(SC5) TARGETING CANCER STEM CELLS WITH BIOLOGICS

• Novel Nanoparticle Fusion Proteins, TR1 and TR4, that Achieve Normal p21 Delivery to p53/p21 Mutated Tumors (TR1) and Inhibition of Notch Signaling (TR4) Resulting in Tumor Eradication
• Differentiation Versus Self-renewal: Changing Cancer Stem Cell Fate by Targeting Stem Cell Pathways
• Cancer Stem-like Cells: Isolation using Biological Criteria and use in Drug Discovery and Development
• CD47: An Adverse Prognostic Factor and Therapeutic Antibody Target on Human Acute Myeloid Leukemia Stem Cells
• Identification of Stem Cell Markers in the Normal Prostate and Prostate Cancer

Course instructors:
Agamemnon Epenetos Ph.D, FRCP, Chairman, Trojantec Ltd.
Austin Gurney, Ph.D., Vice President, Molecular and Cellular Biology, OncoMed Pharmaceuticals Inc.
Jennie P. Mather, Ph.D., Senior Vice President, Stem Cell Research, MacroGenics Inc.
Ravi Majeti M.D., Ph.D., Assistant Professor, Division of Hematology, Stanford Cancer Center
Institute for Stem Cell Biology and Regenerative Medicine
Kevin G. Leong, Ph.D., Scientist, Tumor Biology and Angiogenesis, Genentech Inc.

(SC6) DEALING WITH THE BLOOD-BRAIN BARRIER

• The physiological basis for the "barrier” nature of the BBB
• Experimental approaches (in vitro/in vivo) that are available for screening for brain penetration
• Medicinal chemistry perspective on in vitro/in silico approaches for optimizing CNS penetration
• Multi-parameter optimization (MPO) for CNS penetration
• In vivo examples where all these concepts are applied together, e.g., consideration of free fractions in various compartments in relation to in vitro pharmacology values
• Projecting human receptor occupancies considering species differences in affinity, free fraction
• Exposure targeting for biomarker studies

Course Instructors:

Christopher L. Shaffer, Ph.D., Associate Research Fellow, Pharmacokinetics, Dynamics & Metabolism, Pfizer, Inc.

Douglas Spracklin, Ph.D., Director, Pharmacokinetics, Dynamics and Metabolism, Pfizer, Inc.

Travis T. Wager, Ph.D. Associate Research Fellow, Neuroscience Discovery, Medicinal Chemistry, Pfizer, Inc.

Afternoon Courses: 2pm – 5pm

(SC7) BEST PRACTICES IN TRANSLATIONAL & PERSONALIZED MEDICINE

• Real world solutions currently in place in pharma, national labs, academia, and industry
• Building collaborations and sharing biological data between Big Pharma
• Bridging the gap between bench and bedside
• Informatics solutions that link data from the clinic with cutting edge research

Course Instructors:

Jeffrey S. Barrett, Ph.D., FCP, Pediatrics Director, Pediatric Pharmacology Research Unit, The Children’s Hospital of Philadelphia

Lisa LaLuna, Senior Vice President, Corporate Development & Implementation, ePharmaSolutions

Jeremy Packer, Head, Bioinformatics, Abbott

Faye D. Schilkey, Associate Director, NM Sequencing Center, National Center for Genome Resources

(SC8) STRATEGIES FOR MOLECULAR DIAGNOSTIC COMPANIES

Achieving Success in Rapidly Changing Markets

• Why and how diagnostics markets have changed
• Strategies for success: conventional or new markets?
•  Major business model questions including partnering
• How to obtain your next round of funding

Course Instructors:

Keith F. Batchelder, Chief Executive Officer, Genomic Healthcare Strategies
Peter S. Miller, Chief Operating Officer, Genomic Healthcare Strategies

(SC9) FRAGMENT-INSPIRED MEDICINAL CHEMISTRY

• Fragment-based approaches as platforms for medicinal chemistry
• Fragment-based methods that inspire fresh approaches to lead generation
• Optimization of fragment hits
• Combining technology with fragment-based methods to advance medicinal chemistry
• Facing the challenge of applying fragment-based approaches when structural information is not available
• Promises and pitfalls of surface plasmon resonance (SPR) for fragment methods

Course Instructors:

Michelle Arkin, Ph.D., Associate Director, Biology, Small Molecule Discovery Center, PharmaceuticalChemistry, University of California, San Francisco

Daniel A. Erlanson, Ph.D., Co-founder, Carmot Therapeutics, Inc.

(SC10) TRANSPORTER-MEDIATED DRUG-DRUG INTERACTION POTENTIAL: STRATEGIES FOR IN VITRO CHARACTERIZATION

• Clinical relevance of transporter DDI’s
• In vitro, cell based models for evaluating transporter interactions of substrates and inhibitors
• Case study: Minimizing p-glycoprotein interactions as a barrier to CNS penetration

Course Instructors:

Phil Burton, Ph.D., Chief Executive Officer & Chief Scientific Officer, ADMETRx, Inc.

Xingrong Liu, Ph.D., Senior Scientist, DMPK, Genentech, Inc.
Joseph A. Ware, Ph.D., Senior Scientist, Clinical Pharmacokinetics and Pharmacodynamics, Development Sciences, Genentech, Inc.

(SC11) Pharma BASIC IMMERSION: CUTTING EDGE SCIENCE & TECHNOLOGY FOR BIOTECH & PHARMA
Download Brochure | Register Today

• Gain a fundamental understanding of the science and technology driving the Biotech/Pharma industry
• Learn basic scientific terminology used by researchers in the life sciences
• Designed for the non-scientist working with or in the biotech/pharma industry
• Immersion course on the biotech basics; Recombinant DNA, Proteins, Stem Cells, Biologics, Drug Discovery and Drug Development

Class Materials Include: The Primer: A Biotechnology Guide for the Non-Scientists

Course Instructor

Karin Lucas, Ph.D., BioTech Primer Instructor and Scientific Advisor

Karin Lucas, Ph.D., has been teaching with BioTech Primer, Inc. for the past five years. As a scientist at Biogen Idec she develops protein pharmaceuticals for the treatment of cancer and multiple sclerosis. Previously, Dr. Lucas was a scientist and project director at Cardinal Health where she worked on the development of over 25 products with multiple pharmaceutical and biotechnology companies. In addition to her laboratory role, she is also trained as a Lean Six Sigma greenbelt. Dr. Lucas is an active community volunteer and has served as the PR chair and later Vice President of the San Diego chapter of AWIS (Association for Women in Science). In 1998, Dr. Lucas was honored as the Cal Poly Physical Chemistry Student of the Year and in 2003 she was selected for the AWIS San Diego Rookie of the Year Award. Dr. Lucas received her B.S. in biochemistry from California Polytechnic State University, San Luis Obispo and went on to complete her Ph.D. in biochemistry at University of California San Diego.

(SC12) DESIGNING RIGOROUS OMICS STUDIES FOR BIOMARKER DISCOVERY AND DEVELOPMENT OF PROGNOSTIC AND PREDICTIVE MOLECULAR DIAGNOSTICS
Download Complimentary Course White Paper: Smarter Studies: Designing Rigorous Omics

Course Agenda

2:00 Experimental Design in Genomics, Proteomics and Metabolomics

Terry Speed, Professor, Department of Statistics, University of California, Berkeley; Head, Bioinformatics, WEHI, Australia

For most experiments in this area, we can recognize three main phases that are relevant to experimental design. I: choice and preparation of experimental material, choice of platform technology; II: assignment of experimental reagents to components of the technology; III: actual conduct of the experiment, including times, places and conditions of experiment, and protocols, reagents, operators and equipment used. In this talk we will mainly focus on examples where the experiments were poorly designed and led to unsatisfactory outcomes. This will show the need for good design

2:45 A Roadmap for Omics Study Design

Juergen von Frese, Ph.D., Managing Director, Data Analysis Solutions DA-Sol GmbH, Germany

Any biomarker or predictive or prognostic signature can only be as good as the data it was derived from. Study design critically determines the inherent signal (i.e. information) in the data. This talk will provide a systematic overview and roadmap for designing omics studies. This will range from defining study aims, determining sample size, randomization and reference measurements to an in-depth discussion of tackling confounding factors. Examples from cancer diagnostics will be given and crucial issues and pitfalls will be highlighted.

3:30 Refreshment Break

4:00 Beyond the Pilot Studies: FDA Perspectives on Biomarker Qualification

Donna Roscoe, Ph.D., Senior Reviewer, FDA/OIVD/DIHD

Major advances in research technology are allowing for the unprecedented identification of multiple genetic and protein biomarkers. The leap from characterizing these signatures to validating them for a specific use and applying them to patient diagnosis, screening, and therapeutic intervention is best accomplished with an understanding of the FDA regulatory process. This talk is intended to provide an overview of the current perspective of the Office of In Vitro Diagnostic Device Evaluation and Safety (OIVD) in the FDA's Center for Devices and Radiological Health (CDRH) on the issues related to assessing analytical and clinical performance of biomarker-based in vitro diagnostic assays for use in clinical practice or medical product development.

4:45 Q&A

5:00 End of Course