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Inflammatory & Immunological Biomarkers

2017-02-212017-02-212017-01-26
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2017 Agenda
Day 1
 
Day 1 - Monday, March 20th, 2017
7:00
Continental Breakfast & Registration
8:00
Opening Remarks
Overcoming Challenges of Clinical Validation & Translation

9:00
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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9:25
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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9:50
Morning Networking Break
10:30
Donor-Derived Cell-Free DNA: An Accurate, Precise, and Dynamic Biomarker for Improved Management of Solid Organ Transplant Patients
 
Marica Grskovic
Associate Director, R&D
CareDx
About Speaker: ... Read Full Bio 
 
 
Marica Grskovic
Associate Director, R&D
CareDx
 
About Speaker:
 
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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10:55
Translating Protein MS Assays into the Clinical Laboratory
 
Dobrin Nedelkov
Research 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
Research 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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11:20
Effects of RG7652, a Monoclonal Antibody Against PCSK9, on LDL-C, LDL-C Subfractions, and Inflammatory Biomarkers in Patients at High Risk of or with Established Coronary Heart Disease: Results of the Randomized, Placebo-Controlled, Phase 2 EQUATOR Study
 
Amos Baruch
Scientist, Development Sciences
Genentech
About Speaker: Dr. Amos Baruch has over 15 years of combined biomedical experience in Translational Biology, Biochemistry, and Pharmacology. He joined Genentech at the turn of 2011 as a Scientist in the Pharmacodynamic Biomarker (PDB) group.  In his role as a PDB ... Read Full Bio 
 
 
Amos Baruch
Scientist, Development Sciences
Genentech
 
About Speaker:

Dr. Amos Baruch has over 15 years of combined biomedical experience in Translational Biology, Biochemistry, and Pharmacology. He joined Genentech at the turn of 2011 as a Scientist in the Pharmacodynamic Biomarker (PDB) group.  In his role as a PDB Scientist he develops and executes biomarker strategies for programs in several disease areas including cardiovascular, metabolism, anti-microbial, ophthalmology and wound healing.  Dr. Baruch received his Ph.D. from Tel-Aviv University in 1998. He then relocated to the US to pursue postdoctoral studies at the Scripps Research Institute with Dr. Bernard Gilula and thereafter in the department of Biochemistry and Biophysics at UCSF with Dr. Matthew Bogyo.  Dr. Baruch then Joined Celera Genomics and lead the chemical proteomics group charged with developing small molecule activity based probes to provide pharmacodynamic support for small molecule programs in the areas of Cancer, Asthma and Autoimmune diseases. In 2006, he joined KAI Pharmaceuticals and championed the preclinical research and development of therapeutic peptides for hyperparathyroidism in renal dialysis patients. The lead candidate from that effort was recently approved.   

 
Abstract: RG7652 is a fully humanized immunoglobulin G1 monoclonal antibody against PCSK9. EQUATOR, a proof-of concept, randomiz...Read More 

RG7652 is a fully humanized immunoglobulin G1 monoclonal antibody against PCSK9. EQUATOR, a proof-of concept, randomized, double-blind, dose-ranging phase 2 study, which evaluated RG7652 when added to standard-of-care therapy in patients at high risk of or with coronary heart disease (CHD). The primary endpoint was change in LDL-C from baseline to day 169. Patients with baseline LDL-C levels of 90–250 mg/dL while on standard-of-care therapy were randomized to receive one of five RG7652 dose regimens or placebo, administered subcutaneously every 4, 8, or 12 weeks for 24 weeks. Significant dose-dependent mean reductions in LDL-C levels from baseline to nadir were observed with all RG7652 regimens; effects persisted to day 169 with the highest doses with no unexpected safety signals. RG7652 also reduced apolipoprotein B and lipoprotein(a) levels. LDL-C sub-fraction analysis by NMR revealed a prominent decrease in large LDL-C and, to a lesser extent, in small LDL-particles. Consistently, there was a significant reduction in mean particle size of LDL-C with RG7652 as determined on day 169. Despite pronounced reduction in LDL-C, there were no significant changes in systemic markers of inflammation including hsCRP, IL-6, and TNFα, following a 24-week treatment with RG7652.

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11:45
Challenges and Experiences in Ovarian Cancer Early Detection Trials Using Longitudinal Algorithms
 
Steven Skates
Associate Professor, Medicine
Harvard Medical School
About Speaker: ... Read Full Bio 
 
 
Steven Skates
Associate Professor, Medicine
Harvard Medical School
 
About Speaker:
12:10
Lunch Provided by GTCbio
Emerging Technologies in Biomarker Development
Sandip Patel, University of California San Diego
1:25
Adding the Phenotypic Dimension: Big Data from Flow Cytometry. Structure, Strategies, Risks, and Promises
 
Anka Ehrhardt
Director Clinical Cytometry
Bristol-Myers Squibb Co.
About Speaker: Dr. Anka Ehrhardt is biophysicist and holds a doctorate in physiology. She is currently working in the United States for Bristol-Myers Squibb. In her current position at BMS, Dr. Ehrhardt directs the fast growing Clinical Cytometry operation, cover... Read Full Bio 
 
 
Anka Ehrhardt
Director Clinical Cytometry
Bristol-Myers Squibb Co.
 
About Speaker:

Dr. Anka Ehrhardt is biophysicist and holds a doctorate in physiology. She is currently working in the United States for Bristol-Myers Squibb.
In her current position at BMS, Dr. Ehrhardt directs the fast growing Clinical Cytometry operation, covering a large portfolio of immuno-oncology, and other disease area clinical studies. Her work focuses on strategies for biomarkers to drive the development of urgently needed new medicines. In this fast-paced environment, Dr. Ehrhardt drives targeted innovation to achieve sustained speed and quality.
Dr. Ehrhardt is currently on the board of different industry organizations, fostering innovation and collaboration between industry, academia and government organizations.

 
Abstract: ‘Classic’ –omics data focus on a census of available ‘building blocs’ Rigorous immunophenotyping will be the first phenoty...Read More 
  • ‘Classic’ –omics data focus on a census of available ‘building blocs’
  • Rigorous immunophenotyping will be the first phenotypic data –omics
  • Technical and technological innovations allowing an immunophenotyping database build will be explained
  • A data analysis strategy taking into account the phenotypic nature of immunophenotyping will be discussed

 

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1:50
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, Inc
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, Inc
 
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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2:15
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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2:40
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.

3:05
Afternoon Networking Break
Big Data Analytics & Bioinformatics for Biomarkers Development

3:35
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: ... 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:
 
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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4:00
Computational Biology Considerations for Translational Biomarker Research
 
Bin Li
Associate Director, Computational Biology
Takeda
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
Associate Director, Computational Biology
Takeda
 
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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4:25
Leveraging Panomic Data for the Development and Validation of Novel Biomarkers for Common, Chronic Diseases
 
Szilard Voros
Founder & Chief Executive Officer
Global Genomics Group
About Speaker: ... Read Full Bio 
 
 
Szilard Voros
Founder & Chief Executive Officer
Global Genomics Group
 
About Speaker:
4:50
ExRNAs as BioMarkers in Placental Function
 
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

 
Abstract: Placental ExRNAs represent a potentially rich source of biomarkers that can enable a clinician to monitor and better u...Read More 

Placental ExRNAs represent a potentially rich source of biomarkers that can enable a clinician to monitor and better understand the developmental status and health of a fetus during pregnancy. Placental dysfunction, commonly referred to as preeclampsia, can result in significant maternal and fetal morbidity/mortality and is likely to be caused by abnormal trophoblast differentiation, invasion, and/or function. Symptoms usually manifests in the second half of pregnancy, but has its origin in events that occur in the first trimester. Properly identifying early stage high risk candidates will permit more intensive patient surveillance with limited resources, as well as provide appropriate populations for novel therapeutic discovery efforts. Current diagnostic tests have low sensitivity, low specificity, are not widely available, and are difficult to implement. Small RNA NGS profiling of normal placental material throughout pregnancy provides a baseline from which to identify preeclamptic ExRNA biomarkers, either through presence/absence, or through alternative temporal expression patterns. Numerous challenges exist in ExRNA biomarker discovery ranging from NGS library bias, to data normalization, to background genetic heterogeneity, and to sample preparation. Robust methods to address these and other issues, must be developed such that signal is not obscured by the noise.

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5:15
Networking Reception & Poster Session
Day - 2 Tuesday, March 21st, 2017
7:30
Continental Breakfast & Registration
Biomarkers of Early Progressive Inflammatory & Immunological Diseases

8:30
Detection of Autoimmune Responses to Exosomes and Candidate Autoantigens in Pre-Diabetic NOD Mice
 
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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8:55
Discovery, Development and Delivery of Multiplexed Protein Biomarker Tests to Support Clinical Decision Making
 
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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9:20
Autodigestion: A Source of Cell Dysfunction and Multiorgan Failure
 
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. 

 

 

 

 

 
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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9:45
Autoantibodies as Predictive Biomarkers in Rheumatoid Arthiritis: anti-PAD2, anti-PAD3 and anti-PAD4
 
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.

10:10
Morning Networking Break
Biomarkers & Surrogate Endpoints for Clinical Trials in Inflammatory Diseases

10:55
Enabling Inflammatory Bowel Disease Interception with a Real–Time, Multidimensional Molecular Activity Score
 
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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11:20
Next Generation Immune Monitoring _ Epigenetic Assays for Immune Cell Measurements in Clinical Trials
 
Ulrich Hoffmueller
Founder & Chief Business Officer
Epiontis GmbH
About Speaker: Since the foundation of Epiontis in 2003, in which Uli participated as a founder, he is Chief Business Officer of the company. Prior to founding Epiontis, he worked at Jerini AG, Berlin, Germany (now part of Shire Pharmaceuticals) from 1999 to 2003 a... Read Full Bio 
 
 
Ulrich Hoffmueller
Founder & Chief Business Officer
Epiontis GmbH
 
About Speaker:

Since the foundation of Epiontis in 2003, in which Uli participated as a founder, he is Chief Business Officer of the company. Prior to founding Epiontis, he worked at Jerini AG, Berlin, Germany (now part of Shire Pharmaceuticals) from 1999 to 2003 as Director of Business Development. Uli completed business school in Berlin, Germany and Cambridge, UK from 2002 to 2004 in parallel to his positions in the biotech industry. He earned his Ph.D. at the Charité University Clinic, Berlin, Germany, in 1998, and was honored with the Charité Research Award in 1997. Uli received his Diplom (Masters) in Biochemistry in 1995 from Humboldt University, Berlin, Germany.

 
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
12:35
Lunch Provided by GTCbio
Microbiomes in Immune Modulation & Inflammatory Diseases

2:10
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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2:35
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

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
4:10
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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4:35
Clinical Proteomics Approach in Severe Asthma Investigations
 
Pierluigi Mauri
Chief of Proteomics & Metabolomics
Proteomics and Metabolomics Laboratory - CNR, Institute for Biomedical Tecnologies (ITB-CNR)
About Speaker: ... Read Full Bio 
 
 
Pierluigi Mauri
Chief of Proteomics & Metabolomics
Proteomics and Metabolomics Laboratory - CNR, Institute for Biomedical Tecnologies (ITB-CNR)
 
About Speaker:
5:00
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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5:25
Networking Reception & Poster Session
Day - 3 Wednesday, March 22nd, 2017
7:30
Continental Breakfast & Registration
Overcoming Commercialization Challenges in Biomarker Development

8:30
Personalised Healthcare Solutions in Oncology and Beyond - Where Next?
 
Bruce Jordan
Vice President, International Business Leader, Personalised Healthcare Solutions (PHCS), Centralised and Point of Care Solutions
Roche Diagnostics
About Speaker: ... Read Full Bio 
 
 
Bruce Jordan
Vice President, International Business Leader, Personalised Healthcare Solutions (PHCS), Centralised and Point of Care Solutions
Roche Diagnostics
 
About Speaker:
 
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
The Evolving CDx Deal Landscape: Are Progressive Deal Structures the Way of the Future?
 
Gary Gustavsen
Vice President
Health Advances, LLC
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, LLC
 
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.

 

 

9:45
Guidance on How to Transform Biomarkers into Clinically Relevant Information
 
Katherine Tynan
President
Tynan Consulting LLC
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 LLC
 
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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10:10
Morning Networking Break
Panel Discussion: Companion Diagnostics Development & Partnering Strategies

10:40
Patricia McLoughlin
Director of Business Development, Personalised Healthcare Molecular Diagnostics
Qiagen
About Speaker: Patricia McLoughlin PhD is Director Precision Diagnostic & Pharma Partnering, Molecular Diagnostics at QIAGEN, where her responsibilities involve establishing and managing partnerships with Pharma clients for the development of Companion Diagnost... Read Full Bio 
 
 
Patricia McLoughlin
Director of Business Development, Personalised Healthcare Molecular Diagnostics
Qiagen
 
About Speaker:

Patricia McLoughlin PhD is Director Precision Diagnostic & Pharma Partnering, 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.
Dr McLoughlin started her career as a Research Assistant at The State University of New York before moving to Affymax Research Institute in California as a Research Associate. Dr McLoughlin then moved to F Hoffmann - La Roche Pharmaceuticals in Basel in 2002 where she worked her way through roles of increasing responsibility in the core Genomics and biomarker technology department of Roche, until she was head of Genomics in 2008. During this time she was a key contributor to the establishment of profiling technologies and biomarker sampling in clinical trials across all disease areas in which Roche is active. Her last role at Roche was VP Global Head of Operations, in the Translational Research Sciences department. Most recently Dr McLoughlin worked at Leica Biosystems as Director of Strategic Alliances – Pharma Partnerships where she established relationships with Pharma clients requiring Immunohistochemistry-based CDx assays.
Dr McLoughlin holds a BSc (hons) in Microbiology from the National University of Ireland, Galway, and a Ph.D. in Molecular Biology from the University of Zurich.

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: ... Read Full Bio 
 
 
Zach Hornby
Chief Operating Officer
Ignyta
 
About Speaker:
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 LLC
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 c... Read Full Bio 
 
 
Felix Frueh
Executive Partner
Opus Three LLC
 
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