Company Overview

1. Personalized Medicine in the Age of Induced Pluripotent Stem Cells (iPSCs): Giving the Right Cell or Drug to the Right PersonTom Novak, Ph.D.Vice President, Strategic PartnershipsCellular Dynamics International

2. Cellular Dynamics International (CDI) is the world’s largest producer of human iPS cells and iPS cell-derived cell types Headquartered in Madison, WI Currently employs ~116 total staff (mostly scientists) ~550 yrs human stem cell experience (in a field ~15 yrs old) >700 patents (owned or licensed) to enable FTO Core competencies Creation and culture of human iPS cells Normal and disease phenotypes Genetic engineering of iPS cells Lineage and pathway-specific markers can be introduced Development of new differentiation protocols Differentiated cells from all three germ layers Manufacture of human iPS cell-derived cell types Scalable production of highly purified cells Partnership with iPS-AJ enables access to CDI’s products in Japan Grants from NHLBI and CIRM to reprogram patient samples Company Overview The Wall Street Journal Gold Winner: CDI Technology Innovation Awards 2011

3. iPS cells are uniquely useful stem cells Derived from adult tissue via non-invasive methods Can be expanded indefinitely Can be differentiated into any cell type in the body Fully pluripotent iPS cells offer distinct advantages to ES cells Can be created via streamlined & non-invasive methods Eliminates political/social issues regarding tissue source Enables diversity of genotype and phenotype iPS Cell TechnologyUnlimited Potential Differentiate iPS cells into any cell type in the body (unlimited numbers) Draw 1 small sample from 1 person Reprogram sample tissue into iPS cells iPS cells multiply and expand in culture indefinitely CDI CONFIDENTIAL

4. Personalized Medicine: Past and Present • CYP variants play a key role in drug metabolism • Patients may suffer side effects when they can’t metabolize a drug or may receive no therapeutic benefit if a metabolite is the active agent • CYP2C9:persons with less-active variant can use lower doses of warfarin (anti-coagulant) • CYP2D6: at least 50 drugs are known to be metabolized primarily by 2D6 • e.g., persons with 2 null alleles do not metabolize codeine to morphine • Examples of drugs whose efficacy is influenced by non-CYP genetics • Gleevec (Novartis)- small molecule inhibitor of BCR-ABL for treatment of CML • Herceptin (Genentech)- mAb against ErbB2 (HER2) for breast cancer • aggressive tumors often overexpress ErbB2 • antibody blocks growth-promoting effect of ErbB2 overexpression • Iressa (Astra Zeneca)- small molecule inhibitor of the EGFR tyrosine kinase • effective in treating nonsmall cell lung carcinoma in patients with hyperactive EGFR • Cell therapy • Use of autologous (or HLA-matched) Hematopoietic Stem Cells following myeloablative therapy

5. The Future of Personalized Medicine Near-term (0-10 years) • Use of iPSC lines to generate in vitro human-specific functional models • Comparison of control and patient-derived lines useful for studying disease biology and drug screening • iPSC-derived cardiomyocytes already being used to assess drug safety • Data has been included in several recent regulatory filings • However, at present, we can only make a small fraction of human cell types • Some diseases will be challenging to model due to low penetrance, long-latency, or lack of cell-autonomy • How many lines need to be compared? • Cheaper sequencing makes comparisons between drug safety/efficacy and genotype easier to predict • Privacy and discrimination issues still exist Longer-term (10-20 years) • Autologous or HLA-matched tissues and organs produced from iPSCs • Banks of HLA homozygous lines being established today • UCB banking replaced by iPSC banks (or other source material)? • Routine use of in vitro “Clinical Trials” to select clinical candidate • Compare several drug candidates of panels of genetically diverse patient-specific iPSC-derived cells • Measure both efficacy and safety • Data used to recruit appropriate patients, reducing size, cost and failure rate of clinical trials