Gene TargetingGene Correction

Clinical TranslationStem Cell Therapies

CAR-TCR-T Cell Therapy

Cell and Gene TherapyBreakthrough Science and Technology in CGT

PhiladelphiaPharmaceuticalSymposium - 2017

DECEMBER 2, 2017 | RADNOR VALLEY COUNTRY CLUB | VILLANOVA, PA

Featured Sponsor Sponsors

Symposium Theme

The goals for SAPA-GP’s Philadelphia Pharmaceutical Symposium include highlighting cutting-edge

scientific breakthroughs, fostering collaboration and the exchange of scientific ideas among pharma-

ceutical and academic professionals. This year’s symposium will focus on cell and gene therapy. We

are privileged to present a panel of esteemed researchers from both academia and industry to show-

case the recent breakthroughs in cell and gene therapy.

More details on this symposium are available on SAPA-GP’s website (http://sapa-gp.org/sapagp/)

Keywords: Stem Cell Therapies; Gene Targeting and Gene Correction; CAR-TCR-T Cell Therapy;

Clinical Translation of Gene and Cell Therapies; Immunological Aspects of Gene and Cell Therapy

SAPA-GP History

The Greater Philadelphia (GP) area is one of the major homes for the world pharmaceutical industry. It

hosts more than half of the world’s top-ten pharmaceutical companies, and many mid/small pharma-

ceutical/biotech companies as well as academic institutions. The Sino-American Pharmaceutical Pro-

fessionals Association - Greater Philadelphia (SAPA-GP) was established in 2002 to serve the rapidly

growing pharmaceutical/biotech/healthcare community in the GP area.

SAPA-GP Mission

To promote pharmaceutical sciences and biotechnology

To contribute to public health education by raising public awareness

To facilitate scientific and business cooperation between US and China

To foster career development of pharmaceutical professionals

AGENDA

8:00-8:55 a.m.

8:55-9:00 a.m.

9:00-9:10 a.m.

9:10-9:15 a.m.

9:15-9:45 a.m.

9:45-10:15 a.m.

10:15-10:30 a.m.

10:30-10:55 a.m.

10:55-11:20 a.m.

11:20-11:45 a.m.

11:45-1:00 p.m.

1:00-1:30 p.m.

1:30-1:55 p.m.

1:55-2:20 p.m.

2:20-2:35 p.m.

2:35-3:00 p.m.

3:00-3:25 p.m.

3:25-3:50 p.m.

3:50-4:15 p.m.

4:15-4:20 p.m.

MORNING

AFTERNOON

Registration

Opening Remarks, Haifeng Cui, Ph.D., GSK Fellow, Scientific Director, GlaxoSmithKline

Introduction of SAPA-GP, Jing Yang, Ph.D., Senior Principal Scientist, Bristol-Myers

Squibb Company

Special Remarks, Christopher P. Molineaux, President, CEO, Life Science PA

Federico Mingozzi, Ph.D. Developing Gene Therapies for Rare Diseases, a Histo-

ry of Synergy Between Academia and Industry

Joseph Tarnowski, Ph.D. Progress in the Delivery of Ex Vivo Cell and Gene Ther-

apy Products

Coffee Break

Wyle Solomon, M.B.A. Novel CAR-T Targets & Technologies: Capital Efficient

Strategies to Clinical Proof of Concept

Shell Li, M.D., M.S. A Phase I Study to Evaluate the Safety of Multi-Antigen Stim-

ulated Cell Therapy-I in Patients with Advanced Solid Tumors

Jeffrey Hung, Ph.D., M.B.A. Solve the Supply Problem of Gene Therapy - AAV

Manufacturing Technology Overview

Lunch Break

Rafael Amado, M.D. T-cell Receptor Engineered T-cell as Cancer Therapeutics

Gong Chen, Ph.D. Brain Repair with In Situ Glia-to-Neuron Conversion

Mike Naso, Ph.D. New Therapeutic Platforms: More Platform than You Think

Coffee Break

Laura Johnson, Ph.D. Gene-Engineered T-cell Therapy – Bench to Bedside and

Back Again

Toby Ferguson, M.D., Ph.D. Clinical Experience with ASOs in Neuromuscular

Disease

Glenn Cowley, Ph.D. CRISPR/CAS9: Modification & Modulation of Genes

Yangbing Zhao, M.D., Ph.D. Using CRISPR/CAS9 to Generate Universal T Cells

and Improve Adoptive Immunotherapy against Solid Tumors

Closing Remarks, Hao Sun, M.S., Senior Scientist, GlaxoSmithKline

Session 1 Chair: Wenyong Wang, Ph.D., Vice President, Science and Technology, University City Science Center

Session 2 Chair: Hao Sun, M.S., Senior Scientist, GlaxoSmithKline

Session 3 Chair: Li Yan, Ph.D., Vice President, Head Unit Physician, Oncology, GlaxoSmithKline

Session 4 Chair: Haifeng Cui, Ph.D., GSK Fellow, Scientific Director, GlaxoSmithKline

SPEAKERS

Rafael Amado, M.D., Chief Medical Officer, Adaptimmune

Dr. Amado has served as Chief Medical Officer of Adaptimmune since March 2015 and has 15 years of experience within the biotech and pharma industries. Dr. Amado leads clinical strategy and is responsible for the execution of clinical development program globally. He formerly served as Senior Vice President and Head of Oncology R&D at GSK, where he was responsible for integrating oncology R&D activities, from drug target identification to clinical development and registration globally.

Dr. Amado joined GSK in 2008 as Vice President of Clinical Development, and served in positions of increas-ing responsibility, including Senior Vice President and Head of Oncology Clinical Development. He oversaw the development and registration globally of over 15 indications across six products and led the develop-ment of a pipeline of products in novel areas of cancer biology such as signal transduction, epigenetics and cancer immunology. Prior to joining GSK, Dr. Amado was Executive Director of Therapeutic Oncology at Amgen from 2003 to 2008 where he was responsible for development activities of several assets. Dr. Ama-do trained as a Hematologist/Oncologist at the University of California, Los Angeles, where he remained as faculty for eight years until joining Amgen in 2003. He holds an M.D. from the University of Seville School of Medicine in Spain, and performed his residency in Internal Medicine at Michael Reese Hospital, a Univer-sity of Chicago Affiliated Hospital, and his fellowship in Hematology/Oncology at the University of Califor-nia, Los Angeles.

Gong Chen, Ph.D., Professor, Penn State University, Verne M. Willaman Chair in Life

Sciences

Dr. Chen is Willaman Chair Professor at Penn State University. Dr. Chen pioneered an innova-tive in situ cell conversion technology for brain and spinal cord repair. Unlike traditional stem cell transplantation therapy, Dr. Chen and his team make use of brain internal glial cells that normally surround neurons to regenerate new neurons in situ. Because glial cells are residen-

tial cells throughout the brain and spinal cord, Dr. Chen’s technology opens a new field that can essentially treat a wide range of neurological disorders, including traumatic brain injury, stroke, Alzheimer’s disease, Parkinson’s disease, spinal cord injury, and ALS, through gene therapy or drug therapy. Chen’s break-through findings have been widely reported in the news media, kindling new hope for millions of patients around the world. To translate his new technology from bench to the bedside, Dr. Chen and team members have filed and obtained a series of patent applications. Dr. Chen is a founder of NeuExcell Therapeutics Inc., a company aiming to lead a revolution in brain and spinal cord repair by transforming his cutting-edge tech-nologies into therapeutic treatments to save millions of lives. Professor Chen is a member of many professional societies, and honored by the Alzheimer’s Association with Zenith Fellows Award. Chen has published many scientific papers in high-impact journals such as Cell Stem Cell, Nature Communications, Nature, Cell, PNAS, and received funding from NIH, NSF, AHA, etc. Chen has presented invited talks and keynote speeches at many international conferences and leading insti-tutions. Dr. Chen organized and Chaired the first symposium in history on in vivo cell conversion in the CNS at the 2014 annual meeting of Society for Neuroscience in Washington DC. This symposium is a milestone marking a new field of in vivo cell conversion in regenerative medicine.

Toby Ferguson, M.D., Ph.D., Medical Director, Neuromuscular Disease, Biogen

Dr. Toby Ferguson is Medical Director of early clinical neuromuscular development , a position he assumed in 2015 shortly after joining Biogen in October 2013. In this role, he serves as a clinical lead for current clinical ALS programs. He has also worked closely with neurology research and colleagues in ALS development to develop a strategy for development of ALS therapies and to improve ALS trial effectiveness. Prior to Biogen, he was the Assistant Professor of Neurology at Temple University in Philadelphia, PA, and had a clinical neuromuscular neurology practice and a

lab focused on peripheral axon injury and regeneration at Shriners Research Center. Dr. Ferguson obtained an M.D. and Ph.D. (Neuroscience) from the University of Florida. He went on to complete his Neurology Residency in 2006 and his Fellowship in Neuromuscular Neurology in 2010 at the University of Pennsylvania.

Jeffrey Hung, Ph.D., M.B.A., Chief Commercial Officer, Vigene Biosciences

Jeffrey Hung, Ph.D., is Chief Commercial Officer of Vigene Biosciences, a leading gene delivery company whose mission is to make gene therapy affordable. As the General Manager of GMP Business, Dr. Hung orchestrated Vigene Biosciences’s acquisition of Omnia Biologics in 2016, a CMO specializing on GMP manufacturing of viruses. Dr. Hung served as the Vice President of GenScript and was instrumental in growing the compa-ny before the IPO in December 2015. Dr. Hung was the Chief Marketing Officer at ATCC before

joining GenScript. Dr. Hung is the author of multiple patents, per reviewed publications and book chapters. Dr. Hung earned his Ph.D. from Cornell University, M.B.A. from UC Berkeley, and B.S. from Peking University.

Glenn Cowley, Ph.D., Senior Principal Scientist, Head of Functional Genomics, Discovery

Sciences, Janssen Pharmaceuticals

B.A. in Biology Brandeis University, Waltham MA (1988)Ph.D. in Genetics from Harvard Medical School, Boston, Massachusetts (2000)Postdoctoral fellow, Abbott Bioresearch Center, Worcester, Massachusetts (2000-2003)Scientist, Abbott Bioresearch Center, Worcester, Massachusetts.

Developed internal capability for arrayed RNAi screening technologies (2003-2007)Senior Group Leader, The Broad Institute, Cambridge Massachusetts (2007-2016)Design, implement, and analyze shRNA and CRISPR/Cas9 pooled genome scale screens in oncology, immunolo-gy, and infectious disease areas in collaboration with the Harvard and MIT laboratoriesProject Manager for Project Achilles, massively parallel genome-wide shRNA and CRISPR/Cas9 synthetic lethality screening in >700 cancer cell linesSenior Principal Scientist, Head of Functional Genomics, Janssen (2016-present)Lead Functional Genomics group in Spring House, PA and Beerse, Belgium. Manage in-house expertise and ex-ternal collaborations for functional genetic screening and gene editing/engineering (CRISPR-Cas9, RNAi) tech-nology to support cross-TA studies in support of new target identification and validation, biomarker support.

SPEAKERS (continued)

Shell Li, M.D., M.S., Global Chief Medical Officer, Vice President, Global Clinical Devel-

opment, HRYZ Biotech Co.

Shell Li, M.D. M.S.,HRYZ(恒瑞源正)Global Chief Medical Officer and VP, Global Clinical Devel-opment. Shell has over 25 years of Pharm/Biotech companies (Merck, Amgen, Novartis, and Boehringer Ingelheim), particularly, over 20 years of Clinical Development experiences in new drug/biologics development in both US and in China. Over the years, her responsibilities was

increased from Clinical Project Manager, Medical Director to Development Head in charging over 100 active clinical studies cross all therapeutical areas. While she was in Amgen as the Medical Director, she conducted multiple early phase oncology clinical studies and successfully delivered those products for later phase clinical development. She was also responsible for Sorafenib’s life cycle management and in charge of multiple indi-cation expansions. In 2011, she left Amgen/Onyx and returned to China for Novartis as the Head of Develop-ment in Vaccine Division, later for Boehringer Ingelheim as Head of Clinical Research and Operation, Greater China in charging all the clinical studies conducted in China and other countries. In this role, Shell was respon-sible for Afatinib phase III registration study in Asia, later led to approval of Afatinib in US and in China. She also actively participated FDA Advisory Committee Meeting preparation and participated the meeting in person to gain first handed experiences. Since 2015, Shell Joined HRYZ (恒瑞源正) as the Global Chief Medical Officer to be responsible for company’s clinical development.

Laura Johnson, Ph.D., Senior Director, Head Translational Medicine, Oncology Cell Thera-

py, GlaxoSmithKline

After completing a Ph.D. in molecular and cellular immunology in Canada at the University of British Columbia, Dr. Laura Johnson did her postdoctoral fellowship with Dr. Steven Rosenberg at the US National Cancer Institute. During that time, she was involved in some of the first T-cell receptor gene-engineered clinical trials for patients with metastatic melanoma, targeting MART-

1 and gp100 antigens. Following her postdoc, she joined the faculty of Neurosurgery at Duke University and her laboratory conducted preclinical research in syngeneic and xenogeneic mouse models of glioblastoma (GBM). In 2012, Dr. Johnson moved to Philadelphia to work with Dr. Carl June at the University of Penn-sylvania, where she specialized in translational research of gene-engineered T cell therapy of solid tumors, including targeting MUC1-Tn and translating EGFRvIII-redirected chimeric antigen receptor (CAR) T cells from the bench into clinical trials for patients with GBM at UPenn and UCSF. Dr. Johnson joined GSK in early 2017 to head up the Translational Medicine division of their Oncology Cell Therapy unit and is actively working to bring gene-engineered T-cell therapies for cancer into a global treatment, widely available to change lives for

Federico Mingozzi, Ph.D., Chief Scientific Officer, Spark Therapeutics

Dr. Federico Mingozzi is the chief scientific officer at Spark Therapeutics. He began his scientific career studying the genetic bases of bleeding disorders. At the Children’s Hospital Philadelphia (CHOP) he conducted pioneering studies on liver gene transfer with adeno-associated virus (AAV) vectors and immunology. He was involved in several first-in-human clinical studies of gene therapy based on the AAV vector platform while serving as the director of translational research the Center for Cellular and Molecular Therapeutics, at CHOP. He also led studies aimed at the

characterization of human immune responses to AAV vectors and the development of strategies to modulate vector immunogenicity. He then joined the French National Institute of Health and Medical Research (INSERM) as Research Director and Genethon, a leading French nonprofit R&D organization focused on gene therapy for rare diseases, as Team Leader. There, he spearheaded the development of in vivo gene therapies for inherited diseases. He currently serves as faculty at the Pierre and Marie Curie University in Paris, France, and Universitat Autonoma de Barcelona, Spain. He received his bachelor’s degree in biology and his Ph.D. in biochemistry and molecular biology from the University of Ferrara in Italy, and his M.B.A. from Drexel University.

Mike Naso, Ph.D., Scientific Director, Gene and Gene Modified Cell Therapy, Janssen Pharmaceuticals

Michael Naso, Ph.D., is a Scientific Director at Janssen Research and Development of Johnson and Johnson. He received his B.S. in chemistry from Dickinson College in Carlisle PA, and his M.S. in Pathology and Cell Biology and Ph.D. in Biochemistry and Molecular Biology from Thomas Jef-ferson University in Philadelphia, PA. His work at TJU focused on the study of the structure and function of extracellular matrix genes in cancer and blistering skin diseases. He then joined Cento-

cor, and worked on the engineering and expression of antibodies and antigens for drug discovery. Previously, Michael led the Molecular Biology and Target Discovery group that focuses on the discovery and validation of antibody drug targets and the development of antibody based therapeutics for those targets. Currently, he leads the Gene and Cell Therapy Discovery group within the Biologics Discovery department of Janssen Bio-therapeutics. His team has been driving rAAV-based gene therapy efforts internally and with external partners, and has developed a potentially disruptive allogeneic engineered T cell platform for oncology and immunology indications. Michael has been with Janssen for 16 years, and has published in the field of antibody structure and function, protein engineering, novel target ID technologies and gene and gene modified cell therapy.

Wyle Solomon, M.B.A., Chief Executive Officer, iCell Gene Therapeutics

Wyle has served as Chief Executive Officer of iCell Gene Therapeutics since June, 2016. He was previously Venture Partner at Nordic Biotech ApS, a Danish Venture Capital Firm. He served as President, CEO and/or Founder of several companies and Executive Chairman of Forward Pharma A/S (Denmark, Nasdaq: FWP) and Esplanade Pharma Group Limited (Hong Kong); He was Director, Commercial and Business Development at Mylan; Head of regulatory affairs and pharmaceutical development at Achillion Pharmaceuticals; and held positions of increasing responsibility at Bris-

tol-Myers Squibb as a scientist in oncology drug discovery and in regulatory affairs. Wyle holds an M.B.A. from McGill University School of Management, an M.Sc. from Temple University School of Pharmacy and a B.S. in biology from the University of Connecticut.

Yangbing Zhao, M.D., Ph.D., Director, T Cell Engineering Lab, Adjunct Associate Profes-

sor, University of Pennsylvania

Dr. Yangbing Zhao is the Director of the T Cell Engineering Laboratory (TCEL), Center for Cellular Immunotherapies at the University of Pennsylvania. He received an M.D. and Ph.D. in Immunolo-gy from the Third Medical University in Chongqing, China. From 1996 – 2000, he was a visiting scientist at The Weizmann Institute of Science in Rehovot, Israel, where he studied tolerance induction through genetically engineered T cells for allogeneic bone marrow transplantation. For

the next two years he was a research associate at Duke University Medical Center, where he worked on RNA transfected dendritic cell vaccines against tumors. In 2003, he joined Dr. Steven Rosenberg’s group as a senior research fellow at the Surgery Branch of the National Cancer Institute, where his research work directly led to clinical trials of treating cancer patients with TCR or CAR engineered T cells against NY-ESO-1, Her2/Neu, CD19 or VEGFR. He joined the faculty of the Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine in 2009 as the Director of TCEL. His Laboratory’s research and development work has led to multiple clinical trials for the treatment of solid tumors with genetically engineered T cells against mesothelioma, pancreatic cancer, breast cancer and glioblastoma.

Joseph Tarnowski, Ph.D., Senior Vice President, Cell and Gene Therapy, Platform Tech-

nology&Science, R&D, GlaxoSmithKline

Joseph Tarnowski is Senior Vice President of Cell and Gene Therapy Platforms, PTS, R&D at GlaxoSmithKline (GSK). Before joining GSK in June, 2010, Joe was the Senior Vice President, Biologics Manufacturing and Process Development in the Technical Operations division of Bris-tol-Myers Squibb Co. in New Brunswick, NJ. Joe was responsible for building the manufacturing

capabilities needed to supply the company’s biologic medicines to worldwide markets, including the con-struction of the company’s new $750 million large-scale multi-product bulk biologics manufacturing facility in Devens, Massachusetts, and the integration of Medarex, Inc. He has spent over 35 years in the pharmaceutical industry focused primarily on the research, development, registration and commercialization of biopharmaceu-tical products made using recombinant DNA technology. He holds 13 patents and has several patent applica-tions pending for biologic products.Joe has held critical roles in the manufacturing, process development, registration, and launch of several FDA and internationally approved human therapeutic products, including Roferon® A, Alpheron N®, Fiblast® Spray, CEPRATE® SC Stem Cell Concentration System, Natrecor®, Erbitux® Orencia®, Yervoy®, Nulojix®, Eperzan®/Tanzeum®, Nucala®, and Strimvelis™. He has a thorough understanding of the biologics drug reg-istration process and has had extensive experience in developing the Chemistry, Manufacturing and Controls (CMC) sections for many Investigational New Drug and Biologics License Applications.

SPEAKERS (continued)

ACKNOWLEGEMENTS Organizing Committee MembersYuchen Bai

Rong Fan

Laura Johnson

Huayu Liu

Heather Steinman

Wenyong Wang

Zhenhua Wu

David Cragin

Meizhen Feng

Xianhua Li

Xin’an Liu

Yongchao Su

Yanjie Wang

Li Yan

Haifeng Cui

Dennis Gross

Yangzhou Li

Fang Shen

Hao Sun

Zhiyun Wen

Jing Yang

Han Dai

Pengbo Guo

Yin Liang

Bin Shi

Aimin Tang

Di Wu

Hanghang Zhang

Patrick Deng

Harris Huang

Aston Liu

Mengjie Si

Hui Wang

Hao Wu

Program DirectorsHaifeng Cui Hao Sun

LUYE Pharma Group Ltd. is a professional pharmaceutical company based on R&D. The company was founded in 1994 and listed on the main board of the Hong Kong Stock Exchange in 2014. The company has state-of-the art production facilities and R&D centers located in China, the USA AND Europe, as well as overseas branch offices in Hong Kong, Singapore and Malaysia with approximately 4,000 employees. LUYE Pharma focus on developing, producing, marketing and selling innovative pharmaceutical products in four of the largest and fastest growing therapeutic areas – oncology, cardiovascular, metabolism and the cen-tral nervous system. LUYE Pharma has established a professional R&D system of international standard, and reached international level in the area of new drug delivery system technologies such as microspheres, liposome and TDS. Currently, LUYE Pharma has multiple innovative preparations and drugs that are undergoing different stages of registry clinical studies in the USA. With the vision of to be one of the most respected global leaders in the healthcare industry, LUYE Boston R&D Center was found July 16th, 2017. The mission of the biology department is to discover innovative next gen-eration biologic therapeutics by the means of the combinational cutting-edge technologies and to improve the quality of patients’ life. The department of “Novel Drug Delivery Technologies” focuses on looking for the cut-ting-edge intelligent drug delivery systems which provide better and more sophisticated treatments with existing pharmaceutical activities. With the strong supports of R&D, LUYE Pharma will keep adhering to the road of innovation and international-ization, and endeavor to become one of the top 100 global pharmaceutical companies by 2020.

Art Design and Brochure EditingPengbo Guo Mengjie Si Di Wu

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