Help Develop Effective Neurological Biomarkers?
11:45
Round Table 3: Value of Longitudinal Testing
11:45
Round Table 4: Does Just the Appearance of Cell-Free DNA & Exosomes (RNA & Proteins) in your Blood Suggest Pathology is Present?
11:45
Round Table 5: Devices & Technologies for Biomarker Isolation & Analysis, Regardless of which Disease or Pathology
11:45
Round Table 6: Are Liquid Biopsies Ready for Primetime?
12:35
Lunch Provided by GTCbio
Microbiomes in Immune Modulation & Inflammatory Diseases
Moderator: Martin Schwickart , MedImmune
1:45
Autoimmunity and Chronic Fungal Infection in Carcinogenesis
 
Yinling Hu
Ph.D., Senior Investigator
National Cancer Institute
About Speaker: Yinling Hu is a senior investigation, head of Inflammation Tumorigenesis section, at the Cancer and Inflammation Program, Center for cancer Research, in the National Cancer Institute, NIH. Currently, Hu’s lab is study the role of IKKalpha in skin, ... Read Full Bio 
 
 
Yinling Hu
Ph.D., Senior Investigator
National Cancer Institute
 
About Speaker:

Yinling Hu is a senior investigation, head of Inflammation Tumorigenesis section, at the Cancer and Inflammation Program, Center for cancer Research, in the National Cancer Institute, NIH. Currently, Hu’s lab is study the role of IKKalpha in skin, lung and esophageal carcinogenesis. Dr. Hu obtained her Ph.D at the University of Melbourne in Australia. She was trained as a postdoc at Michael Karin’s lab of the UCSD in La Jolla California.

 
Abstract: The role of environmental fungi in human carcinogenesis is largely unknown. A causal relationship between T cell-mediated autoimmunity and fungal i...Read More 

The role of environmental fungi in human carcinogenesis is largely unknown. A causal relationship between T cell-mediated autoimmunity and fungal infection in carcinogenesis has not been appreciated. Human patients with autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), a T-cell-driven autoimmune disease caused by impaired central tolerance, are susceptible to developing chronic fungal infection and esophageal squamous cell carcinoma (ESCCs). We establish a new mouse model, kinase-dead Ikkalpha knockin mice, which recapitulates the disease of APECED patients and further demonstrate that the autoreactive CD4 T cells initiate fungal infection; autoinflammation sustains chronic fungal infection; and the autoreactive CD4 T cells and chronic fungal infection drive esophageal carcinogenesis. These findings highlight that the importance of environmental fungi and impaired IKKalpha in human esophageal carcinogenesis.

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2:10
Tools and Techniques for Biomarker Discovery with the Microbiome
 
Justine Debelius
Postdoctoral Scholar
University of California, San Diego
About Speaker: Justine Debelius is a postdoctoral researcher in the Knight Lab at the University of California, San Diego. Her work focuses on the role of the gut microbiome in the development and management of chronic diseases, especially autoimmune diseases. Dr D... Read Full Bio 
 
 
Justine Debelius
Postdoctoral Scholar
University of California, San Diego
 
About Speaker:

Justine Debelius is a postdoctoral researcher in the Knight Lab at the University of California, San Diego. Her work focuses on the role of the gut microbiome in the development and management of chronic diseases, especially autoimmune diseases. Dr Debelius received her PhD in 2015 from the University of Colorado, Boulder, where her dissertation focused on the influence of lifestyle factors on the gut microbiome.

 
Abstract: The microbiome contains more than 100 times more genes than the human genome. Despite numerous studies demonstrating a relationship between the mic...Read More 

The microbiome contains more than 100 times more genes than the human genome. Despite numerous studies demonstrating a relationship between the microbiome and disease, the use of the microbiome as a biomarker for disease remains challenging. Some of the problems stem from the multiple factors which influence microbial communities. Understanding factors which effects observed microbial communities and how to control for these confounders will help simplify study comparisons. The use of open source techniques and tools can help minimize technical variation. Analyses which account for the structure of microbiome data can also be used elucidate new features. Multi-omic integration will help best identify the set of features that serve as markers for disease state. While it may be early to use the microbiome as a marker for complex disease, the potential for the near future is strong.

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Inflammatory Biomarkers in Respiratory Diseases
Moderator: Bruce McManus, The Centre of Excellence for Prevention of Organ Failure
2:35
Type 2 Biomarkers Enrich for Seasonal, IL-13 Dependent Asthma Exacerbations
 
David Choy
Principal Scientific Researcher
Genentech
About Speaker: David Choy is a principal scientific researcher in the department of Biomarker Discovery OMNI at Genentech where he is responsible for biomarker discovery and research strategy for asthma. With a passion for computational, human genetics, and transla... Read Full Bio 
 
 
David Choy
Principal Scientific Researcher
Genentech
 
About Speaker:

David Choy is a principal scientific researcher in the department of Biomarker Discovery OMNI at Genentech where he is responsible for biomarker discovery and research strategy for asthma. With a passion for computational, human genetics, and translational biology approaches, his work has contributed to the discovery of periostin, a biomarker of Type 2 asthma.

 
Abstract: Epidemiologic studies have implicated aeroallergens and respiratory infections as triggers underlying seasonal increas...Read More 

Epidemiologic studies have implicated aeroallergens and respiratory infections as triggers underlying seasonal increases in asthma exacerbations in spring and autumn months. These seasonal factors may trigger or amplify airway inflammation in atopic, Type 2 high asthma patients that precipitates acute worsening of symptoms. Biologic therapies targeting Type 2 cytokine pathways have demonstrated efficacy in reducing the rate of severe asthma exacerbations, particularly in patients selected on the basis of Type 2 biomarkers. In children with asthma, increased inhaled corticosteroid or anti-IgE therapy has been found to reduce the rate of seasonal exacerbations. We hypothesized that lebrikizumab (anti-IL-13) therapy would likewise be effective in reducing seasonal exacerbations in adults with asthma.

We conclude that seasonal spikes in exacerbations may be primarily dependent on Type 2 inflammatory processes. The molecular pathways underlying asthma exacerbations are heterogeneous and therapeutic strategies targeting Type 2 biology alone may have the greatest efficacy in limiting seasonal spikes in exacerbation rates. Increased therapeutic efficacy may require targeting multiple distinct pathways in asthma.

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3:00
Afternoon Networking Break
3:45
IgE Heterogeneity in Allergy
 
Toshiaki Kawakami
Professor, Division of Cellular Biology
La Jolla Institute for Immunology & Allergy
About Speaker: 1978 MD, 1983 PhD from The University of Tokyo 1984-88 Postdoc at the NCI/NIH 1990 Assistant Professor, La Jolla Institute for Allergy and Immunology (LJI) 2000 Professor, LJI... Read Full Bio 
 
 
Toshiaki Kawakami
Professor, Division of Cellular Biology
La Jolla Institute for Immunology & Allergy
 
About Speaker:

1978 MD, 1983 PhD from The University of Tokyo
1984-88 Postdoc at the NCI/NIH
1990 Assistant Professor, La Jolla Institute for Allergy and Immunology (LJI)
2000 Professor, LJI

 
Abstract: ...Read More 

IgE molecules have a tremendous heterogeneity in their ability to influence mast cell biology. We have recently extended this line of study by showing that histamine-releasing factor (HRF) directly binds a subset of immunoglobulins (Igs) including IgE via interactions of two Ig-interacting sites within HRF with the Fab portion of Igs. As HRF can be present as a disulphide-linked dimer, HRF can activate HRF-reactive IgE-sensitized mast cells.
In an IgE/FcεRI/mast cell-dependent food allergy model, diarrhea and type 2 intestinal inflammation were induced by repeated ovalbumin (OVA) gavages in OVA-immunized BALB/c mice, accompanied by increased mast cell numbers and HRF dimer levels. Confocal microscopy revealed that orally administered HRF inhibitors that block HRF-IgE interactions preferentially target mast cells in the jejunum. Both prophylactic and therapeutic administration of HRF inhibitors reduced diarrhea occurrence, intestinal inflammation, and reduced mast cell activation. Relevance of the mouse data was studied by measuring serum levels of HRF and HRF-reactive IgE and IgG before and after oral immunotherapy (OIT) of human patients allergic to hen eggs. Egg allergy patients had higher plasma levels of HRF-reactive IgE and IgG than healthy controls. Interestingly, the patients who exhibited high sensitivity to allergen within 2 weeks of allergen avoidance after 12 months of maintenance dosing, unlike those who exhibited low sensitivity, had higher HRF-reactive IgE levels than their levels one week after the OIT initiation. Therefore, HRF promotes diarrhea development and intestinal Type 2 inflammation in food allergic mice and blood levels of HRF-reactive IgE might serve as a biomarker for a long-term success of OIT of human food allergy.

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Pierluigi Mauri
Chief of Proteomics & Metabolomics
Proteomics and Metabolomics Laboratory - CNR, Institute for Biomedical Tecnologies (ITB-CNR)
About Speaker: Pierluigi Mauri is chief of Proteomics and Metabolomics Labat ITB-CNR. He has a notable experience in the development and application of several technologies (HPLC, capillary electrophoresis, mass spectrometry) for the identification and quantificati... Read Full Bio 
 
 
Pierluigi Mauri
Chief of Proteomics & Metabolomics
Proteomics and Metabolomics Laboratory - CNR, Institute for Biomedical Tecnologies (ITB-CNR)
 
About Speaker:

Pierluigi Mauri is chief of Proteomics and Metabolomics Labat ITB-CNR. He has a notable experience in the development and application of several technologies (HPLC, capillary electrophoresis, mass spectrometry) for the identification and quantification of several biomolecules in complex matrices. He has been involved in the study of the bioavailabily of several drugs in humans and animals.
He has set-up a proteomics laboratory based on the MudPIT (Multidimensional Protein Identification Technology), a gel-free approch (using two dimensional chromatography coupled to tandem mass spectrometry, 2DC-MS/MS) and a parallel computing system for proteomics. This instrumentation allows for the proteomic profiling of biological samples, without limitations, according to to protein molecular weight, pI or hydrophobicity and guarantees high productivity. Using this methodology, secreted proteins from tumor (FASEB 2005) and immune system cells (JEM, 2011, Immunity, 2016) have been characterized.
Furthermore, direct analysis of biological samples (JMS 2007), enzymatic complexes (Biochimie 2006), development of novel methodological approaches for studying switchredox proteins (JBC 2005 and 2006, JMB 2006), degenerative diseases (Blood, 2012) and structural and functional characterization of proteins (Biol. Chem. 2004, FEBS 2006) have been performed. His main investigations concern molecular profiles obtained by MS-based methodologies and focused on clinical applications (J Proteome Research,
2014; JACC 2013; Nature Commun, 2014; Cardiovasc Res. 2011; Biol Reprod. 2013), including computational methods (J Clin Bioinforma. 2013; Methods Enzymology, 2008) He is co-author of more than 150 scientific publications, he has participated in numerous international congresses and has taught in several metabolomics and proteomics courses.

 
Abstract: Asthma is a chronic inflammatory disease that affects over 300 million patients (Bel EH, N Engl J Med 2013); it is a heterogeneous disease process ...Read More 

Asthma is a chronic inflammatory disease that affects over 300 million patients (Bel EH, N Engl J Med 2013); it is a heterogeneous disease process characterized by chronic inflammation, variable airflow obstruction and airways hyperresponsiveness.
Recently, the US Severe Asthma Research Program (SARP) analyzed an extensive dataset of patients with severe and non-severe asthma, in order to identify and describe robust subgroups of asthma patients with distinct features which possibly could be, but as yet has not been shown to be, an informative guide to personalized therapy.
Recent molecular and genetic studies have identified clinical and inflammatory phenotypes that associate with specific biomarkers for T-Helper type-2 lymphocytes (Th2) driven-inflammation, including Nitric Oxide (FeNO) levels, eosinophilia and periostin levels. Treatment of “Th2-high” asthmatic patients with biologic agents targeting Immunoglobulin E (IgE) and the canonical Th2-related cytokines are emerging as efficacious asthma therapies (Gandhi NA et al., Nat Rev Drug Discov. 2016).
Although the targeted biologic agents are efficacious in treating some phenotypes of asthma and allergic disease, some patients might respond better to one biologic agent than another or not at all; the reasons for these differential responses are not known.
Recently, two different phenotypes of severe asthma were identified (Riccio et al., Int J Immunopathol Pharmacol. 2012) on the basis of histological evaluation of airways remodeling after 12 months of anti-IgE treatment. In addition, according to a omics strategy described by Holgate (Trends Immunol 2012) we applied proteomic analysis of paraffined biopsies from baseline (t0) and after 12 and 36 months of anti-IGE treatment. In particular, it was applied Multidimensional Protein Technology Identification (MudPIT), combined with label-free quantitative analysis (Mauri & Scigelova, Clin Chem Med Lab, 2009) and systems biology. In this way it was possible to perform a “Clinical Proteomics” approach (Mauri et al. Imm. Lett. 2014), combining clinical and molecular data and obtaining a good agrement between clinical and proteomic stratification. In addition to identified biomarkers (such as galectin-3), another benefit of Clinical Proteomics approach involves the investigation of molecular mechanisms correlated to disease and anti-IgE treatment. Of note, galectin-3 and its interactors have been identified in lymphocytes, RBC and other circulating cells.

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4:35
Discovery of Non-Invasive Biomarkers of IL-17 Activity from Experimental Models of Asthma
 
Tracy Staton
Scientist, OMNI Biomarker Development
Genentech
About Speaker: Tracy completed her graduate studies in the Immunology Program at Stanford University School of Medicine, where she worked in the laboratory of Dr. Eugene Butcher to characterize molecular mechanisms of lymphocyte homing. She then did her postdoctora... Read Full Bio 
 
 
Tracy Staton
Scientist, OMNI Biomarker Development
Genentech
 
About Speaker:

Tracy completed her graduate studies in the Immunology Program at Stanford University School of Medicine, where she worked in the laboratory of Dr. Eugene Butcher to characterize molecular mechanisms of lymphocyte homing. She then did her postdoctoral training at Harvard University School of Public Health in the laboratory of Dr. Laurie Glimcher. During that time, she studied immune cell development and the importance of effector lymphocyte populations in preclinical models of disease. Tracy came to Genentech in 2011 and holds the position of Scientist in the Biomarker Development group. Her interests are in understanding the mechanisms of action of novel therapeutics in human diseases, and using translational pharmacology to improve the effectiveness of clinical development. At Genentech, she has been involved in developing diagnostic and pharmacodynamic biomarker strategies and assays for programs in asthma.

 
Abstract: Asthma is a heterogeneous disorder regulated by distinct molecular mechanisms and there are increasing efforts to deve...Read More 

Asthma is a heterogeneous disorder regulated by distinct molecular mechanisms and there are increasing efforts to develop therapeutics to target these pathways. The utilization of biomarkers early in clinical development can facilitate efficient and effective drug development. Although Type 2 inflammation contributes significantly to disease pathophysiology, there is evidence for the role of other pathways including IL-17. IL-17 elicits the expression of factors involved in neutrophil maturation and migration and may contribute to corticosteroid resistant neutrophilic airway inflammation. We set out to identify IL-17 induced genes that could serve as non-invasive biomarkers of IL-17 pathway activity in asthma and may have utility in predicting clinical response and determining pharmacodynamic effects of IL-17 pathway targeted therapies. Using data from in vitro stimulation cultures, we have identified a panel of IL-17 pathway biomarkers and were able to show that at least two of these candidates (G-CSF, CXCL1) were modulated in the serum by IL-17 blockade in the House Dust Mite mouse model of asthma. Given these biomarkers are detectable in serum of asthmatics, they have potential as clinical predictive or pharmacodynamic biomarkers to monitor IL-17 activity and pathway modulation in the clinic.

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5:00
Day 2 Concludes
Day - 3 Wednesday, March 22nd, 2017
7:30
Breakfast
Overcoming Commercialization Challenges in Biomarker Development
Moderator: Bruce Jordan , Roche Diagnostics
8:30
Personalised Healthcare Solutions in Oncology and Beyond - Where Next?
 
Bruce W.M. Jordan
Vice President, International Business Leader, Personalised Healthcare Solutions (PHCS), Centralised and Point of Care Solutions
Roche Diagnostics
About Speaker: Dr. Bruce W.M. Jordan, Ph.D, FIBMS VP, International Business Leader – Personalised Healthcare Solutions Bruce joined the Roche Pharmaceuticals Herceptin team in Basel in 2005, leading the global HER2 Testing/Companion Diagnostics (CDx) acti... Read Full Bio 
 
 
Bruce W.M. Jordan
Vice President, International Business Leader, Personalised Healthcare Solutions (PHCS), Centralised and Point of Care Solutions
Roche Diagnostics
 
About Speaker:

Dr. Bruce W.M. Jordan, Ph.D, FIBMS
VP, International Business Leader – Personalised Healthcare Solutions

Bruce joined the Roche Pharmaceuticals Herceptin team in Basel in 2005, leading the global HER2 Testing/Companion Diagnostics (CDx) activities. Since January 2013 he is VP - International Business Leader – Personalised Healthcare Solutions at Roche Diagnostics – Centralised and Pont of Care Solutions at their HQ in Rotkreuz, Switzerland.

He has worked on CDx development & commercialization out of both the Pharmaceuticals and Diagnostics Divisions over the past 11 years at Roche, across therapeutic areas such as Neurology, Immunology, Fertility and Oncology.

Bruce trained in Laboratory Medicine, graduating from the University of Portsmouth U.K. in 1994 with a BSc. (Hons.) Biomedical Sciences, subsequently gaining State Registration in Haematology and Transfusion Medicine at Southampton General Hospital. He has also worked at the Royal Marsden Hospital, London, and has a Ph.D. in cancer research from the University of Würzburg in Germany.

Bruce has also represented the diagnostics industry as a Corporate Representative at the International Federation of Clinical Chemistry and Laboratory Medicine (IFCC), and is a Fellow of the Institute of Biomedical Sciences (FIBMS) in the U.K., and a member of the European Society of Pharmacogenomics and Personalised Therapy (ESPT)

 
Abstract: Medical need for a more patient-tailored approach has resulted in a greater number of targeted therapies focused on be...Read More 

Medical need for a more patient-tailored approach has resulted in a greater number of targeted therapies focused on better defined patient groups. Within the past few years, Roche has embarked on a systematic approach for the development of medicines, interweaving diagnostic and pharmaceutical expertise to pave the way for Personalised Healthcare (PHC).

We have already begun to provide healthcare professionals with more powerful diagnostic tools and targeted treatments that are based upon new insights into how disease arises at the molecular level. Up until now, this has happened mostly in the fields of oncology, and new developments will address high areas of unmet medical need such as Fertility and Alzheimer’s Disease.

It is clear that the growing number of game-changing targeted therapies in development will require ever more innovative diagnostic tools to guide their use. Their limited number of PHC approaches suggests there are still many challenges, as well as opportunities in this emerging field of personalized medicine.This presentation will highlight approaches in oncology and beyond.

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8:55
Regulatory Trends and Insights in Biomarker Development and Clinical Translation
 
Melina Cimler
Senior Vice President, Quality & Regulatory
Adaptive Biotech
About Speaker: Melina Cimler is Senior Vice President of Regulatory & Quality at Adaptive Biotechnologies. She has over 27 years of experience in the life science and FDA-regulated diagnostic industry leading regulatory, quality systems, clinical affairs, resea... Read Full Bio 
 
 
Melina Cimler
Senior Vice President, Quality & Regulatory
Adaptive Biotech
 
About Speaker:

Melina Cimler is Senior Vice President of Regulatory & Quality at Adaptive Biotechnologies. She has over 27 years of experience in the life science and FDA-regulated diagnostic industry leading regulatory, quality systems, clinical affairs, research, and product development organizations.
Prior to joining Adaptive, Dr. Cimler served as Head of Global Quality and Vice President of Quality, Regulatory, Clinical and Government Affairs at Illumina Inc. She defined and executed on the regulatory strategy resulting in the first next generation sequencing platform (Illumina’s MiSeqDx) receiving FDA marketing authorization.
Previously, she held leadership positions in quality, regulatory and government affairs as Senior Vice President at Beckman Coulter Inc. She also served in senior quality, clinical and regulatory roles at Abbott Molecular, Gen-Probe Inc., and C.R. Bard, and as head of Product Development at Epitope, Inc. (now OraSure Technologies). Dr. Cimler holds a Ph.D. in Pharmacology from the University of Washington.

 
Abstract: Regulatory trends and insights in biomarker development and clinical translation This presentation will examine the changing regulatory path...Read More 

Regulatory trends and insights in biomarker development and clinical translation

This presentation will examine the changing regulatory pathways for taking novel biomakers from development to clinical use. Pharma and diagnostic companies have to partner closely to deliver on the promise of precision medicine in an ever changing and complex global regulatory framework.
Learn what is required to obtain successful regulatory approval for these niche products.

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9:20
Guidance on How to Transform Biomarkers into Clinically Relevant Information
 
Katherine Tynan
President
Tynan Consulting
About Speaker: Dr. Tynan is a seasoned biotechnology entrepreneur with a focus on business development, startup entrepreneurship, fundraising and strategic business planning for clinical diagnostic companies and investors. She has a background in product developmen... Read Full Bio 
 
 
Katherine Tynan
President
Tynan Consulting
 
About Speaker:

Dr. Tynan is a seasoned biotechnology entrepreneur with a focus on business development, startup entrepreneurship, fundraising and strategic business planning for clinical diagnostic companies and investors. She has a background in product development, financial analysis, operations, regulatory, evidence development, reimbursement and market access for clinical diagnostics in the US and EU. Currently Katherine works with a number of early stage and established diagnostic companies guiding them through product development choices, market entry strategies, funding across a diversified set of indications and technologies. http://www.tynandx.com/

 
Abstract: Healthcare in the US is in the middle of a transformative change. Reform initiatives are focused on improving and quan...Read More 

Healthcare in the US is in the middle of a transformative change. Reform initiatives are focused on improving and quantifying patient outcomes, tying provider reimbursement to quality metrics rather than traditional fee for service payment. High deductible plans and increasing cost transparency are forcing consumers to shop around for cost effective care. Meanwhile the science of medicine and medical technology is exploding. Bridging the gap between the discovery of biomarkers and turning them into clinically relevant diagnostics that can provide patient/health system benefit and can be successfully commercialized, will be the subject of this presentation.

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9:45
The Evolving CDx Deal Landscape: Are Progressive Deal Structures the Way of the Future?
 
Gary Gustavsen
Vice President
Health Advances
About Speaker: Gary Gustavsen joined Health Advances in 2005 and leads its Personalized Medicine Practice. A noted writer and workshop leader in the field of companion diagnostics and personalized medicine, his work focuses on commercialization strategy, indication... Read Full Bio 
 
 
Gary Gustavsen
Vice President
Health Advances
 
About Speaker:

Gary Gustavsen joined Health Advances in 2005 and leads its Personalized Medicine Practice. A noted writer and workshop leader in the field of companion diagnostics and personalized medicine, his work focuses on commercialization strategy, indication prioritization, pricing and reimbursement strategy, system economics, and business development opportunities for both diagnostic and therapeutic clients.
Prior to joining Health Advances, Gary was a researcher at Brookhaven National Lab evaluating a proprietary line of synthetic growth factors. Gary also worked in the Cell & Tissue Technologies group at Becton Dickinson, the Exploratory Cancer Research group at OSI Pharmaceuticals, and most recently the Corporate Strategy group at Millennium Pharmaceuticals. Gary received his Bachelors degree in Biomedical Engineering from Duke University and his Masters degree in Biomedical Engineering from Stony Brook University.

 

 

 
Abstract: Historically, companies developing CDx have been regarded as vendors by therapeutics companies and incentives between the two partners have been mi...Read More 

Historically, companies developing CDx have been regarded as vendors by therapeutics companies and incentives between the two partners have been misaligned. However, multiple factors are impacting the CDx deal landscape pointing towards the need for a true partnership between the diagnostics and therapeutics companies. Through discussion of the factors impacting the deal landscape, as well as case studies, the potential for more progressive deal structures will be discussed.

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10:10
Morning Networking Break
Panel Discussion: Companion Diagnostics Development & Partnering Strategies
Moderator: Gary Gustavsen, Health Advances, LLC
10:40
Melissa Reuter
Associate Director, Business Development, Partnering for Precision Diagnostics
Qiagen
About Speaker: Melissa Reuter MS, MBA is Associate Director, Partnering for Precision Diagnostics, Molecular Diagnostics at QIAGEN, where her responsibilities involve establishing and managing partnerships with Pharma clients for the development of Companion Diagno... Read Full Bio 
 
 
Melissa Reuter
Associate Director, Business Development, Partnering for Precision Diagnostics
Qiagen
 
About Speaker:

Melissa Reuter MS, MBA is Associate Director, Partnering for Precision Diagnostics, Molecular Diagnostics at QIAGEN, where her responsibilities involve establishing and managing partnerships with Pharma clients for the development of Companion Diagnostic assays for approval with their drugs.

Melissa started her career as a Histology Technician at the Johns Hopkins Medical Institution. She then moved into the Life Science industry in 1999 where she worked her way through roles of increasing responsibility in Sales and Account Management until she was National Account Manager for Government and Public Health at QIAGEN in 2013. At this point, Melissa transitioned into a Business Development role with Thermo Fisher’s OEM and Out-licensing team. During this time, she was a key contributor to the incorporation of tools, technologies, and intellectual property into assays of commercial diagnostic developers. Her last role, before returning to QIAGEN, was at Personal Genome Diagnostics (PGDx), where she was Marketing Director. While at PGDx, she assisted in the development of strategies and implementation of promotional tactics to maximize sales, market share, and profitability for PGDx’s in-house tissue and liquid biopsy services and distributed testing model.

Melissa holds a BSc in Biology from the Pennsylvania State University, a MS in Biotechnology from Johns Hopkins University, and a MBA also from Johns Hopkins University.

10:40
Catherine Brownstein
Scientific Director, Manton Center for Orphan Disease Research
Boston Children's Hospital
About Speaker: Dr. Brownstein is a research associate in Genetics and Genomics, Instructor in Pediatrics, and the manager of the Molecular Genetics Core Facility at Boston Children’s Hospital. Recently named the Scientific Director for the Manton Center for Orpha... Read Full Bio 
 
 
Catherine Brownstein
Scientific Director, Manton Center for Orphan Disease Research
Boston Children's Hospital
 
About Speaker:

Dr. Brownstein is a research associate in Genetics and Genomics, Instructor in Pediatrics, and the manager of the Molecular Genetics Core Facility at Boston Children’s Hospital. Recently named the Scientific Director for the Manton Center for Orphan Disease Research and specializing in gene discovery, Dr. Brownstein has been instrumental in the elucidation of several new disease genes for conditions such as intellectual disability, nemaline myopathy, psychosis, SIDS, and hypophosphatemic rickets. Her current work focuses on advancing the fields of next generation sequencing and analysis, as evidenced in her management of the international CLARITY and CLARITY Undiagnosed competitions.

10:40
Zach Hornby
Chief Operating Officer
Ignyta
About Speaker: Zachary Hornby brings a dozen years of life sciences business experience to Ignyta. Prior to joining Ignyta, Mr. Hornby was senior director of business development at Fate Therapeutics, where he was responsible for pharmaceutical partnerships and non... Read Full Bio 
 
 
Zach Hornby
Chief Operating Officer
Ignyta
 
About Speaker:

Zachary Hornby brings a dozen years of life sciences business experience to Ignyta. Prior to joining Ignyta, Mr. Hornby was senior director of business development at Fate Therapeutics, where he was responsible for pharmaceutical partnerships and non-dilutive funding. Mr. Hornby has also served in various business and commercial roles at Halozyme Therapeutics (NASDAQ: HALO), Neurocrine Biosciences (NASDAQ: NBIX), and TKT (now Shire Human Genetic Therapies), and he was a life sciences consultant at L.E.K. Consulting. Mr. Hornby is a director of Independa, Inc.

Mr. Hornby holds B.S. and M.S. degrees in biology from Stanford University and an M.B.A. from Harvard Business School.

10:40
Alex Parker
Vice President, Biopharma
Foundation Medicine
About Speaker: Dr. Parker has served as the Vice President of Foundation Medicine’s Business Development team since 2014, where he partners with biopharmaceutical companies to bring transformative cancer care to patients. Alex began his journey at Foundation Medi... Read Full Bio 
 
 
Alex Parker
Vice President, Biopharma
Foundation Medicine
 
About Speaker:

Dr. Parker has served as the Vice President of Foundation Medicine’s Business Development team since 2014, where he partners with biopharmaceutical companies to bring transformative cancer care to patients. Alex began his journey at Foundation Medicine in 2010 as the company’s Vice President of Process Technology. Prior to joining Foundation Medicine, Dr. Parker was Principal Scientist at Amgen and held various roles at Millennium Pharmaceuticals. He received his Ph.D in Molecular Genetics from the University of Maine and a bachelor’s in Philosophy and Biology from the University of New Mexico.              

10:40
Felix Frueh
Executive Partner
Opus Three
About Speaker: Dr. Felix Frueh is a respected thought leader in personalized medicine with twentyfive years of R&D, management, and policy experience. He founded Opus Three in 2012 after spending several years at the FDA and in the payer environment working... Read Full Bio 
 
 
Felix Frueh
Executive Partner
Opus Three
 
About Speaker:

Dr. Felix Frueh is a respected thought leader in personalized medicine with twentyfive years of R&D,
management, and policy experience. He founded Opus Three in 2012 after spending several years at the FDA
and in the payer environment working closely with the pharmaceutical and diagnostic industry.
Dr. Frueh is also Co-Founder and CEO of Intellos Health, a new diagnostic business focused on the development
of affordable tests for the identification of patients requiring confirmatory testing to receive appropriate
therapies.
Prior, he was Chief Scientific Officer at Human Longevity, Inc. and Entrepreneur-in-Residence at Third Rock
Ventures. Dr. Frueh was President of the Medco Research Institute, leading the PBM’s research initiatives in
health economics and outcomes research after having formed Medco’s personalized medicine R&D
organization. Prior to Medco, he was the first Associate Director for Genomics at the FDA, where he oversaw
the development and implementation of policies around biomarker qualification and companion diagnostics.
Dr. Frueh is a Board member at Enterome Biosciences, Ariana Pharmaceuticals, Intellos Health, and
CredibleMeds, and also served on the Board of the Personalized Medicine Coalition. He is a Fellow of the
American College of Clinical Pharmacology and was a faculty member at Georgetown University in
Washington, DC and at the University of North Carolina. Dr. Frueh received his education at Stanford
University and the University of Basel in Switzerland, where he graduated magna cum laude in biochemistry.

11:30
Lunch Provided by GTCbio
12:45
Conference Concludes