2015 Harvard Chinese Life Science Symposium Harvard Chinese Life Science

Annual Research Symposium

March 28, 2015

Jimmy Fund Auditorium, Harvard Medical School, 35 Binney Street, Boston, MA, 02115

Harvard Medical School

Chinese Scholars & Scientists Association

Harvard School of Public Health Chinese Students & Scholars Association 1

2015 Harvard Chinese Life Science Symposium

Organizing Committee Advisors

Xi He, Professor, Boston Children’s Hospital, Harvard Medical School Xiaole Shirley Liu, Professor, Dana-Farber Cancer Institute, Harvard School of Public Health Yi Zhang, Professor, Boston Children’s Hospital, Harvard Medical School Jiping Wang, Assistant Professor, Brigham and Women’s Hospital, Harvard Medical School William Pu, Professor, Boston Children’s Hospital, Harvard Medical School Jean Zhao, Associate Professor, Dana-Farber Cancer Institute, Harvard Medical School

Jing Ma, Associate Professor, Brigham and Women’s Hospital, Harvard School of Public Health

Frank Hu, Professor, Harvard School of Public Health Jianzhu Chen, Professor, Massachusetts Institute of Technology

Organizing Committee

Harvard Medical School - Chinese Scientists and Scholars Association (HMS-CSSA)

Jin Zhang, Shaokun Shu, Zhe Ji, Qing Li, Shaojun Tang, Zhiqiang Lin, Dongpo Cai, Xiaofeng Wang, Wei Li, Tengfei Xiao, Chunxiao Yu, Xingxing Kong, Min Tan, Chunyao Wei, Wen Chen, Jiaren Liu, Ji Li, Hao Huang, Jun Huang, Hongguang Xia, Xiaoyang Zhang, Wenqing Cai, Xuezhe Han, Bin Li, Yu Qian, Dapeng Yan, Yiying Zhu, Song Yang; Sui Wang

Harvard School of Public Health - Chinese Students and Scholars Association (HSPH-CSSA)

Zhaozhong Zhu, Qian Di, Yinyin Xu, Xihao Li, Xiaoyu Li, Haiyue Zhang

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Agenda

9:00am – 9:30am Registration and Light Refreshment Opening Remarks

Dr. Edward Benz 9:30 am – 9:45 am President and CEO, Dana-Farber Cancer Institute, Harvard Medical School (Facilitator: Dr. Jin Zhang)

Technology Innovation Drives Development in Medicine (Facilitator: Drs. Shirley Liu and Xi He)

Life at the Single Molecule Level

9:45 am – 10:25 am Dr. Xiaoliang Xie Mallinckrodt Professor, Department of Chemistry & Chemical Biology, Harvard University

Understanding active demethylation and SCNT through tool development

10:25 am – 11:05 am Dr. Yi Zhang Professor, Boston Children’s Hospital, Harvard Medical School, Investigator, Howard Hughes Medical Institute

Genome Editing Technologies and Applications

11:05 am – 11:45 pm Dr. Feng Zhang Assistant Professor, Broad Institute, Massachusetts Institute of Technology

DNA probes for highly multiplexed super-resolution imaging

11:45 pm – 12:25 pm Dr. Peng Yin Associate Professor, Wyss Institute, Harvard Medical School

12:25 pm - 1:30 pm Lunch (Location: Yawkey Building dinning pavilion 3rd floor)

Cancer Research (Facilitator: Dr. Jiping Wang)

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A perspective in immune modulation therapy of cancer

1:30 pm – 2:10 pm Dr. Lieping Chen Professor, Yale University Director, Cancer Immunology Program at Yale Cancer Center Targeting the PI3K/PTEN pathway in cancer: from mouse genetics to human therapies

2:10 pm – 2:50 pm Dr. Jean Zhao Associate Professor, Dana-Farber Cancer Institute, Harvard Medical School

2:50 pm – 3:20 pm Distinguished Research Award Winner Talk

3:20 pm – 3:35 pm Tea Break

Translational Medicine (Facilitator: Dr. William Pu)

Update on use of gene therapy for monogenic diseases: the bad, the good and the future

3:35 pm – 4:15 pm Dr. David Williams Professor, Director of Translational Medicine, Boston Children’s Hospital, Harvard Medical School Viral Vector-Based In Vivo Gene Therapy

4:15 pm – 4:55 pm Dr. Guangping Gao Professor, University of Massachusetts Medical School

4:55 pm – 5:25 pm Distinguished Research Award Winner Talk

2015 Harvard Chinese Life Science Distinguished Research 5:25 pm – 5:50 pm Award Ceremony (Dr. Xi He)

6:15 pm – 9:00 pm Reception (Invited only)

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Speakers

Dr. Edward J. Benz Jr. President and Chief Executive Officer of Dana-Farber Cancer Institute

Dr. Benz is the Chief Executive Officer of Dana-Farber/Partners Cancer Care, Principal Investigator and Director of the Dana-Farber/Harvard Cancer Center, Director of the Dana Farber/Brigham and Women’s Cancer Center, and a member of the Governing Board of the Dana- Farber/Boston Children's Cancer and Blood Disorders Center. He is also the Richard and Susan Smith Professor of Medicine, Professor of Pediatrics, Professor of Genetics and Faculty Dean Emeritus for Oncology at Harvard Medical School. An internationally recognized hematologist, Dr. Benz has authored more than 300 peer-reviewed articles, reviews, chapters, and abstracts. His accomplishments have been recognized by a number of distinctions, including membership in the Institute of Medicine of the National Academy of Sciences, the Association of American Physicians, and the American Academy of Arts and Science. Dr. Benz is a graduate of Princeton University and Harvard Medical School. He received his training in internal medicine and hematology at Brigham and Women's Hospital, the Children’s Hospital of Boston, the National Institutes of Health, and the Yale University School of Medicine.

Dr. Xiaoliang Xie Mallinckrodt Professor, Department of Chemistry & Chemical Biology, Harvard University Member of the National Academy of Sciences

Xiaoliang Sunney Xie received a B.S. from in 1984, and his Ph.D. from the University of California at San Diego in 1990, followed by a short postdoctoral experience at the . In 1992, Xie joined Pacific Northwest National Laboratory, where he later became a Chief Scientist. In 1999, he was appointed Professor of Chemistry at Harvard University. He is now the Mallinckrodt Professor of Chemistry and Chemical Biology at Harvard, and the Cheung Kong Visiting Professor at Peking University, Biodynamics Optical Imaging Center (BIOPIC). Xie has made major contributions to the emergence of the field of single-molecule biophysical chemistry and its application to biology. His team also pioneered the development of coherent Raman scattering microscopy and single cell whole genome sequencing. His honors include the Harrison Howe Award, Biophysical Society Founders Award, E.O. Lawrence Award in Chemistry, Leibinger Innovation Prize, the NIH Director's Pioneer Award, the Sackler Prize for Physical Sciences. Xie is a fellow of the American Academy of Arts and Sciences and a member of the National Academy of Sciences.

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Dr. Yi Zhang Professor, Boston Children’s Hospital, Harvard Medical School Investigator, Howard Hughes Medical Institute

Dr. Zhang is currently an Investigator of the Howard Hughes Medical Institute and a Fred Rosen Chair Professor of the Department of Genetics and Department of Pediatrics of the Harvard Medical School. He is also a senior Investigator of the Program of Cellular & Molecular Medicine of the Boston Children’s Hospital. Before he moved to Harvard, he was a Kenan Distinguished Professor of University of North Carolina at Chapel Hill. The major interest of Dr. Zhang is to understand the epigenetic regulation in early development, stem cell reprogramming, differentiation, and reward-related learning and memory. Dr. Zhang is also interested in how dysregulation of chromatin modifying contribute to various human diseases, such as cancer, diabetes, and drug addiction. His group contributed to the identification and characterization of several classes of epigenetic enzymes that include histone methyltransferases, JmjC-containing histone demethylases, and the Tet family of dioxygenases for DNA demethylation. Dr. Zhang was Top 10 author of high-impact papers by ScienceWatch in Genetics and Molecular Biology from 2002-2006, and is also one of the most influential scientists in the world in the past 10 years.

Dr. Feng Zhang Assistant Professor, Departments of Brain and Cognitive Sciences and Biological Engineering. MIT Broad Institute of MIT and Harvard

Feng Zhang is a Core Member at the Broad Institute, an investigator at the McGovern Institute for Brain Research at MIT, and the W. M. Keck Career Development Professor at MIT. His lab develops molecular technologies and applications for interrogating biological systems, with an emphasis on the nervous system and its diseases. He is widely recognized for playing a central role in the development of technologies such as optogenetics and genome editing using CRISPR-Cas9. He has also played a leading role in making molecular technology reagents and know-how openly accessible.

Dr. Peng Yin Associate Professor, Harvard Medical School Core Faculty Member, Wyss Institute

Dr. Yin directs the Molecular Systems Lab at Harvard. His research interests lie at the interface of information science, molecular engineering, and biology. The current focus is to engineer information directed self-assembly of nucleic acid (DNA/RNA) structures and devices, and to exploit such systems to do useful molecular work. Such de novo designed systems are composed of small synthetic DNA/RNA monomers capable of conditional configuration change and can be

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programmed to self-assemble, move, and compute. They can serve as programmable controllers for the spatial and temporal arrangements of diverse functional molecules (e.g. fluorophores, proteins), with a wide range of applications in nano-fabrication, imaging, sensing, diagnostics, and therapeutics. He is a recipient of a 2010 NIH Director's New Innovator Award, a 2011 NSF CAREER Award, a 2011 DARPA Young Faculty Award, a 2011 ONR Young Investigator Program Award, a 2013 NIH Director's Transformative Research Award, a 2013 NSF Expedition in Computing Award, a 2014 ACS Synthetic Biology Young Investigator Award, and a 2014 Finalist for Blavatnik National Award for Young Scientists. See his lab's work at http://molecular-systems.net.

Dr. Lieping Chen United Technologies Professor in Cancer Research Professor of Immunobiology, Dermatology & Medicine Director of Cancer Immunology Yale University School of Medicine

Dr. Lieping Chen earned his medical degree from Fujian Medical School in . After completion of clinical training in immunology and oncology in Fujian Union Hospital and Union Medical College, he earned PhD from Drexel University in Philadelphia and did a postdoctoral fellowship in the University of Washington in Seattle. He worked as a research scientist in Bristol-Myers Squibb-Seattle 1990 - 1997. He became Associate Professor of Immunology in Mayo Clinic and promoted to the rank of Professor in 2000. He was Professor of Oncology and Dermatology in Johns Hopkins School of Medicine from 2004. Dr. Chen joined Yale University since 2011. Lieping Chen studies cell surface molecules and their functions in the modulation of immune responses. His laboratory is the first to use costimulation to enhance tumor immunity (by transfection of B7-1 gene into tumor cells). This study establishes the principle for costimulation and immune checkpoint blockade for cancer therapy. Dr. Chen co-discovered the PD-1/PD-L1 pathway and elucidated the role of the PD-1/PD-L1 pathway in tumor site immune suppression. More importantly, he invented anti-PD-1/PD-L1 therapy for cancer. His laboratory also discovered or co-discovered multiple immune modulation pathways, their functions and application in human disease treatment including 4-1BB (CD137), B7-H2 (ICOSL), B7-H3, B7-H4, B7-H5/CD28H, PD-1H (VISTA) and LIGHT/HVEM. Dr. Chen’s studies led to the development of anti-PD-1/PD-L1 therapy against broad spectrum of terminal stage human cancer (FDA approved drugs in 2014). In addition, agonist anti-4-1BB (CD137) antibodies (cancer), B7-H4Ig (rheumatoid arthritis) and anti-B7-H3 antibodies (cancer) are all in different stages of clinical development due to his laboratory discoveries. Dr. Chen has published more than 300 papers, review, book chapters and edited two books. He has received several awards and professional recognitions including United Technologies Endowed Chair in Cancer Research in Yale University (2013) and William B. Coley Award (2014). His work in discovery of the PD-1/PD-L1 pathway in cancer therapy was cited as the #1 breakthrough of the years by Science magazine (2013).

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Dr. Jean J. Zhao Associate Professor, Dana-Farber Cancer Institute, Harvard Medical School

Dr. Zhao received her PhD from Tufts Medical School in 1999 and had her postdoctoral research with Dr. Thomas Roberts at Dana-Farber, and joined HMS in 2006 as Assistant Professor. Dr. Zhao’s research centers on understanding kinase signaling pathways in cancer. She has pioneered a new front of understanding signal transduction by integrating mouse genetics and pharmacological approaches and changed the way we think about important problems in the targeted therapy of cancer. Specifically, the Zhao lab has conducted the leading work to determine distinct roles of isoforms of PI3K in the normal physiological functions and in the pathogenesis of cancer. Her work has laid a foundation for the new field of targeting PI3K isoforms in cancer and guided the design of current clinical trials of PI3K inhibitors both here at DFCI and internationally. Dr. Zhao has been highly recognized for her innovative research and excellence in the field of cancer research.

Dr. David A. Williams Chief of the Division of Hematology/Oncology, Boston Children’s Hospital Director of Clinical and Translational Research, Boston Children’s Hospital Associate Chairman, Department of Pediatric Oncology, Dana- Farber Cancer Institute Leland Fikes Professor of Pediatrics, Harvard Medical School

Dr. Williams is the Chief of Hematology/Oncology and Director of Clinical and Translational Research at Boston Children’s Hospital and Associate Chairman, Department of Pediatric Oncology at Dana-Farber Cancer Institute. He is Director of the Pediatric Hematology/Oncology Fellowship Training Program at BCH/DFCI. He was a Howard Hughes Medical Institute Investigator for 16 years and his laboratory has been continuously NIH funded since 1986. He has trained over 45 fellows and post-doctoral fellows and numerous residents and medical students in the laboratory, the majority of which are still in academic medicine. He is a member of the Institute of Medicine of the National Academy of Sciences. He has published over 230 peer-reviewed manuscripts, over 100 invited reviews and multiple textbook chapters. He formerly served on the NIH Recombinant DNA Advisory Committee and Gene Therapy Safety Assessment Board. He is actively involved in gene therapy trials for blood, immunodeficiency and neurological genetic diseases and has been the investigator, co- investigator or sponsor (IND holder) of four previous gene therapy trials and is sponsor, investigator or co-investigator of four current trials. He has recently served as a counselor for the American Society of Hematology (ASH) and served on the Joint Oversight Committee of ASH/European Hematology Association Translational Research Training in Hematology. He is currently the president of ASH. He served as the Editor-In-Chief of Molecular Therapy from 2004-2009. He is co-founder of the Transatlantic Gene Therapy Consortium and the North American Pediatric Aplastic Anemia Consortium. His basic research has focused on

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hematopoietic stem cell biology, including genetic diseases of the blood and specifically molecular and biochemical analysis of the interaction between hematopoietic stem cells and the bone marrow supporting environment. He has multiple issued patents, several of which have been licensed including IL-11 (NeumegaTM) and the use of fibronectin in gene transfer (RetronectinTM).

Dr. Guangping Gao Professor, University of Massachusetts Medical School

Dr. Gao received his Bachelor Degree in Medicine from the West China Medical School of Sichuan University in 1982. He completed his Ph.D. training in molecular genetics at Florida International University in 1994 with his work involving the isolation and characterization of the human aspartoacylase gene and the genetic mutations responsible for Canavan disease, a server form of inherited neurodegenerative diseases. Dr. Gao joined the University of Pennsylvania (UPenn) in 1994 where he has developed his career in viral vector biology for gene therapy. Dr. Gao made major contributions to the development of the 3rd generation adenovirus vectors and in 1996 was appointed as the Assistant Director of the Institute for Human Gene Therapy (IHGT) at UPenn with the primary responsibility of clinical adenovirus vector production. He was promoted to Associate Director and then to the Director of Vector Program of IHGT to oversee the vector discovery and development, process development, and vector core and quality control testing in 1998 and 2001 respectively. He was the driving force behind the discovery and vectorology of a novel primate adeno-associated virus (AAV) family and the development of simian adenovirus vector based vaccine programs. Dr. Gao joined the University of Massachusetts Medical School (UMMS) in April 2008 as the Founding Director of the Gene Therapy Center and Vector Core, the Scientific Director of the UMMS-China Translational Research Initiative (which was later renamed UMMS-China Program), and Professor of Microbiology and Physiology Systems. In September 2010, he was officially named the Penelope Booth Rockwell Professor in Biomedical Research. Dr. Gao’s primary research interests include molecular mechanisms of AAV evolution and diversity, molecular interactions between endogenous AAV, vector and host genomes, molecular and cellular mechanisms of AAV vector transduction, microRNA functional in adult mammals, biology and clinical application of extracellular miRNAs, and gene therapy of CNS disorders using rAAV to cross BBB for global CNS gene transfer. Dr. Gao has published more than 160 papers in peer-reviewed journals and 30 patented inventions. He is frequently invited to speak at international gene therapy conferences. In two consecutive months, October and November of 2003, one of his PNAS papers on novel AAV vector discovery was selected by ISI as among the most cited papers (top 1%) and hottest break-through papers (top 0.1%). Dr. Gao is well recognized in the field of viral vector-based gene therapy. Dr. Gao served on international committees such as the membership Committee and International Committee, viral vector committee of American Society of Gene and Cell Therapy (ASGCT), the AAV Vector Manufacturing and Reference Standard Committees. Dr. Gao is the senior editor of the Book Series on Gene and Cell Therapy publishing by ASGCT and Springer Publisher, He serves on Advisory Board of Advances in Experimental Medicine and Biology. He also serves on the board of directors of ASGCT, the editorial boards of Human Gene Therapy and Gene Therapy,

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Molecular and Cellular Therapy, Journal of Neurological Disorders, and many study sections and steering committees for NIH and other national and international research organizations. He is also a scientific founder of Voyager Therapeutics, a Cambridge (MA, USA) based biopharmaceutical company that focuses on developing rAAV gene therapeutics for treating a variety of devastating CNS disorders.

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2015 Harvard Chinese Life Science Distinguished Research Award Recipients

Name Title Affiliation

Dana-Farber Cancer Institute, Harvard Bo Duan Postdoctoral Fellow Medical School Massachusetts General Hospital, Harvard Changning Wang Postdoctoral Fellow Medical School Brigham and Women’s Hospital, Harvard Chuan Wu Instructor Medical School Boston Children's Hospital, Harvard Medical Feilong Meng Postdoctoral Fellow School Boston Children's Hospital, Harvard Medical Hao Wu Postdoctoral Fellow School

Jianlong Sun Postdoctoral Fellow Boston Children’s Hospital, Harvard University

Jing Liao Postdoctoral Fellow Harvard University

Boston Children’s Hospital, Harvard Medical Lei Gu Postdoctoral Fellow School Boston Children's Hospital, Harvard Medical Li Shen Postdoctoral Fellow School Dana-Farber Cancer Institute, Harvard Song Yi Postdoctoral Fellow Medical School

Sui Wang Postdoctoral Fellow Harvard Medical School

Ting Xie Graduate Student Harvard Medical School

Wen Chen Postdoctoral Fellow Harvard Medical School

Massachusetts General Hospital, Harvard Ying-Hua Wang Instructor Medical School Boston Children's Hospital, Harvard Medical Yuting Liu Postdoctoral Fellow School Boston Children’s Hospital, Harvard Medical Zhiqiang Lin Instructor School

Yongyue Wei Postdoctoral Fellow Harvard School of Public Health

Yuan Lu Postdoctoral Fellow Harvard School of Public Health

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Sponsors

Biocytogen http://www.biocytogen.com Developing Novel Gene-Targeted Models to Advance Life Science Research

Life Technologies, a Thermal Fisher Scientific Brand www.lifetechnologies.com Integrated Systems for genetic Analysis

Beijing Genomics Institute (BGI) http://www.genomics.cn/en/index BGI, founded in 1999 with the vision of using genomics to benefit the human race, is now the world’s largest genomics organization

Hikewell Gene (Beijing) Institute http://www.s-ama.org/index.html Technology platform to solve life password and improve health quality

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