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THURSDAY, FEBRUARY 26

7:00 am Registration and Morning Coffee

7:20 Plenary Keynote Introduction
Deepak Srivastava, M.D., Professor and Director, Gladstone Institute of Cardiovascular Disease University of California San Francisco

Plenary Keynote 
7:30 Tissue Engineering Strategies for Musculoskeletal Regenerative Medicine in Civilian and Military Applications
Michael J. Yaszemski, Ph.D., M.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. 

8:25 Chairperson’s Remarks
Robert Deans, Ph.D., Senior Vice President, Regenerative Medicine, Athersys, Inc.

THERAPEUTIC THEMES: CONSIDERATIONS FOR CARDIAC REGENERATION

Featured Presentations
8:30 Directed Differentiation of Pluripotent Stem Cells for Heart Disease
Deepak Srivastava, M.D., Professor and Director, Gladstone Institute of Cardiovascular Disease University of California San Francisco 

9:00 Stem Cells for Cardiac Regeneration 
Eduardo Marbán, M.D., Ph.D., Founding Director, Heart Institute, Cedars-Sinai 

9:30 Human Embryonic Stem Cells in Safety Pharmacology: A Powerful Tool to Predict Arrhythmias?
Martin Traebert, Ph.D., Head, Safety Pharmacology EU, Preclinical Safety, Novartis Pharma AG 
An introduction to the field of cardiosafety testing and the pharmaceutical industry background of differentiation of human embryonic stem cell into cardiomyocytes will presented. In addition, the potential future of the technology within the Novartis test strategy and biochemical and electrophysiological characterization of these cardiomyocytes.

10:00 Cardiology Panel of Experts
Biologic Perspective: Deepak Srivastava, M.D., UCSF
Clinical Perspective: Eduardo Marbán, M.D., Ph.D., Cedars-Sinai
Screening: Martin Traebert, Ph.D., Novartis Pharma AG
Clinical Trial Design: Dawn Driscoll, Ph.D., DCi Biotech
Imaging: Joseph Wu, M.D., Ph.D., Stanford University 
Manufacturing: Nicolas L'Heureux, Ph.D., Cytograft 

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

11:30 MMTC Breakout Groups Hosted by the Cardiac Panel of Experts
Motivating Meeting for the Therapeutic Community
Join the focused discussion tables hosted by:

Table 1
Generating Appropriate Therapeutic Stem Cells for Heart Disease
Host: Deepak Srivastava, M.D., Professor, UCSF
Discussion topics include:

• What are best cell sources?
• What are hurdles in differentiation?
• What are risks of therapy?
• What are hurdles for expansion?

Table 2
Realistic Forecasting of Cell Therapy Sales
Host: Dawn Driscoll, M.B.A., Ph.D., Principal, DCi Biotech
Your Investors, Your Manufacturing group, Your HR department, even your sub-conscience wants to know, "How much of this therapy are you really going to sell?" This interactive discussion will address:

• Basics of forecasting for both allogeneic and autologous cell therapies, in order to support decision making for clinical development, investor presentations, and manufacturing capacity;
• Determining realistic patient numbers, the impact of reimbursement on sales, the impact of projected sales on GMP manufacturing capacity;
• Staffing needs to support a given forecast.

Table 3
Imaging of Adult & Embryonic Stem Cells in the Heart
Host: Joseph Wu, M.D., Ph.D., Stanford University
Discussion topics:

• Which imaging modality to choose?
• Do stem cells survive long-term in the heart?
• Why understanding stem cell fate is important?

12:30 pm Luncheon Presentation Ultrasensitive Multiplexed Biomarker Assays from Discovery to the Clinic Pankaj Oberoi, Ph.D., Director, Qualified Kit Development, Meso Scale Discovery Meso Scale Discovery (MSD) has an electrochemiluminescence platform that is fast (1-3 minutes per plate independent of plate density), robust (non-fluidics instrument), radioactive free, sensitive (detection limits near 10 attomoles) and has a wide dynamic range (5 logs) with multiplexing capabilities. The performance (sensitivity, reproducibility, and ease of use) of multiplexing cytokines, cell signaling pathways, metabolic assays, Alzheimer's assays and multiplexed toxicology biomarkers assays will be presented. The platform is an open platform that is amenable to assay development and has been used extensively for immunogenicity and development of novel cell based assays.

Sponsored by

1:30 Plenary Keynote Introduction 

1:40 Plenary Keynote 
Engineering Cells to Death
James A. Wells, Ph.D., Chair, Professor of Pharmaceutical Sciences, Departments of Pharmaceutical Chemistry and Cellular & Molecular Pharmacology, 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 
The Brave New World of Personalized Medicine: The Experimental Man Project, One Man Takes the Ultimate High-Tech Exam
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 OF SHOW AWARDS (Last Chance for Viewing Exhibits & Posters)

HARNESSING CELLS FOR REGENERATIVE HEALING

3:55 Chairperson’s Remarks
Lee Buckler, B.E.d, LLB, Principal, Cell Therapy Group

4:00  miRNA Mimic/Inhibitor Screen Identifies miRNA Involvement in Mesenchymal Stem Cell Differentiation Queta Smith, Ph.D., Associate Director Tech Communications, Thermo Fisher Scientific MicroRNAs play a fundamental role in a wide array of developmental events. Here, we have used a library of miRNA mimics and inhibitors to identify miRNAs that play a role in mesenchymal stem cell osteogenic differentiation. The miRNAs identified in the screen were able to trigger the expression of early osteogenic markers in the absence of other external signals (e.g., dexamethsome, ascorbate) and restore osteogenic potential to high passage number human MS cells. These findings define a role for miRNAs in human MS cell osteogenic differentiation and demonstrate the value of mimic/inhibitor screens in identifying biologically relevant miRNAs.

Sponsored by

4:30 Programming Cells in Situ
Omar Ali, Ph.D., Post Doctoral Fellow, Laboratory for Cell and Tissue Engineering, Harvard University 
There are hundreds of clinical trials of cell therapy currently underway, with the goal of curing a variety of diseases, but simple cell infusions lead to large-scale cell death and little control over cell fate. We propose a new approach, in which material systems are first used either as cell carriers or attractors of host cell populations, and in either case the material then programs the cells in vivo and ultimately disperses the cells to surrounding host tissues or organs to participate in tissue regeneration or destruction.

5:00 Genetically Engineered MSCs for CNS Regeneration
Casey Case, Ph.D., Vice President, Research, SanBio 
MSCs transiently transfected with Notch-1 are more effective than unmanipulated MSCs in models of stroke regeneration. We have new data concerning mechanism of action. In addition, we have established GMP manufacturing, successfully presented our protocol to the Recombinant DNA Advisory Committee (RAC) and submitted an IND to the FDA.

5:30 ES Cell Therapies for Muscular Dystrophy
Radbod Darabi, M.D., Ph.D., Senior Research Fellow, Lillehei Heart Institute and Department of Medicine, University of Minnesota 
Embryonic stem (ES) cells are endowed with self-renewal and broad differentiation potential. However, the generation of a population of myogenic progenitors from differentiating ES cells with significant regenerative potential has proven elusive. We have recently shown that it is possible to circumvent the defective EB environment by over-expressing Pax3, the master regulator of the myogenic program. This strategy, in concert with a cell purification method based on paraxial and lateral plate mesoderm surface markers, enabled us to generate a teratoma-free early population of myogenic progenitors from ES cells, capable of promoting extensive engraftment of adult myofibers and improvement in contractile function. We are currently assessing the long-term regenerative potential of the cells and whether they seed the satellite cell compartment in vivo. These results will be discussed.

6:00 Close of Day Two

Overview | Day 1 | Day 2 | Day 3 | Download Brochure | Breakout Discussions