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THURSDAY, FEBRUARY 26 

7:20am Plenary Keynote Introduction

7:30 PLENARY KEYNOTE 
Tissue Engineering Strategies for Musculoskeletal Regenerative Medicine in Civilian and Military Applications
Michael J. Yaszemski, M.D., Ph.D., Brigadier General, United States Air Force Reserves; Professor, Orthopedic Surgery and Biomedical Engineering, College of Medicine, Mayo Clinic
Tissue regeneration via tissue engineering strategies requires some combination of cells, a scaffold upon which the cells can attach and express their phenotypic function, and signaling molecules to direct the cells down the desired differentiation path. This cellular component often includes stem cells. This lecture will present current concepts regarding musculoskeletal tissue regeneration and the issues to be considered for its translation to clinical practice, as well as the unique reconstructive challenges encountered in combat injuries. 

OPTIMIZING TRANSLATION TO FIH STUDIES

8:25am Chairperson’s Remarks 

8:30 T-lymphocyte Targeting Treatment Strategies in Chronic Inflammatory Disorders: Translation from Preclinical Disease Models to PoM Studies
Gerhard Wolff, M.D., Ph.D., Global Clinical Director, Translational Medicine Leader, Clinical Research and Exploratory Development, Hoffmann-La Roche, Inc.

9:00 Translational Research in Neurodegenerative Diseases: From Discovering the Causes of Disease to Developing Cures
B. Michael Silber, Ph.D., Chief, Drug Discovery R&D, Institute for Neurodegenerative Diseases, Department of Neurology; Adjunct Professor, Neurology and Biopharmaceutical Sciences, School of Medicine, University of California San Francisco
There are no effective therapies or preventions for any of the neurodegenerative diseases. A fundamental reason for this is our continued lack of understanding of basic disease biology, availability of predictive cell-based assays and predictive animal bioassays, clinically relevant biomarkers of disease diagnosis, severity, progression, and response to therapy for each disorder. This talk examines how we can address these gaps in basic and translational medical knowledge toward the development of effective research and development efforts that will yield robust treatments and cures.

9:30 Translational Considerations for FIH Dose Selection: Application of Proof-of-Mechanism Biomarkers 
Mohammad Tabrizi, Ph.D., Director, Translational Sciences, Global PK-PD and Bioanalysis, MedImmune
Effective information flow and translation of accumulated knowledge across various development phases remain a major challenge in drug development. Design of successful translational strategies from early phases of development process is not only necessary to lessen the development time and cost, but also to foster implementation of rational decision making processes. This presentation highlights a science-based decision making approach, through application of proof-of-mechanisms biomarkers in designing effec-tive strategies, for translation of preclinical data into First-in-Human (FIH) clinical studies. 

10:00 Protein Biomarkers: Bridging the Gap between Discovery and the Clinic Sponsored by
Daniel Chelsky, Ph.D., Chief Scientific Officer, Caprion Proteomics, Inc.
Identification of predictive biomarkers of response to a specific therapy is of significant interest to most pharmaceutical companies. Multiple candidate biomarkers can be identified by comparing the plasma proteome of responders and non-responders to a drug, to find those proteins that correlate with response. Until recently, however, validat-ing these candidate markers in a larger patient population has been a challenge. Multiplexed multiple reaction monitoring (MRM) assays are developed rapidly and can be used to qualify up to fifty biomarkers in a subsequent clinical study.

10:30 Poster Competition Refreshment Break & Raffles in the Exhibit Hall

11:30 From Early Discovery to First-in-Human: Realities of the Post-Technology Revolution
Kailash Swarna, Ph.D., Executive Director, Research & Development, Amgen, Inc.

12:00pm Human Microdosing Phase 0
Colin Garner, Ph.D., President, Chief Executive Officer, Xceleron Inc.
Using one hundredth of the predicted pharmacologic dose, developmental drug candidates can be dosed into human volunteers straight out of discovery. With reduced pre-clinical toxicology and GMP-like drug substance, this technique has been used to obtain very early pharmacokinetic data on drug candidates to ensure that only the most promising drug candidates are taken forward. I’ll discuss a recent microdose study, comparing 7 drugs at both a microdose and pharmacologic dose. 

12:30 Luncheon Presentation (Opportunity available) or Lunch on your own

1:30 Plenary Keynote Introduction

1:40 PLENARY KEYNOTE
Engineering Cells to Death 
James A. Wells, Ph.D., Chair, Department, Pharmaceutical Chemistry; Professor, Pharmaceutical Sciences, Departments of Pharmaceutical Chemistry and Cellular & Molecular Pharmacology; Director, the Small Molecule Discovery Center, University of California, San Francisco 
Apoptosis, or programmed cell death, represents an ultimate fate decision in cell biology. This process is critical for cellular differentiation and remodeling of tissues, and for anti-viral and anti-tumor defense. The study of apoptotic pathways has important ramifications for determining what is critical for cellular homeostasis, and for the development of potential anti-cancer therapeutics. A distinct molecular feature of apoptosis is the widespread but controlled cellular proteolysis, that is predominantly mediated by eight members of the caspase family of cysteine proteases. These enzymes are like demolition experts that cleave protein targets critical for cellular life. We have designed new enzymes, and antibodies, and small molecules to study and activate individual caspases and the proteins they cleave. For example, a robust proteomic method for global profiling of proteolysis (“degradomics”) in cells has been developed. Key to this is an engineered enzyme, subtiligase, that permits selective labeling and enrichment for the protein N-termini created as a result of proteolysis. Using this approach we have already identified >300 caspase substrates from Jurkat cells that were induced to undergo apoptosis by treatment with the chemotherapeutic agent etoposide. The proteins fall into a wide range of functional classes, and reveal much about the molecular components, logic, and timed sequence of events that drive a cell from life to death. We believe these engineered enzymes and proteomic approaches will be useful for characterizing the proteolysis of apoptosis induced by various agents or in different cell types, and will be generally useful for dissecting protease signaling pathways. 

2:25 PLENARY KEYNOTE
Brave New Age of Personalized Medicine
David Ewing Duncan, Chief Correspondent, NPR Talk’s “Biotech Nation” and Best Selling Author “Masterminds”
This focus of this presentation will be on "Creative Disruptions", and will demonstrate the walking scientific response to the question: "Can they really do that?" The most important and controversial topics of today’s scientific research will be discussed, from stem cells and synthetic biology, to rising drug prices and reforming the FDA. Recently, there has been attention on science’s most significant story: a species’ potential to self-evolve. As the founder of the independent BioAgenda Institute for Life Science Studies and, more recently, as the founder of the new Center for Life Science Policy at UC Berkeley, the passion for what comes next after new technologies appear will be explored -- what happens in business, politics, science, philosophy, the media, the arts, and to society as a whole.

3:05 Ice Cream Refreshment Break in the Exhibit Hall with BEST NEW PRODUCT AWARDS (Last chance for viewing posters and exhibits)

IMPLEMENTING PERSONALIZED MEDICINE

3:55 Chairperson’s Remarks

4:00 Translational Strategies for Personalized Medicine
Bruce H. Littman, M.D., President, Translational Medicine Associates, LLC (formerly Vice President, Global Head, Translational Medicine, Pfizer)
Molecular definition of disease is rapidly replacing traditional pathology-based disease descriptions in cancer, partly because of its utility in identifying optimal treatment regi-mens. It’s clear that the same changes are coming for many common chronic diseases. Utilizing molecular disease definitions, these chronic disease phenotypes can be split into distinct subpopulations with important implications for safe and effective personalized treatment choices.

4:30 Bringing Personalized Medicine into Practice: The National Medco Experience
Teresa DeLuca, M.D., M.B.A, Vice President, Department of Personalized Medicine - Business Solutions, Medco
This talk provides in-depth information drawn from recent national Personalized Medicine programs for several disease areas, discussing the type of evidence required by pay-ers for coverage, the barriers and accelerators related to physician adoption and those related to patient adoption as well. For those developing drugs or tests, this talk pro-vides real-life experience from hands-on work with more than100 different payers across the US.

5:00 Paradigms on the Development of Personalized Medicine: A Regulatory Perspective
Francis Kalush, Ph.D., Network Leader, Diagnostics, Office of the Center Director, Center for Devices and Radiological Health, Food and Drug Administration
Personalized medicine has the potential to improve patient care, optimize therapeutics development with the help of new diagnostics; and improve benefit/risk ratio to patients. However, major challenges such as development of strong scientific evidence, evolving business/regulatory models and changes to the current reimbursement process are essential to bring these tests into routine clinical practice. This talk focuses on the regulatory perspective, highlighting recent FDA initiatives to facilitate the integration of pharmacogenomics into drug development and clinical practice.

5:30 Expert Panel: Translational Medicine Best Practices

• What strategies can reduce late-stage attrition?
• What predictive tools are most valuable in evaluating drug candidates? How can these predictive tools be used in combination for optimum results?
• Are animal models representative of human response? How can they be improved?
• How can iterative learning between clinical and pre-clinical development be efficiently implemented? What lessons can be taken from the clinic back into development?
• What are the unmet data/knowledge management needs in integrating pre-clinical and clinical information and enabling efficient bi-directional information flow?
• How are the patient needs changing and what does that mean for pharmaceutical R&D?
• Is there benefit to consortia in translational medicine?

6:00 Close of Day 

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Julia Boguslavsky, Executive Director, Conferences
E-mail: juliab@healthtech.com  

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781-972-5471 or dinerstein@healthtech.com 

Jon Stroup, Manager, Business Development
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