Day 1
Day 2
Hotel & Travel
PDF Download
Sponsorship/ Exhibits
Press Pass 
Request Brochure 



eCliniqua Logo 

Science Magazine – AAAS 

Insight Pharma Reports 


The Scientist 

Gene Therapy Net 


Pharm Cast 


Stem Cells 

Wednesday, November 2, 2011

7:30-8:30 am Conference Registration and Morning Coffee

8:30-8:40 Welcoming Remarks from Conference Director

Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute


Stem Cells in Drug Screening and Toxicology

8:40-8:45 Chairperson’s Opening Remarks

Timothy J. Nelson, M.D., Ph.D., Assistant Professor of Medicine and Pharmacology, Mayo Clinic

8:45-9:15 The Role of Stem Cell-Derived Tissues in Early Safety Screening

Liang Guo, M.D., Senior Scientist, Non-Clinical Safety, F. Hoffmann-La Roche

Safety-related attrition is a major concern for pharmaceutical development, yet organ toxicities are not assessed until late stage development. Human pluripotent stem cell-derived tissues offer an opportunity to create functional models amenable to high-throughput, low compound usage assays. Our work has focused on cardiomyocytes, specifically developing a pro-arrhythmia assay to screen for pro-arrhythmic potential early in development.

9:15-9:45 Combining HTS and HCS with Pluripotent Stem Cells in the Search for Treatments of Monogenic Diseases

Marc Peschanski, M.D., Ph.D., Head, Stem Cell Research and Therapy, I-Stem

Pluripotent stem cell lines derived from donors who carry a mutant gene at the origin of a monogenic disease can be used to screen libraries of compounds in a search for new treatments. Robust read-outs relevant to the pathological mechanisms should first be identified. On this basis, a screening platform either in high-throughput or in high content can be implemented, as derivatives of pluripotent stem cells can be obtained at near homogeneity and are amenable to miniaturization and standardization of cell processes. In parallel, functional genomics can also be implemented on large-scale platforms, in a search for yet unknown mechanisms and proteins involved in pathological signaling pathways.

9:45-10:15 Talk Title to be Announced

Hirdesh Uppal, Ph.D., Scientist, Genentech

10:15-10:45 Using Embryonic Stem Cells for Toxicological Studies

Marianne Barrier, Ph.D., Biologist, Systems Biology Branch, Integrated Systems Toxicology Division, National Health and Environmental Effects Research Laboratory, U.S. Environmental Protection Agency

10:45-11:30 Networking Coffee Break in the Exhibit Hall with Poster Viewing

11:30-12:30 Panel Discussion: How Are Stem Cells Impacting Drug Discovery and Development?

12:30 pm-2:00 Enjoy Lunch on Your Own


Stem Cell Models for Functional Analysis and
Target Discovery

2:00-2:05 Chairperson’s Opening Remarks

Timothy J. Nelson, M.D., Ph.D., Assistant Professor of Medicine and Pharmacology, Mayo Clinic

2:05-2:35 Regenerative Theranostics: Customizing Platforms for Rare Diseases

Timothy J. Nelson, M.D., Ph.D., Assistant Professor of Medicine and Pharmacology, Mayo Clinic

Nuclear reprogramming generates iPS cells endowed with the unlimited potential to reconstruct genetically identical tissues. This biomedical tool offers unprecedented opportunities to develop scalable, yet personalized cell-based reagents. The emerging platform of regenerative theranostics provides a unique approach to expose mechanisms of disease etiology in the context of dysfunctional cell biology. Focusing on rare diseases of unknown etiology, iPS cell technology offers a robust platform to efficiently dissect molecular dynamics for the purpose of developing diagnostic or therapeutic applications. Here, we will discuss metabolic deficiencies and the role of nuclear reprogramming to enable next-generation, patient-specific tools to accelerate pharmaceutical or biologic solutions for rare diseases.

2:35-3:05 Electrophysiological Studies of Ion Channels Expressed in Neurons Derived from Human Stem Cells

Robert F. Halliwell, Ph.D., Professor, School of Pharmacy, University of the Pacific

Neurons derived from human stem cells (hSCs) represent a powerful tool in drug discovery yet there is little data on the receptors and ion channels expressed in these cells. Our lab has used patch-clamp electrophysiology to address the neuropharmacological properties of neurons derived from the human stem cell line, TERA2.cl.SP12. Neurons derived from these hSCs expressed a range of voltage and ligand-gated ion channels with a pharmacology consistent with that of native human neurons. This study therefore supports the hypothesis that stem cells may provide a valuable source of human neurons for future neuropharmacological studies.

3:05-3:35 Stem Cell-Based Mouse Models for Translational Research

Yinghui Zhou, Ph.D., Principal Scientist, AVEO Pharmaceuticals

Genetically engineered mouse models have shown great promise to faithfully capture the complexity of genomic changes seen in cancer and have demonstrated their utility in cancer drug development. By applying genetic engineering in mouse embryonic stem cells, as well as mouse or human tissue stem cells, we have developed several novel approaches for modeling cancer in mice allowing for fast and flexible model generation. Tumors developed in these models feature human relevant genetic changes and complex response to cancer therapeutics as seen in human patients, and constituted the foundation of our population-based archive platform for translation research and biomarker discovery.

3:35-4:20 Networking Refreshment Break in the Exhibit Hall with Poster Viewing

4:20-4:50 Using iPS Cells to Study Cellular and Molecular Phenotypes Associated with Human Brain Disorders

Alex Shcheglovitov, Ph.D., Postdoctoral Fellow, Department of Neurobiology, Stanford University

The outstanding question of contemporary neuroscience is to understand the molecular and cellular basis of the human brain disorders. This knowledge would allow us to develop effective strategies to intervene and correct pathological changes. Induced pluripotent stem cells (iPSCs) are a new tool in our arsenal to study neurobiology in humans. These cells have the ability to generate any cell type from the human body including neurons. As iPS-derived neurons share the precise genetic background of a patient, they offer unique opportunities to probe the cellular and molecular properties of the human brain, to generate in vitro models of human diseases, and to develop high-throughput screens to identify potential therapeutics. In my presentation I will discuss the progress we have been making in the generation of a specific population of functional iPS-derived neurons and our attempts to find and rescue the cellular phenotypes associated with Phelan-MacDermid syndrome (a rare autism-related neurodevelopmental disorder).


4:50-5:20 Physiologically Functional Cellular Models for Modern Drug Discovery

Marsha Roach, Ph.D., Executive Vice President, Research & Development, GigaCyte

Early identification of safety and efficacy issues for drugs in humans is often difficult with lead discovery and screening occurring in animal cells or tumor cells expressing targets of interest. This can be overcome by early evaluation in normal human cells displaying physiologically relevant expression, metabolism and responses. To assist in the generation of these contextual safety and efficacy models we have focused our efforts on developing physiologically-relevant functional cell types from renewable stem/progenitor cells to produce predictive cellular assay systems. Here data will be presented that demonstrates how liver biomatrix is the key to producing functional human hepatocytes on a scale needed for safety and efficacy evaluation early in the drug discovery process; how media formulation directs the differentiation of human neural stem cells into very mature excitatory and inhibitory neurons for high-throughput screening; and how process optimization impacts purity and quality of human islets for drug testing.

5:20-6:15 Networking Reception in the Exhibit Hall with Poster Viewing

Group Discounts are Available! Register a group of 2 or more and save up to 30%. For details, contact David Cunningham at 781-972-5472.