BRM2018 Speakers / Session Chairs
Spark Therapeutics, Inc.
Katherine A. High, M.D.
President and Head of R&D
Dr. Katherine High is currently President and Head of R&D at Spark Therapeutics, a biotech company that she co-founded in 2013. Under Dr. High’s leadership, Spark received FDA approval of the first AAV gene therapy product in the US, a treatment for a rare form of congenital blindness. Dr. High was formerly a Professor at the Perelman School of Medicine at the University of Pennsylvania, an Investigator of the Howard Hughes Medical Institute, and the Founding Director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. An elected member of the National Academy of Medicine and the American Academy of Arts and Sciences, she has published over 200 scientific papers, and holds a number of patents related to gene therapy.
Food and Drug Administration Center for Biologics Evaluation and Research
Director, Office of Tissues and Advanced Therapies
Wilson Bryan is a neurologist who graduated from the University of Chicago Pritzker School of Medicine. Dr. Bryan is board certified in neurology and neuromuscular medicine. He served on the Neurology faculty of the University of Texas Southwestern Medical School for 13 years. He has been an investigator on clinical trials in cerebrovascular disease and neuromuscular disorders, particularly amyotrophic lateral sclerosis. Dr. Bryan joined the United States Food and Drug Administration (FDA) in 2000, and now serves as Director of the Office of Tissues and Advanced Therapies (OTAT) in the FDA’s Center for Biologics Evaluation and Research.
U.S. Food and Drug Administration
Peter Marks, M.D., Ph.D.
Director, Center for Biologics Evaluation and Research
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for the Center for Biologics Evaluation and Research and became Center Director in January 2016.
Ainslie Little, Ph.D.
Director, Intellectual Property
Ainslie Little has focused on patent strategy and protection of stem cell technologies for almost a decade. At BlueRock Therapeutics, Ainslie is responsible for developing and executing intellectual property strategies, building and managing global patent and trademark portfolios, conducting due diligence to support business objectives, and developing and implementing IP policies and education programs.Before joining BlueRock Therapeutics, Ainslie practiced at leading law firms in Canada and the United States where she focused on drafting and prosecuting patent applications and advising universities, SMEs, and multinational pharmaceutical companies on patentability, validity, infringement and freedom-to-operate matters. Ainslie is a registered patent agent in the United States and Canada. She holds a B.Sc. from the University of Guelph and a Ph.D. in microbiology from the University of Wisconsin-Madison.
Division of Pediatric Stem Transplantation and Regenerative Medicine Stanford Health Care/ Stanford School of Medicine
David DiGiusto PhD
Executive Director - Stem Cells and Cellular Therapeutics Operations Founding Director – Stanford Laboratory for Cell and Gene Medicine Senior Research Scientist –
Dr. David DiGiusto is currently the Chief Technical Officer for Semma Therapeutics. He has over 25 years of experience in the scientific, clinical and regulatory aspects of cells as therapeutic agents including the isolation, characterization and genetic modification of hematopoietic stem cells and T-cells for clinical applications. He has been instrumental in the creation of 6 GMP compliant biologics manufacturing facilities and associated quality systems, production and QC testing programs. Under his direction, plasmid DNA, CAR-T-cells, regulatory T-cells, engineered stem cell grafts and gene modified hematopoietic stem cell products have been manufactured and released for use in Phase I/II clinical trials. Dr DiGiusto is a major contributor to first in human studies for Cancer and HIV Gene Therapy and has developed methods for assessing ex-vivo stem cell manipulations using in vitro and in vivo models. Dr. DiGiusto is a member of the NIH recombinant DNA advisory committee (RAC).
University of Pennsylvania
Gerald P. Linette, MD, PhD
Medical Director, Sean Parker Institute of Cancer Immunotherapy
Gerald P. Linette, MD, PhD is a graduate of the Medical Scientist Training Program, Georgetown University School of Medicine. He completed training in Internal Medicine and Molecular Oncology at Washington University in St. Louis followed by fellowship in Hematology-Oncology at Massachusetts General Hospital/DFCI. Dr. Linette is currently Professor of Medicine and Medical Director, Sean Parker Institute of Cancer Immunotherapy at the Perelman School of Medicine, University of Pennsylvania. His primary interest is the development of cellular immunotherapies for melanoma and other solid tumors. His laboratory research is focused on human tumor neoantigen discovery. Dr. Linette is board certified in Internal Medicine and Medical Oncology and he maintains an active clinical practice at the University of Pennsylvania Abramson Cancer Center.
Tmunity Therapeutics, Inc.
Usman “Oz” Azam, MD
Dr. Azam was appointed President CEO of Tmunity Therapeutics in Dec 2016. Most recently Dr Azam was the Head of the Cell & Gene Therapies Unit at Novartis Pharmaceuticals and was a member of the Pharmaceutical Executive Committee. He has an extensive U.S., Global and International pharmaceutical industry background, with strong experiences in science, clinical development, regulatory and commercial activities. While at Novartis, he lived and worked in the USA and Switzerland. Previous roles at Novartis included Head of Global Medical Affairs & HEOR General Medicines, Chief Scientific Officer and Head of US Clinical Development & Medical Affairs and Global Head Regulatory Affairs for the Neurosciences & Ophthalmic Franchise. Prior to Novartis he was the Chief Executive Officer of Novaccel Therapeutics LLC, a company he founded focusing on accelerating assets in the translational medicine setting. Prior to this he served as Chief Medical Officer at Aspreva Pharmaceutics Inc, a company that focused on orphan and rare autoimmune disease states. Aspreva was acquired by Galenica SA in 2008.Prior to this he worked at Johnson & Johnson in Pharmaceutical Research & Development and in Medical Devices at Johnson & Johnson Ethicon. He started his life-sciences career with Warner Lambert-Parke Davis in 1998. Dr Azam is a graduate of the University of Liverpool, United Kingdom, where he completed undergraduate studies in Human Biology & Anatomy. He graduated from the University of Liverpool School of Medicine & attained board certification as an Obstetrician & Gynecologist in the U.K
President and CEO of AVROBIO
Geoff MacKay is President and CEO of AVROBIO, Inc, a clinical stage cell and gene therapy company based in Cambridge MA. The company focus is in rare disease and immune-oncology. Geoff was founding CEO of eGenesis Inc., which applies CRISPR-Cas 9 gene editing to the field of xenotransplantation. Mr. MacKay spent 11 years as President and Chief Executive Officer at Organogenesis Inc., building the company into the leading regenerative medicine company in the world as measured by FDA approvals, patients treated and revenue. Mr. MacKay held numerous leadership positions within Novartis AG, such as Vice-President and Business Unit Head, Transplantation and Immunology at Novartis Canada, Vice-President of Tissue-Engineering in Novartis USA, Head of Global Sales Immunology and Transplantation based in Basel, Switzerland, and Sales and Marketing Manager of Novartis Biotech Europe. Mr. MacKay has served as Chairman of the Board of the Alliance for Regenerative Medicine (ARM), a Washington DC-based multi-stakeholder advocacy group whose mission is to advance the field of regenerative medicine. ARM’s 200 members include companies, academic research institutions, patient advocacy groups, foundations, health insurers and investors dedicated to improving patients’ lives through regenerative medicine therapies. Additionally, he is an Advisory Council member for the Health Policy Commission (HPC) for the Commonwealth of Massachusetts, a state agency that monitors the reform of the healthcare delivery and payment systems in Massachusetts, and develops health policy to reduce overall cost growth while improving the quality of patient care.
Bruder Consulting, Venture Group
Scott Bruder, MD, PhD
Founder and CEO
Scott P. Bruder, MD, PhD, is an insightful and energetic healthcare leader with a 25-year history of bridging basic science, clinical medicine, and industrial development expertise to deliver innovative, commercially successful products around the world. Experience in medical devices, diagnostics, biotechnology, and life science research tools fortify an expansive analytical skill set for this resilient, poised and influential C-Suite executive. An award-winning scientist and clinician, equally comfortable in the laboratory, in the Boardroom or on Capitol Hill, he built the "Bruder Consulting & Venture Group" to deliver impactful results by inspiring teams to be collaborative, rigorous and decisive. This seasoned Executive, University Professor, and FDA Advisory Committee Member provides a unique bench-to-bedside perspective on unmet needs, development strategy and the path to commercialization.
Georgia Institute of Technology
Andrés J. García, Ph.D.
Rae S. and Frank H. Neely Chair and Regents’ Professor
Dr. García’s research program integrates innovative engineering, materials science, and cell biology concepts and technologies to create cell-instructive biomaterials for regenerative medicine and generate new knowledge in mechanobiology. This cross-disciplinary effort has resulted in new biomaterial platforms that elicit targeted tissue repair in various applications, innovative technologies to study and exploit cell adhesive interactions, and new insights into the interplay of mechanics and cell biology. He has received several distinctions, including the NSF CAREER Award, Arthritis Investigator Award, Young Investigator Award from the Society for Biomaterials, and the Clemson Award for Basic Science from the Society for Biomaterials. He is an elected Fellow of Biomaterials Science and Engineering, Fellow of the American Association for the Advancement of Science, Fellow of the American Society of Mechanical Engineers, and Fellow of the American Institute for Medical and Biological Engineering.
Raymond James & Associates, Inc.
Reni J. Benjamin, Ph.D.
Managing Director, Biotechnology Equity Research
Dr. Reni Benjamin is a Managing Director and Senior Biotechnology Analyst at Raymond James. His expertise and coverage consists of companies in the oncology and stem cell sectors. Dr. Benjamin has previously been ranked among the top analysts for recommendation performance and earnings accuracy by StarMine, been cited in a variety of sources including the Wall Street Journal, Business Week, Financial Times, and Smart Money, and has made appearances on Bloomberg television/radio and CNBC. He has also authored a chapter in a book entitled “The Delivery of Regenerative Medicine and Their Impact on Healthcare”, a past member of the UAB School of Health Professions Deans Advisory Board, and presently serves on the Advisory Board for Phacilitate Cell & Gene Therapy. Prior to joining Raymond James, Dr. Benjamin was a Managing Director and Senior Biotechnology Analyst at H.C. Wainwright, Burrill Securities and Rodman & Renshaw. He earned his doctorate in Biochemistry and Molecular Genetics and his expertise includes biochemistry, functional genomics, gene therapy and molecular biology. Dr. Benjamin has presented at various regional and international conferences and published in peer-reviewed journals. Dr. Benjamin received his Ph.D. from the University of Alabama at Birmingham and his Bachelors of Science degree in Biology from Allegheny College.
University of Pennslyvania
D. Dan Huh
Wilf Family Term Assistant Professor
Dan Huh is an Assistant Professor and Wilf Family Term Endowed Chair in the Department of Bioengineering at the University of Pennsylvania. He is a pioneer of “organ-on-a-chip” technology, and his research group at Penn focuses on developing microengineered models of human organs in health and disease for a wide variety of biomedical applications. Dr. Huh has won several honors and awards including the CRI Technology Impact Award, the John J. Ryan Medal from the Royal College of Surgeons in Ireland, Design of the Year Award from London Design Museum, NIH Director’s New Innovator Award, Analytical Chemistry Young Innovator Award, TEDx Fellow, NC3Rs Annual Award, Lifetime Membership from the MOMA, SLAS Innovation Award from the Society for Lab Automation and Screening, Scientific Breakthrough of the Year from American Thoracic Society, Best Publication Award and Best Postdoctoral Award from the Society of Toxicology, Wyss Technology Development Fellowship from Harvard, Distinguished Achievement Award from Michigan, Widmer Award from microTAS, and Horace H. Rackham Predoctoral Fellowship.
Brigham and Women's Hospital, Harvard Medical School
Professor of Medicine
Jeff Karp is a professor of Medicine at Brigham and Women's Hospital Harvard Medical School. His work spans the fields of drug delivery, medical devices, stem cell therapeutics, and tissue adhesives. He has published >100 peer-reviewed papers (with >14,500 citations), has given >250 national and international invited lectures, and has >100 issued or pending national/international patents. Several technologies developed in his lab have formed the foundation for multiple products on the market and currently under development and for the launch of six companies that have raised over $100 Million. Karp is also dedicated to the career development of next generation bioengineers to work at the forefront of translational science.
Jonathan Kimmelman, PhD
Jonathan Kimmelman, PhD, is Associate Professor at McGill University in Biomedical Ethics Unit / Social Studies of Medicine, with a cross appointment in Experimental Medicine. His research centers on ethical, policy, and scientific dimensions of drug and diagnostics development, and he founded and directs the Studies of Translation, Ethics and Medicine (STREAM). In addition to his book, Gene Transfer and the Ethics of First-in-Human Experiments (Cambridge Press, 2010), major publications have appeared in Science, JAMA, BMJ, and Hastings Center Report. Kimmelman received the Maud Menten New Investigator Prize (2006), a CIHR New Investigator Award (2008), and a Humboldt Bessel Award (2014). He has served on various advisory bodies within the National Heart Lung and Blood Institute, the U.S. National Academies of Medicine, and the Canadian Institutes for Health Research, and makes frequent appearances in the news media. He chaired the International Society of Stem Cell Research Guidelines for Stem Cell Research and Clinical Translation revision task force 2015-16.
Aaron D. Levine, PhD
Aaron D. Levine is an Associate Professor in the School of Public Policy at Georgia Tech, Co-Director for Engineering Workforce Development for the NSF Engineering Research Center for Cell Manufacturing Technologies and Guest Researcher in the Division of Reproductive Health at the CDC. His research focuses on the intersection between public policy and bioethics and recent work has examined the development of stem cell science, translation of cell therapies, and oversight of assisted reproductive technology. He completed his Ph.D. in Public Affairs at Princeton University and also holds an M.Phil. in Biological Sciences from the University of Cambridge.
Mark C. Zimmerman, Ph.D
Vice President, Strategy and Business Development, Interim Vice President of Operations
Mark Zimmerman, Ph.D., a leader in diabetes and regenerative medicine for over two decades, is responsible for strategy and business development and is the interim Vice President of Operations. He is seconded to ViaCyte, after Johnson and Johnson completed a business transaction with ViaCyte and merged the assets of BetaLogics, a business unit of Janssen R&D LLC, into ViaCyte. The mission of BetaLogics was to discover and develop a cellular product to treat diabetes. Dr. Zimmerman was appointed Executive Director of BetaLogics in 2002, and Venture Leader/Vice President of BetaLogics in 2009. Before the establishment of BetaLogics, Dr. Zimmerman was a Principal Scientist and Group Leader at Johnson and Johnson heading up regenerative medicine projects related to orthopedic surgery, wound healing, vascular biology, and diabetes. Prior to Johnson and Johnson, Dr. Zimmerman was an Associate Professor of Surgery at UMD-New Jersey Medical School. Dr. Zimmerman received his B.S. in Biology from Syracuse University and his M.S. and Ph.D. in Biomedical Engineering from Rutgers University.
Miguel Forte, MD PhD
Currently serving as Chief Commercialization Officer and Chair of the Commercialization Committee of the International Society of Cellular Therapy (ISCT). Currently also the CEO of Zelluna Immunotherapy and visiting Professor at the Lisbon and Aveiro Universities in Portugal. From February 2006 to January 2010 was VP of Global Medical Affairs at UCB. In 2004, joined Nabi Pharmaceuticals as the VP of Clinical, Medical and Regulatory Affairs in Europe. After several clinical, academic and regulatory positions in the public sector in Portugal and at the EMA, spent six years with Bristol-Myers Squibb in various positions including Country Medical Director, Executive Director of Infectious Diseases, Immunology and Dermatology and VP of International Medical Organization in Portugal and Belgium. Holds an M.D. from the Faculty of Medicine of the University of Lisbon, Portugal, and a Ph.D. in Immunology from the University of Birmingham, UK, an accreditation as Specialist in Infectious Diseases and a certificate on Health Economics of Pharmaceuticals and Medical Technologies (HEP). He is Fellow of the Faculty of Pharmaceutical Medicine of the RCP in the UK.
Caribou BioSciences, Inc.
Rachel Haurwitz, PhD
President and CEO
Rachel is a co-founder of Caribou Biosciences and its President and CEO. She has a research background in CRISPR-Cas biology, and is also a co-founder of Intellia Therapeutics. In 2014, she was named by Forbes Magazine to the "30 Under 30" list in Science and Healthcare, and in 2016, Fortune Magazine named her to the "40 Under 40" list of the most influential young people in business. Rachel is an inventor on several patents covering multiple CRISPR technologies. She earned an A.B. in Biological Sciences from Harvard College and a Ph.D. in Molecular and Cell Biology from the University of California, Berkeley.
DEKA Research and Development Corp.
Thomas Bollenbach, PhD
Chief Technology Officer
Thomas Bollenbach, Ph.D. is a scientist at DEKA Research and Development Corp., and Chief Technology Officer at the Advanced Regenerative Manufacturing Institute (ARMI). Prior to joining DEKA, Tom served as Vice President of Research and Development at Harvard Apparatus Regenerative Technology, where he led the development of cell- and biomaterial-based tracheal, bronchial and esophageal implants. Tom joined the HART from Organogenesis, where he implemented and completed IND-enabling preclinical programs for a bioengineered living skin graft, and maintained strong cross-functional interactions with Clinical Operations, Manufacturing and Business Units to provide scientific support to corporate strategies. Tom received his B.Sc. in Biochemistry from the University of Waterloo, a Ph.D. in Biochemistry from the University of Notre Dame, and completed postdoctoral research at Cornell University.
Pepper Hamilton, LLP
Daniel Scolnick, PhD
Dr. Daniel M. Scolnick is a partner and registered patent attorney with Pepper Hamilton LLP. Dr. Scolnick concentrates his practice advising pharmaceutical and biotech clients of all sizes, with a focus on start-ups and mid-size biotech companies. He advises his clients by devising and implementing strategic patent prosecution strategies to protect novel therapeutics. Dr. Scolnick also assists his clients that are considering investing or acquiring a new technology by performing due diligence projects and drafting and negotiating commercial agreements. Prior to becoming an attorney, Dr. Scolnick received his PhD from the University of Pennsylvania where his thesis work focused on the discovery and characterization of a novel cell-cycle checkpoint protein, which was published in the journal Nature.
UC Davis School of Medicine
Jan A. Nolta, PhD
Director of Stem Cell Program
Jan A. Nolta, Ph.D., is the Director of the Stem Cell Program at UC Davis School of Medicine, and directs the Institute for Regenerative Cures. She also serves as the Scientific Director of the large UC Davis Good Manufacturing Practice Facility, and as Scientific Director of the California State Umbilical Cord Blood Collection Program. The UC Davis stem cell program has a large number of basic, translational, and clinical scientists collaborating to work toward regenerative medicine-related cures for a spectrum of diseases and injuries. Dr. Nolta is helping UC Davis teams develop numerous clinical trials of gene and cell therapy, with 24 stem cell/regenerative medicine therapies already in the clinic, and over twenty in the pipeline. A California Institute for Regenerative Medicine “Alpha Clinic” Award was just granted to UC Davis to administer FDA-approved stem cell and regenerative medicine therapies.
University of Pennsylvania
Hongjun Song. Ph.D.
Professor of Neuroscience
Hongjun Song, Ph.D. is the Perelman Professor of Neuroscience at University of Pennsylvania, and a member of the Institute for Regenerative Medicine and Institute of Epigenetics. Dr. Song received his B.S. from Peking University, B.A. from Columbia University, and Ph.D. from UCSD. Dr. Song’s laboratory has been applying human stem cell and genomics technologies for organogenesis, regenerative medicine and drug development. Dr. Song is among the world top 1% most highly cited researchers in the field of Neuroscience and Behavior. He serves on multiple editorial boards. So far Dr. Song has co-founded four start-up biotechnology companies.Web: http://www.med.upenn.edu/songlab/
University of Pennsylvania
Saar Gill, MD, PhD
Dr. Gill obtained his medical degree and Ph.D in immunology from the University of Melbourne in Australia, and a post-doctoral fellowship in cellular therapy at Stanford University. Dr. Gill is now an assistant professor of medicine at the University of Pennsylvania where he specializes in the treatment of patients with leukemia and in bone marrow transplantation. He has led clinical trials of chimeric antigen receptor (CAR) T cell trials for chronic and acute leukemias. Dr. Gill’s research laboratory focuses on the interface between adoptive cellular therapy and genetic engineering.
University of Pennsylvania
Sarah E. Millar, Ph.D
Albert Kligman Professor and Vice-Chair of Basic Research
Sarah E. Millar, PhD is Albert Kligman Professor and Vice-Chair for Basic Research in the Department of Dermatology at the University of Pennsylvania Perelman School of Medicine. Dr. Millar is internationally recognized as a leading researcher in epithelial biology. Her research group has made seminal discoveries on the roles of Wnt signaling and epigenetic regulatory mechanisms in development and regeneration of the skin and its appendages. She is an Editorial Board member for Developmental Cell and Experimental Dermatology, a Deputy Editor for the Journal of Investigative Dermatology, and has served on numerous national and international grant review committees. Dr. Millar has received several awards in recognition of her research, including an NIH MERIT Award for her work on Wnt signaling in the skin, and the 2017 William Montagna Lectureship Award of the Society for Investigative Dermatology.
Penn Institute for Regnerative Medicine
Foteini Mourkioti, PhD
Assistant Professor, Department of Orthopaedic Surgery/ Co-Director, Musculosketal Regneration Program
Dr. Mourkioti is an Assistant Professor at UPenn (Departments of Orthopaedic Surgery and Cell & Developmental Biology) and the co-Director of Penn IRM, Musculoskeletal Program. Dr. Mourkioti completed her PhD at the Max Planck Institute in Germany and her post-graduate studies at the European Molecular Biology Laboratory (EMBL ranks first in Europe and fourth in the world after CSHL, MIT, and the Salk by citation impact). After that, she got a position at Stanford University where she developed a new mouse model (mdx/mTR) that mimics the disease progression of Duchenne Muscular Dystrophy, the most common inherited form of myopathy. Dr. Mourkioti’s lab is interested in understanding how stem cells in our skeletal muscles communicate with their environment to coordinate muscle regeneration and dissect why although these cells have the potential to fix a damage, they are unable to do so if the injury is large or in case of muscle diseases.
Wharton School Univeristy of Pennsylvania
Steven Nichtberger, M.D.
Adjunct Professor, Healthcare Management Senior Fellow, Vagelos LSM Program
Dr. Nichtberger is a serial internet and biotech entrepreneur. He is managing partner of GBF, LLC investing and providing advisory services. He is co-founder of Tycho Therapeutics, Limelight Bio, ControlRad, Tengion, and Coupco. He previously held senior commercial leadership roles at Merck. At Wharton, he leads the Vagelos Life Sciences & Management capstone course on healthcare company formation, financing, and leadership. At Tengion from 2004-2011, novel cell therapy based regulatory approaches, manufacturing facilities, and clinical pathways were developed under his leadership. In 2008, Dr. Nichtberger was recognized as National Ernst and Young Entrepreneur of the Year.
Penn Center for Innovation, Univeristy of Pennsylvania
John S. Swartley, MBA, PhD
Associate Vice Provost for Research and the Managing Director of the Penn Center for Innovation
John S. Swartley, MBA, PhD is Associate Vice Provost for Research and the Managing Director of the Penn Center for Innovation (PCI) at the University of Pennsylvania. He leads a multi-faceted team that focuses on facilitating new product development, corporate partnerships, industrial R&D alliances, and new venture creation based on faculty expertise and technologies created at Penn. Prior to joining Penn in 2007, Dr. Swartley served as Senior Vice President and General Partner of BCM Technologies (BCMT), the venture capital investment subsidiary of Baylor College of Medicine. Dr. Swartley joined BCMT in 2003 from the Yale University Office of Cooperative Research where he was Associate Director of the Medical Campus Office. Over his career, Dr. Swartley has negotiated hundreds of commercialization and research partnership agreements and has participated in the formation and oversight of dozens of university spin-out companies that have collectively raised over a billion dollars of investment capital. He holds a B.S. in Biology from Bates College, an MBA from the Goizueta School of Business at Emory University, and a PhD in Microbial & Molecular Genetics from Emory University.