Prof. Chris Mason, MBBS, PhD, FSB, FRCSI, FRCS
Chief Science Officer, AVROBIO, USA and Regenerative Medicine Bioprocessing Unit, Advanced Centre for Biochemical Engineering, University College London, UK
Prof. Chris Mason is a clinician-scientist with over 20 years of cell and gene therapy experience including Full Professor of Regenerative Medicine Bioprocessing at University College London. He has a multidisciplinary track record spanning R&D, clinical medicine, bioprocessing, regulation, healthcare economics, reimbursement and commercialization. He is co-founder of the London Regenerative Medicine Network, trustee of the UK Stem Cell Foundation and on the UK-Israel Science Council. Current SAB appointments include; public and private companies, the UK Cell Therapy Catapult and the Canadian Centre for the Commercialization of Regenerative Medicine. Prof. Mason is a general spokesperson for the cell and gene therapy sector including frequent newspaper, radio and TV interviews plus social media including @Prof_ChrisMason on Twitter
Editorial Advisory Board
Head of Department of Genetics, UCL Institute of Ophthalmology, UK
Director, Wake Forest Institute of Regenerative Medicine, USA
Anthony Atala, MD, is the Director of the Wake Forest Institute for Regenerative Medicine, and the W.H. Boyce Professor and Chair of the Department of Urology at Wake Forest University. Dr Atala is a practicing surgeon and a researcher in the area of regenerative medicine. His current work focuses on growing new human cells, tissues and organs. Dr Atala has led or served several national professional and government committees, including the National Institutes of Health working group on Cells and Developmental Biology, the National Institutes of Health Bioengineering Consortium, and the National Cancer Institute’s Advisory Board. Dr. Atala heads a team of over 300 physicians and researchers. Over ten applications of technologies developed in Dr. Atala's laboratory have been used clinically. He is the editor of twelve books, including Essentials of Stem Cell Biology, Principles of Regenerative Medicine, Foundations of Regenerative Medicine, Methods of Tissue Engineering, and Minimally Invasive Urology. He has published more than 400 journal articles and has applied for or received over 200 national and international patents.
BHF Professor of Translational Cardiovascular Sciences, University of Glasgow, UK
Andrew graduated from the University of London in 1990 with a First Class BSc (Joint Honours) in pharmacology and toxicology and then studied for his PhD with the Leukaemia Research Fund at the University of Wales College Of Medicine, graduating in 1994. He then joined the group led by Professor Andrew Newby for his post-doctoral work in Cardiff and developed adenoviral vectors for gene delivery studies in the cardiovascular system. He then transferred to a lectureship at the University of Bristol (Bristol Heart Institute) to continue studies on adenovirus-mediated gene transfer to assess vascular function and gene therapy. In 1999, Dr. Baker joined Professor Anna Dominiczak at the University of Glasgow as a Senior Lecturer in Molecular Medicine, then as Reader and in 2005 as Professor of Molecular Medicine. Andrew was awarded the Blandsford Prize (1990) in pharmacology and the “Update in Thrombolysis Research” (Berlin, 1998) for his publication entitled “Divergent effects of tissue inhibitor of metalloproteinase-1, -2 or -3 overexpression on rat vascular smooth muscle cell invasion, proliferation and death in vitro: TIMP-3 promotes apoptosis” which was published in the Journal of Clinical Investigation. In 1999, he was awarded the British Cardiac Society Young Investigator Research Prize for his work “Gene therapy for vein grafting: Tissue inhibitor of metalloproteinases-3 (TIMP-3) inhibits neointima formation in vitro and in vivo in part by promoting apoptosis”. He was awarded the MakDougall-Brisbane prize from the Royal Society of Edinburgh in 2008 and a fellowship from the Society in 2010. Also in 2010 he was awarded an Outstanding Achievement Award from the European Society of Cardiology and in 2011 received a Royal Society Wolfson Research Merit Award. From August 2010 to November 2011 he was Acting Director of the Institute for Cardiovascular and Medical Sciences at the University of Glasgow and in 2011 he was awarded a British Heart Foundation Chair of Translational Cardiovascular Medicine.
Director, Cell Therapy Processing/Advanced Cell Therapy Labs, Yale University, USA
Managing Partner, Proteus Venture Partners, USA
Greg is the Founder & Managing Partner of Proteus, LLC. Mr. Bonfiglio was an early investor in the field of stems cell & regenerative medicine, and he continues to actively invest in the field. Proteus currently is managing the Proteus Growth Fund, which invests in publicly traded regenerative medicine companies. Proteus also is raising a new early stage, venture fund focused exclusively on regenerative medicines.He is the Chairman of the Board of the Centre for Commercialization of Regenerative Medicine, a RM Translation Center in Toronto, Canada. He also is a Member of the Board of the MaRs Discovery District, an Innovation Hub in Toronto. He is a Member of the ISSCR and is on their Advisory Board, as well as their Finance Committee. He also is a Member of the ISCT.
Mr. Bonfiglio has served on the Boards of several RM companies, including VistaGen Therapeutics (in vitro tools from hESCs); California Stem Cell (hESC tools & therapeutics); and StemCyte, Inc. (cord blood storage & therapeutics). Mr. Bonfiglio is a frequent speaker at Regenerative Medicine Conferences. In 2013, Mr. Bonfiglio was listed among the Top 50 Stem Cell Influencers in the world (Ranked 14th).
Vice President, Research, Juno Therapeutics, USA
Mark obtained his PhD in immunology in the lab of Jim Allison at UC Berkeley where he studied T cell developmental biology, with an emphasis on molecular immunology and T cell differentiation. He did his post-doctoral training at SyStemix, in Palo Alto, studying HIV pathogenesis and continued work at SyStemix as a senior scientist focusing on hematopoietic stem cell-based gene therapy for treating HIV. In 1997, he joined Xcyte Therapies in Seattle as Director, and later Vice President of Research, where he directed research that led to a series of T cell-based immunotherapy clinical trials. He was also responsible for collaborations related to multiple gene and cell therapy applications. In 2006, Dr. Bonyhadi joined Invitrogen/Life Technologies, where he was responsible for identifying and implementing business opportunities aligned with their cell therapy/cellular medicine portfolio, participating in the development of new products optimized for cell therapy applications, as well as working with academia and industry to accelerate the development of novel cell-based therapeutics. Currently, he is Vice President for Research at Juno Therapeutics, we he is focused on building commercially viable gene-engineered T cell platforms for treating human disease.
Director, Consulting on Advanced Biologicals, UK
Christopher founded Consulting on Advanced Biologicals Ltd at the end of 2009 in order to focus his activities within the Regenerative Medicine sector. CAB Ltd provides EU regulatory services to the regenerative medicine industry in addition to business and regulatory research and analysis to identify and focus on the real barriers to commercialisation of regenerative medicine. Christopher has spent most of his career working in regenerative medicine: starting with a PhD in xenotranplantation immunology he first moved into industry in 1998 when he joined Imutran Ltd (A Novartis Pharma AG Co.) developing novel transgenic pigs to treat human organ failure. Following the closure of Imutran, Christopher moved to Intercytex and subsequently made the transition to regulatory affairs by joining the MHRA as a quality (CMC) assessor (biologicals and biotechnology unit). During this time Christopher was involved with National implementation of the new Advanced Therapies Regulation and also involved through his participation in the CHMP’s cell products working party (CPWP) in implementation at the EMA level including drafting guidelines.
Associate Dean Research, School of Public Health, University of Alberta, Canada
Dr Tania Bubela BSc Hons, PhD, JD is Professor and Associate Dean (Research) in the School of Public Health and Adjunct Professor in the Alberta School of Business at the University of Alberta, Canada. She joined the faculty of the U Alberta in 2004 after clerking for The Honourable Louise Arbour at the Supreme Court of Canada, articling at Field Law LLP in Edmonton, and being called to the bar (Law Society of Alberta) in 2005. Her research program in intellectual property and health law related to translational biomedical research brings together her legal training and a PhD in biology and expertise in genetics and molecular biology. Her research program focuses on large collaborative science networks in genomics, gene therapy, and stem cell biology, addressing barriers to the effective translation of new technologies. These are varied and include ethical issues, effective communication of risks and benefits among stakeholder groups, commercialization and regulation. She provides advice for Government Health and Science agencies as well as life sciences research communities, and patient organisations.Her research is funded by the Canadian Institutes of Health Research, the Canadian Stem Cell Network, Genome Canada, and Alberta Innovates - Health Solutions, among others. She co-leads the PACEOMICS program on the development of cost-effective personalized medicine and the Alberta Ocular Gene Therapy Team, which is developing novel gene therapies and conducting a phase I clinical trial of the NighstaRx AAV2-REP1 product for choroideremia. She has nearly 100 publications in law, ethics and science policy journals including Nature, Nature Biotechnology, Cell Stem Cell, PLoS Biology, Trends in Biotechnology, American Journal of Bioethics and Science Translational Medicine.
VP, Business & Corporate Development, Replicel, Canada
Lee has been an executive in the cell therapy sector since 2000 beginning with Malachite Management in the Stem Cell Technologies group of companies. Most recently, he was the Managing Director of Cell Therapy Group, a firm he formed in 2008 where he did business development consulting for companies and organizations in or interested in the cell therapy sector. His work included deal-targeting, transactions, market intelligence, competitive analyses, strategic assessments, and market profile planning for companies ranging from top-tier multinationals to start-ups.
Lee has a Bachelor’s Degree in Education and a Law Degree. After law school, Lee did a one year judicial clerkship with the B.C. Supreme Court and was a practising attorney for three years at Edwards, Kenny & Bray. Lee served six years as the Executive Director of the International Society for Cellular Therapy and just over two years as Director of Business Development for Progenitor Cell Therapy. He is on the editorial advisory boards of the journal Regenerative Medicine and the BioProcess International magazine, as well as the Co-Chair of the Alliance for Regenerative Medicine’s Communications and Education Committee. He co-founded Cell Therapy News, founded Cell Therapy Blog, founded and continues to manage the LinkedIn Cell Therapy Industry Group, co-founded Regenerative Medicine Jobs, and is an active industry commentator in publications and in social media. Lee serves on numerous industry conference advisory boards, is an advisory board member for BioCision and RoosterBio, and is on the Board of Directors for Hemostemix.
Hematopoietic Innovative Therapies Div/, CIEMAT/CIBERER & IIS-Fundación Jiménez Díaz, Spain
Director, Center for Stem Cell Research & Regenerative Medicine, Tulane University, USA
Bruce Bunnell is the Director of the Tulane Center for Stem Cell Research and Regenerative Medicine and Professor in the Department of Pharmacology in the Tulane University School of Medicine. In addition, he holds the Aron Family Regents Distinguished Chair in Gene Therapy. Dr Bunnell obtained his PhD in Microbiology from the University of Alabama at Birmingham School of Medicine. He then pursued Postdoctoral Fellowship research at the Howard Hughes Medical Institute in the School of Medicine at the University of Michigan and the National Human Genome Research Institute at the National Institutes of Health in Bethesda, MD. Dr Bunnell was an Assistant Professor at the Nationwide Children’s Hospital Research Institute, part of the Ohio State University School of Medicine prior to joining the faculty at Tulane University in 2002. Dr. Bunnell's research program is focused on both the basic science and translational applications of adult stem cells isolated from the bone marrow and adipose tissue. Dr. Bunnell investigates the use of mesenchymal stem cells isolated from the bone marrow or adipose tissue as a therapeutic intervention for both Multiple Sclerosis (MS) and acute lung injury. He is currently working towards a human clinical trial for the treatment of MS with these cells. He has served as a reviewer of stem cell, regenerative medicine and tissue engineering grants for the National Institutes of Health, Department of Defense and several state funded programs including Maryland, New York, Virginia and Pennsylvania. He has also served on grant review panels for several foreign countries including Denmark, Ireland, Germany and Spain. Dr. Bunnell serves as an Editorial Board Member for several journals, including Stem Cells, BMC Genomics, and Regenerative Medicine. He is currently a standing member of the NIH TAG study section.
Director of Research, INSERM, France
Professor of Medicine, Paris Descartes University, France USA
Programme Director – Scientific Affairs, National Institute for Health and Care Excellence, UK
CTO, TiGenix, Belgium
Mr Wilfried Dalemans holds a PhD in molecular biology from the Universities of Hasselt and Leuven. Before joining TiGenix, Wilfried held several senior management positions at GlaxoSmithKline Biologicals, Belgium. As director regulatory strategy and development, he was responsible for the worldwide registration of GlaxoSmithKline's flu franchise. With this firm, he also served as director of molecular biology and research, responsible for the development of nucleic acid and tuberculosis vaccines, as well as immunology research activities. Prior to joining GlaxoSmithKline, he worked at Transgène, France, where he was responsible for the cystic fibrosis research program. Wilfried also served as a supervisory director of Arcarios B.V. and a director of Arcarios NV.
Senior Vice President, Gene Therapy, Biogen Idec, USA
Olivier also served as chief scientific officer at Genethon and senior director of research at Somatix Therapy Corporation. He has held several senior roles at the French National Centre for Scientific Research(CNRS) and at the Institut Pasteur in Paris. Olivier is the former president and a founding member of the European Society of Gene and Cell Therapy.
Olivier received a Master’s in Genetics and Molecular Biology at University of Paris Orsay, and his PhD in Biology at the Pasteur Institute and University of Paris Diderot.
CSO, Rubius Therapeutics, USA
Robert Deans is responsible for regenerative medicine technology development at Athersys Inc. and its European subsidiary, ReGenesys. Athersys is developing cell therapeutics based on adherent stem cells (MultiStem) isolated from adult bone marrow. Athersys has active Phase II clinical development activity in acute myocardial infarct, stroke, ulcerative colitis, acute respiratory distress syndrome and for adjunctive therapy of allogeneic bone marrow transplant. Dr. Deans has also been chairman of the ISCT Commercialization Committee, co-chair of the Science and Technology Committee of ARM, and serves on the ISSCR Clinical Translation Committee.
Dr Deans has more than 20 years of experience in stem cell therapeutics, having previously served at Osiris Therapeutics as VP of Research. Dr Deans was previously Director of R&D at Baxter Healthcare, where he developed biological components of the Isolex300i hematopoietic stem cell purification platform. In addition, Dr Deans served on the faculty at USC Medical School from 1984 to 1992. He holds degrees from MIT and the University of Michigan, and postdoctoral training in molecular immunology at UCLA.
Director, Program in Cord Blood Transplantation, Fred Hutchinson Cancer Research Center, USA
Dr Delaney’s research interests focus on the role of the Notch signaling pathway in hematopoietic stem cell regulation and ex-vivo expansion of umbilical cord blood stem and progenitor cells for clinical applications. Her goal is to improve the outcome of patients in need of a cord blood transplant who remain at increased risk of slow hematopoietic recovery and early transplant related mortality secondary to the low cell dose associated with a cord blood graft. Her group has developed a novel and clinically feasible method for the ex vivo expansion of cord blood derived hematopoietic stem and progenitor cells in the presence of Notch ligands. This work was translated into a novel pilot study investigating the use of ex vivo expanded cord blood progenitors to augment conventional cord blood transplantation. The results of her clinical trial were published by Nature Medicine. She has since extended this work to investigate the potential of cryopreserved, non-HLA matched “off the shelf” ex vivo expanded cord blood progenitor cells to provide rapid but transient myeloid reconstitution in the setting of cord blood transplant and following dose-intensive chemotherapy.In addition to Dr Delaney’s primary research focus on ex vivo expansion, her group is actively investigating the kinetics of engraftment, graft-versus-graft effects and the kinetics of immune reconstitution in transplant recipients enrolled on several actively accruing cord blood transplant protocols.
Head Lab of Cell Biol. & Advanced Cancer Therapies, Univ. Modena & Reggio Emilia, Italy
Massimo is a clinical scientist developing cell and gene therapy approaches around cancer patients. He got his MD degree at the University of Pavia (Italy) then internship, residency and post-doctoral training between the Institute of Haematology, Vienna University (Austria), the Division of Immuno-haematology, Ferrara University (Italy) and St Jude Children's Hospital, Memphis (USA). After that hospital physician, assistant professor of Medical Oncology and head of the Laboratory of Cellular Therapies at University/Hospital of Modena. In December 2013 associate professor of Medical Oncology (Nat. Board on Scientific Habilitation). Twenty three between research grants and awards. Eighty-four papers on stem cells, tissue regeneration, experimental oncology and hematology with over 8000 citations. Author of 3 books, 3 chapters and 6 patents. Founder of the University start-up Rigenerand. Founder and scientific coordinator of the Mirandola science & tecnology park. Co-editor, editorial board nember and referee for several scientific Journals. Reviewer for 13 national & international founding Bodies. He has been co-founder of the Forum of Italian Researcher on MSC (FIRST), board member of JACIE, WBMT and scientific advisor for the Italian Minister of Health on stem cells. He has been member of ISCT, ASH, ESCGT, IFATS. He is the current President of the International Society for Cellular Therapy (ISCT).
Co-Director of the Oxford Stem Cell Institute, Oxford University, UK
Paul Fairchild began his research career in Oxford, where he studied for a doctorate in the Nuffield Department of Surgical Sciences, focussing on the dual role of dendritic cells in allograft rejection and immunological self-tolerance. After spending five years as a Post-doctoral Fellow investigating the aetiology of autoimmune disease in the Department of Pathology, University of Cambridge, he returned to Oxford, where he is currently a Associate Professor within the Sir William Dunn School of Pathology and a Fellow of Trinity College. In 2008, Paul Fairchild founded the Oxford Stem Cell Institute (OSCI), for which he continues to serve as Co-Director. It is within this context that he continues to apply his background in transplantation immunology, to investigate the nature of the immune response to tissues differentiated from pluripotent stem cells and to develop approaches to the induction and maintenance of immunological tolerance. He has also developed a programme of research aimed at exploiting the properties of pluripotent stem cells to address unmet medical needs with an immunological basis.
Head of the Research Dept. Cell and Gene Therapy, University of Hamburg, Germany
JOSEPH GLORIOSO III
Professor of Microbiology & Molecular Genetics, University of Pittsburgh, USA
Joseph has established a 35-year history of research related to the basic biology and genetics of herpes simplex virus. His contributions to the field include defining antiviral immune responses to infection, the genetics of viral pathogenesis and latency, and mechanisms of viral infection. Furthermore, he has been a pioneer in the design and application of HSV gene vectors for the treatment of nervous system diseases such as peripheral neuropathies, chronic pain, and brain tumors. He continues to be a worldwide leader in the HSV gene vector field through the creation of innovative gene vectors and the development of manufacturing methods for Phase I and Phase II human clinical trials for pain gene therapy. His enkephalin vector showed considerable promise in a Phase I human trial to treat cancer pain and phase II testing is underway. These trials were sponsored by Diamyd Medical AB in Stockholm.
Dr Glorioso’s most recent research has focused on (i) the design and application of HSV gene vectors for exploring the molecular events that occur in sensory afferents that are involved in the transition from acute to chronic pain, (ii) the development of retargeted oncolytic HSV vectors for specific infection and replication in human glioblastomas and applications to treatment of xenograft models of human brain human brain tumors, (iii) the creation of novel HSV vectors that cross the blood brain barrier by transcytosis followed targeted infection and gene expression in spiny neurons of the striatum; these vectors are being applied to treatment of animal models of Huntington’s disease and (iv) the use of HSV gene vectors for the creation of induced pluripotent stem (iPS) cells and the identification of transcriptional regulatory and signaling processes that participate in cellular reprogramming.
PHILIP D GREGORY
CSO, Bluebird Bio, USA
Philip D Gregory, D.Phil. joined Bluebird Bio as its Chief Scientific Officer and a member of the leadership team having served as Chief Scientific Officer of Sangamo Biosciences from July 2009 and Senior Vice President, Research since January 2014 to June 2015. He joined Sangamo in December 2000 as a Scientist, became a Team Leader in October 2001, Senior Director, Research in July 2003 and Vice President, Research in October 2005. Prior to joining the company, Dr Gregory was at the University of Munich, Germany, where he studied the role of chromatin structure in gene regulation and published extensively in this field. He has served as a member of the Scientific Advisory Board of Keystone Symposia since December 2009 and was named by Thomson Reuters as one of “The World’s Most Influential Scientific Minds 2014”. Dr Gregory earned a D.Phil. in biochemistry from the University of Oxford and holds a B.Sc. in microbiology from the University of Sheffield.
Head of DNA Vector Research, German Cancer Research Centre (DKFZ), Germany
Executive Director, TUMCells, Technical University Munich, Germany
Martin Hildebrandt studied Medicine at the Free University Berlin, performed research in the Department of Biochemistry with Werner Reutter and was a visitor to Jonathan D Gitlin’s lab in Washington University School of Medicine, St. Louis. After receiving his board Certifications in Internal Medicine, Hematology/ Oncology and Blood Transfusion specialties, Prof Hildebrandt has been in charge of Clinical Units and GMP facilities in Berlin and Hannover, Germany, before taking over the responsibility for establishing TUMCells, the Interdisciplinary Center for Cellular Therapies at TUM School of Medicine which he chairs as Executive Director and qualified Person. The major area of research of Martin Hildebrandt is T-cell mediated immunomodulation and Transplant optimisation.
Martin Hildebrandt is member of the working group on blood-associated infectious risks at the Federal Ministry of Health, Chair of the Ethics Committee of the State of Berlin, Vice President-elect of ISCT Europe, and member of several editorial boards, national and international professional societies.
CSO, Cell Therapy Catapult, UK
Johan was appointed Chief Scientific Officer of the Cell Therapy Catapult in May 2013. As Chief Scientific Officer, he is responsible for external scientific relations, assessment of candidate projects and technical oversight of preclinical programmes. He has extensive experience of the life sciences industry, having worked in cell and tissue companies in Europe and the US for over 15 years. Johan has been involved in numerous transactions and collaborations, establishment of scientific facilities, and the management of scientific programmes in industry and academia. Johan has a PhD from the University of Gothenburg in Sweden and is currently adjunct professor at Linköping University in Sweden.
Director, Cell Therapy Program, University of Toronto, Canada
Armand is Professor of Medicine and also a Professor in the Institute of Biomaterials and Biomedical Engineering, at the University of Toronto, Toronto, Canada. For the past 19 years he was Director, Division of Hematology and Epstein Chair in Cell Therapy and Transplantation at the University of Toronto as well as Chief of Medical Services at Princess Margaret Hospital/Ontario Cancer Institute in Toronto for a decade. He has served as president of the American Society of Blood and Marrow Transplantation and was also president of the American Society of Hematology. He is currently Director of the Cell Therapy Program and the Orsino Cell Therapy Translational Research Laboratory at Princess Margaret Cancer Centre. His interests focus on cell-based tissue regeneration, anti-cancer cell therapy, and blood and marrow transplantation. He has conducted laboratory, translational and clinical research in these areas, particularly on the biology and clinical application of mesenchymal stromal cells and NK cells.
CEO and CSO, Intercytex, UK
Paul has been involved in commercial regenerative medicine for over 25 years. He has a successful track record in virtually all aspects of the business from founding biotech companies, raising significant public and private funding in Europe, US and Asia; designing, building and operating GMP facilities in the US and UK; designing and operating clinical trials including extensive interactions with the MHRA, EMA and FDA; operating multinational, multi-center Phase I, II and III studies, and finally, launching marketing and selling cell based products both in the US and UK.
VP and Development Head, Gene Therapy, GSK, UK
Dr Sven Kili is the VP and Development Head for the Gene Therapy division of GSK where he leads the teams developing ex-vivo Gene Therapies for a variety of genetic disorders. They are currently developing solutions for a number of diseases including ADA-SCID; WAS; Metachromatic Leukodystrophy (MLD) and Beta-Thalassemia. Prior to this, he was Senior Director, Cell Therapy and Regenerative Medicine for Sanofi (Genzyme) Biosurgery where he led the clinical development and medical affairs activities culminating in the granting of the first combined ATMP approval in the EU for MACI®. His team also prepared and submitted regulatory filings for Australia and the US, including health technology assessments. Before joining Genzyme, Sven worked for Geistlich Pharma where, in addition to leading the cell therapy medical activities, he oversaw all UK regulatory functions and was the QPPV for the EU. Sven trained as an Orthopaedic surgeon in the UK and South Africa and since leaving full-time clinical practise has developed expertise Cell and Gene Therapy in clinical development, regulatory compliance, value creation, risk management and product safety, product launches and post-marketing activities. He sits on the board of a Swedish Stem Cell company and still maintains his clinical skills in the UK NHS and serves as an ATLS Instructor in his spare time.
Director, Carolinas Cord Blood Bank, Duke University, USA
President and Chief Executive Officer, Sangamo BioSciences, Inc., USA
Partner, Hempsons, UK
James has always worked in the health sector. He qualified as a solicitor in 2000 after training with a specialist healthcare firm. After qualifying he became increasingly specialised in the regulation of assisted reproduction and embryo research, and subsequently in broader human tissue and cell based therapies and research. He later joined the litigation team at Clifford Chance before setting up his own niche life sciences firm in 2010. James joined Hempsons in 2015. James advises a large number of clinics, hospitals, universities and research centres licensed by the Human Fertilisation and Embryology Authority (HFEA), the Human Tissue Authority (HTA) and the Care Quality Commission (CQC). He has been involved in most of the leading cases relating to assisted reproduction, embryo and stem cell research.
In addition to his private practice, James was a Lecturer in Law and Medicine at the University of Newcastle and a Visiting Research Fellow at Durham University Law School. He is now an Honorary Lecturer in the Department of Biochemical Engineering at University College London.
Professor of Virology, Kings College London, UK
Director of Cellular Therapy & Biobanking, University College London, UK
Mark trained as an immunopathologist at the Royal London Hospital and moved to the Royal Free Hospital / UCL in 1994 to set up the immunotherapy programme in malignant haematology. He has specialist knowledge of cellular therapeutics and is the UK representative on the Joint Accreditation Committee ISCT/EBMT (JACIE) which is responsible for setting and co-ordinating standards for cellular therapies across Europe. Mark holds Qualified Person status for the release of investigational somatic cell therapy medicinal products in the EU and is a Designated Individual under a Human Tissue Authority licence for therapeutic cells.
Managing Partner, Proteus Venture Capital, USA
Geoff is a skilled Chief Executive with proven success leading highly innovative businesses. He has focused in the field of Regenerative Medicine for the last 15 years and is credited with building the first cell-based technology business model which has attained material commercial success as measured by revenue and profit.
Most recently, Geoff spent 11 years as C.E.O. of Organogenesis Inc. in Boston. This company has treated roughly 1 million patients with living cell-based therapies which constitutes more than the rest of the regulated world combined. Paramount to the success of the company was addressing the major hurdles faced in the field of cell-based regenerative medicine; cost-effective manufacturing, navigating regulators and payers, building a reliable supply chain and an effective commercial infrastructure. These learnings, developed and refined, are directly applicable to the broader field. In addition to financial success, relevant metrics include obtaining the first FDA CBER approval for a mass-produced cell therapy and successfully implementing >50 FDA approved process changes to on-market products.
ABY J MATHEW
Senior Vice President & Chief Technology Officer, BioLife Solutions, Inc, USA
Aby Mathew was part of the founding team of BioLife Solutions and is a co-developer of BioLife’s biopreservation media solutions. He is a co-inventor on multiple issued and pending patents related to methods, devices, and formulations for the preservation of cells, tissues, and organs. He holds a PhD in Biological Sciences within the Biochemistry, Cell and Molecular Biology Program from Binghamton University and a BS in Microbiology from Cornell University. Dr Mathew has been researching low temperature biopreservation since 1994, and his studies contributed to the development of BioLife’s current commercial HypoThermosol® and CryoStor® product platforms and intellectual property foundation. Dr. Mathew was part of the scientific team that linked cell death via apoptosis (programmed cell death) to exposure to hypothermic and/or freezing temperatures. These discoveries were integral to the development of BioLife’s improved intracellular-like biopreservation solutions, and also contributed to improvements in cryosurgical ablation of cancer. Dr Mathew was BioLife’s first Director of Manufacturing, established BioLife’s initial Quality system, and is currently Senior Vice President and Chief Technology Officer for BioLife Solutions. Dr. Mathew is currently active in, or previously a member of, the International Society for Cell Therapy (ISCT), AABB (formerly the American Association of Blood Banks), BEST (the Biomedical Excellence for Safer Transfusion collaborative), the Alliance for Regenerative Medicine (ARM), Tissue Engineering & Regenerative Medicine International Society (TERMIS), Society for Cryobiology , International Society for Biological and Environmental Repositories (ISBER), American Society for Cell Biology, and the Society for In Vitro Biology. Dr Mathew is a member of the Advisory Panel of the Parent’s Guide to Cord Blood Foundation, the Scientific Advisory Board of HemaCare Corporation, and the founding Board of Directors of the Cord Blood Association.
Fellow in Science and Technology Policy, Baker Institute, Rice University, USA
Kirstin is a fellow in science and technology policy at the Baker Institute. She is also a lecturer in the Wiess School of Natural Sciences and an adjunct lecturer in the Department of Sociology at Rice University. Matthews manages the activities of the Baker Institute Science and Technology Policy Program, which include overseeing events, conducting policy research, and writing policy publications. Her research focuses on the intersection between traditional biomedical research and public policy, which she publishes both through the Baker Institute and in peer-reviewed journals. Current projects include the Baker Institute International Stem Cell Policy Program, the Civic Scientist Lecture Series and Outreach Program, and policy studies in research and development funding. Matthews came to Rice University as a postdoctoral research associate in the Department of Physics and Astronomy and a research assistant at the Baker Institute in 2003. From 2004 to 2006, Matthews was also the project director for the task force Access to Health Care in Texas: Challenges of the Uninsured and Underinsured. The task force released the report “Code Red: The Health of Texas” in April 2006, followed by an update, “Code Red 2008,” in March 2008. Matthews has a B.A. in biochemistry from The University of Texas at Austin and a Ph.D. in molecular biology from The University of Texas Health Science Center at Houston.
CEO, Centre for Commercialization of Regenerative Medicine, Canada
Michael completed his PhD in Chemical Engineering at the University of Toronto in 1998 as a NSERC Scholar and was awarded the Martin Walmsley Fellowship for Technological Entrepreneurship.
Michael is currently the Chief Executive Officer of CCRM. Prior to CCRM, Michael was the President, Chief Operating Officer and co-founder of Rimon Therapeutics Ltd, a Toronto-based regenerative medicine company developing novel medical polymers that possess drug-like activity. Michael sits on a number of Boards and advisory committees, including: MaRS Innovation, Rimon Therapeutics Ltd, 20/20 Vision, the Advanced Regenerative Tissue Engineering Centre, the Department of Chemical Engineering and Applied Chemistry, at the University of Toronto and the McMaster Mohawk Biotechnology Program.
JEFFREY A MEDIN
MACC Fund Professor, Medical College of Wisconsin, USA
Dr Jeffrey A. Medin received his PhD in Biochemistry from the University of Kentucky. He then trained at the NIH working on gene transcription and gene transfer/therapy. Following that he was appointed as an Assistant Professor of Medicine at the University of Illinois-Chicago. In 2001, Dr. Medin relocated to Toronto, Canada. He was recently a Senior Scientist at the University Health Network and a Full Professor in the Department of Medical Biophysics at the University of Toronto. In January 2016, Dr. Medin started a new position as the MACC Fund Endowed Professor in the Department of Pediatrics at the Medical College of Wisconsin in Milwaukee. He is also Vice Chair of Research Innovation for the Department of Pediatrics, Research Director within the Section of Pediatric Hematology/Oncology, GMP Vector Facility Director, and holds appointments in the MCW Cancer Center and the Blood Research Institute as well.
Dr Medin is an Academic Founder and member of the SAB of AVROBIO LTD. He is also a member of the SAB of Plexcera Therapeutics and an editorial board member of the World Journal of Stem Cells, Biomedicines, and Cell and Gene Therapy Insights. Dr. Medin has published more than 100 peer-reviewed papers, edited an immunotherapy book, and presented at more than 100 invited conferences and workshops.
CMO, uniQure, Netherlands
Professor, Department of Clinical Gene Therapy, Osaka University, Japan
Ryuichi Morishita, M.D., Ph.D., graduated Osaka University Medical School in 1987, and received Ph. D. from Osaka University in 1991. After following postdoctoral Fellow at Stanford University School of Medicine, he served as an Assistant Professor, Department of Geriatric Medicine, Osaka University Medical School from 1994 to 1998. Then, from 1998 to 2003, he was Associate Professor, Division of Gene Therapy Science, Osaka University Medical School. Then, he became Professor & Chairman of Division of Clinical Gene Therapy, Graduate School of Medicine, Osaka University Medical School from 2003. Professor Morishita received over 20 awards from various academic societies including Harry Goldbratt Award in Council of High Blood Pressure, American Heart Association, Award in Japanese of Japan Medical Society, Sato Award in 27th annual meeting of the Japanese Circulation Society and Invitrogen-Nature-Biotechnology Award.
Currently, he is a member of Regulatory Affairs and also Strategic Adviser of Health Care & Medical Strategy in Abe Cabinet. He is also Special Strategic Adviser in Osaka City and Osaka Prefecture. Previously, he worked as a member of Intellectual Property Committee of Koizumi & Abe Cabinet. His professional activities & appointments include Committee for Industry-Academia Relationship, Ministry of Education, Science and Culture of Japan, Vice Chairman, Bio-Venture Association Originated from Universities, and others.
Reader in Immunology and Immunotherapy, University College London, UK
Emma Morris is a Reader in Immunology and Immunotherapy at UCL and Honorary Consultant in Haematology/Stem Cell Transplantation at UCLH and the Royal Free. She trained in Medicine at Cambridge University graduating in 1992. Following qualification she completed general medical training at Guys’ Hospital London and specialist Haematology training at The London and St Bartholomew’s Hospitals London, Addenbrookes’ Hospital Cambridge and subsequently UCLH, London. During this time she secured a Wellcome Trust Clinical Research Training Fellowship and completed a PhD in haematopoietic stem cell biology at the University of Cambridge. On completion of speciality training in 2002 she has established her own Research Group, working alongside Professor Hans Stauss, funded by Leukaemia and Lymphoma Research, the Medical Research Council and Cancer Research UK. Her research team (currently 6 scientists and 1 clinical trial co-ordinator) is developing novel gene and cell therapies for the treatment of haematological malignancies. She is the Chief Investigator of 3 Phase I ‘first time in man’ studies. These studies involve the testing of genetically modified immune cells in leukaemia and/or stem cell transplant patients. She is a Board Member of the British Society for Gene and Cell Therapy, on the Editorial Board of the British Journal of Haematology, Director of the UCL/UCLH Biomedical Research Centre Inflammation, Infection and Immunity Programme, Immunotherapy Theme Lead of the UCL Experimental Cancer Medicine Centre, Chair of the UCL GM Safety Committee for Clinical Trials.
Department of Molecular Medicine, The Scripps Research Institute, USA
Kevin Morris received his BS degree from Humboldt State University in 1996 and his PhD in 2001 from the University of California Davis. During his post-doctoral training at the University of California San Diego (2001-2004) he determined that small non-coding RNAs were capable of modulating gene transcription in human cells. The Morris lab has since determined the mechanistic underpinnings of non-coding RNA mediated regulation of gene transcription in human cells, with evidence suggesting a role for longer forms of non-coding RNAs as endogenous effectors involved in epigenetically remodeling target loci in human cells. The lab is specifically interested in utilizing the recently described endogenous non-coding RNA pathway in human cells to epigenetically modulate gene expression in those genes involved in HIV-1 cancer and cystic fibrosis. Notably, evidence suggests that this endogenous pathway can be utilized to either epigenetically silence or activate a genes expression. The Morris lab is also active in developing cell targeted non-coding RNA delivery vehicles, such as lentiviral derived conditionally replicating vector systems, cell targeted aptamers and nanoparticles.
Group Leader - Junior Group Genome Engineering, University of Freiberg, Germany
Claudio Mussolino is a junior group leader at the Institute for Cell and Gene Therapy and the Center for Chronic Immunodeficiency in Freiburg (Germany) since 2012. He graduated at University of Napoli “Federico II” in 2004 and received his PhD in 2009 at the TIGEM, Telethon Institute of Genetics and Medicine, in Napoli (Italy) where he established a mutation-independent approach to treat dominant forms of the blindness disorder Retinitis Pigmentosa using zinc finger-based designer transcription factors. Afterwards he served as postdoctoral fellow at the Hannover Medical School (Germany) where he developed transcription activator-like effector nucleases (TALENs) for the development of novel anti HIV therapeutics before becoming a junior group leader in Freiburg.
Currently, Dr Mussolino research focuses on the development of novel and long-term therapeutic strategies for HIV patients based on editing hematopoietic stem cells in a way that the derived immune system is resistant to HIV infection. Additional current projects include the adaptation of this methodology to other acquired and congenital disorders of the immune system.
Director of the Katharine Dormandy Haemophilia Centre, Royal Free Hospital, UK
Professor Amit C Nathwani is the Director of the Katharine Dormandy Haemophilia Centre at the Royal Free Hospital and a Senior NIHR Investigator. He is also Professor of Haematology at UCL. He graduated in Medicine from the University of Aberdeen in 1984. His PhD was on the regulation of the tissue factor gene. In 1997 he moved briefly to St Jude Children’s Research Hospital, Memphis, Tennessee, USA to work with Dr Arthur Nienhuis on adeno-associated virus mediated gene transfer, which is where he started his pioneering work on gene therapy for hemophilia B. In 2001 he returned to University College London as a Senior Lecturer in Haematology and a Consultant to the National Blood Services in the UK. He was the first to show successful correction of bleeding diathesis in patients with severe hemophilia B using a distinct gene transfer approach developed in collaboration with Drs Davidoff and Nienhuis at St Jude Children’s Research Hospital. Prof Nathwani’s team of clinical and non-clinical scientists and students are currently engaged in a diverse range of translational research including gene therapy for hemophilia A, congenital bleeding disorder, chronic lymphocytic leukemia and age-related macular degeneration.
CSO, Cell & Gene Therapy Unit, Novartis, USA
Dr. Perry has held a variety of positions in Biotech and Pharmaceutical companies over his career and has a proven track record in R&D innovation with emphasis on product development/approval, strategic portfolio management, business development, and driving shareholder value. Prior to re-joining Novartis in 2012, he was a Venture Partner with Bay City Capital, LLC from 2005 to 2012. Dr. Perry was a co-founder of Extropy Pharmaceuticals, Inc. and served as its Chairman and CEO from 2003 to 2005. He served as President and CEO of Pharsight Corporation from 2002 to 2003 and as Worldwide Head of R&D for Baxter BioScience from 2000 to 2002. From 1994 to 2000, Dr. Perry served initially as Vice President of Regulatory Affairs for Novartis Pharma (then, Sandoz Pharmaceuticals) and later as President and CEO of two wholly owned cell and gene therapy subsidiary companies of Novartis - SyStemix, Inc. and Genetic Therapy, Inc. From the late 1980's to the mid 1990's he held various executive positions at Syntex Corporation (later, Roche Pharma), Schering-Plough Corporation (later, Merck) and BioResearch Corporation, a subsidiary of Connaught Laboratories.
Dr. Perry currently serves as Adjunct Professor at the University of Colorado, School of Medicine, Anschutz Medical Campus in the Gates Center for Regenerative Medicine and Stem Cell Biology.
Wellcome Trust-MRC Cambridge Stem Cell Institute, University of Cambridge, UK
Global Head of Clinical Business, Miltenyi Biotec, Germany
Kai Pinkernell joined Miltenyi Biotec GmbH, Bergisch Gladbach, Germany, in 2009 and is, as the Global Head of Clinical Business, responsible for clinical marketing, sales and development, worldwide. Miltenyi Biotec, with more than 1400 employees, develops and sells translational research products as well as a large clinical product portfolio to enable immunoadsorption and cellular therapy in different therapeutic areas, like hematopoetic stem cell transplantation, immunotherapies in cancer and regenerative medicine applications.
Dr Pinkernell has previously been with Cytori Therapeutics Inc., San Diego, USA, from 2005 to 2009, as Senior Director of Regenerative Cell Technology and Clinical Applications Director. He received his medical degree from the Westfaelische-Wilhems University in Muenster, Germany, and pursued a residency/fellowship in internal medicine/cardiology at the German Heart Center and Klinikum Rechts der Isar, Technical University, in Munich, Germany. He further received a Master of Business Administration from the Marshall School of Business at the University of Southern California, Los Angeles, USA.
Executive Director, Harvard Stem Cell Institute, USA
Brock Reeve is Executive Director of the Harvard Stem Cell Institute. In partnership with the Faculty Directors, he has overall responsibility for the operations and strategy of the Institute whose mission is to use stem cells, both as tools and as therapies, to understand and treat the root causes of leading degenerative diseases. HSCI is comprised of the schools of Harvard University and all its affiliated hospitals and research institutions. Under the leadership of the Executive Committee, HSCI invests in scientific research and its faculty has grown to include over 300 Principal and Affiliated members. The Institute is engaged with several leading pharmaceutical companies and foundations in joint research projects and its faculty have founded several stem cell-related startup companies and serve on leading Scientific Advisory Boards.
Brock came to this role from the commercial sector with extensive experience in both management consulting and operations for technology-based companies, with a focus on life sciences. Brock received a BA and MPhil from Yale University and an MBA from Harvard Business School.
PI, Research Institute at Nationwide Children’s Hospital, USA
Louise is a principal investigator in the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital. She is also an assistant professor in the Department of Pediatrics in the Ohio State University College of Medicine. She received her PhD in molecular genetics and performed her post-doctoral studies developing gene therapy strategies to treat muscular dystrophies. She has a very active laboratory that develops adeno-associated virus based therapies for Duchenne (DMD) and Limb Girdle Muscular Dystrophies (LGMDs). She has over 10 years’ experience in translating benchside efforts to the bedside and has participated in several clinical trials conducted in the Center for Gene Therapy.
Director, Regenerative Medicine Program, University of Ottawa, Canada
Michael Rudnicki is a Senior Scientist and the Director of the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute. He is a Professor in the Department of Medicine at the University of Ottawa. Dr Rudnicki is the Scientific Director of the Canadian Stem Cell Network. Dr Rudnicki received his PhD at the University of Ottawa in 1988 with Dr Michael McBurney where he examined the control of gene expression during embryonal carcinoma cell differentiation. Dr Rudnicki trained at the post-doctoral level at the Massachusetts Institute of Technology in the Whitehead Institute with Dr Rudolf Jaenisch. His post-doctoral studies involved the genetic dissection of the function of the MyoD-family of transcription factors by gene targeting. Dr Rudnicki was appointed Assistant Professor at McMaster University in 1992. He moved to Ottawa In 2000 to join the Ottawa Hospital Research Institute.
Dr Rudnicki's laboratory works to understand the molecular mechanisms that regulate the determination, proliferation, and differentiation of stem cells during embryonic development and during tissue regeneration. The lab has conducted leading studies into both embryonic myogenesis and the function of muscle stem cells (satellite cells) in adult regenerative myogenesis. In particular, they have worked extensively to understand the molecular mechanisms that regulate the function of satellite cells in skeletal muscle. Towards this end, the lab employs molecular genetic and genomic approaches to determine the function and roles played by regulatory factors. They identified Pax7 as a transcription factor required for the specification of satellite cells, and identified Wnt7a signaling as playing an important role in muscle stem cell function.
Chair, Committee for Advanced Therapies, European Medicines Agency (EMA), Finland
Dr Paula Salmikangas is a biochemist by original training, with a Ph.D. in muscle cell biology. Her main research work career has been in cell and molecular biology of various inherited diseases. Since 2006, she has been an Adjunct Professor of Biochemistry for the University of Helsinki.
Paula has worked as a senior researcher at Finnish Medicines Agency, Finland since 2003. Her main areas of expertise are biological medicinal products, especially cell-based medicinal products and combination products. She has been a member and chair of Cell Products Working Party (CPWP) in the European Medicines Agency (EMA) during 2005 and 2012. She has been the Vice-Chair and member of the Committee for Advanced Therapies (CAT) from 2009 to 2014 and since February 2014, the Chairperson of the CAT.
BEATRIZ SAN MARTIN
Partner, Fieldfisher, UKd
Beatriz is an IP specialist, with a focus on patents, Supplementary Protection Certificates (SPCs) and a passion for the life sciences sector. Prior to pursuing a legal career, Beatriz was a scientist with a degree and a Wellcome Trust PhD and postdoc from the University of Cambridge specialising developmental biology. Beatriz has significant experience litigating before the UK Courts and the Court of Justice of the European Union, having acted in a number of leading reported IP cases. Her expertise includes assisting clients to develop, implement and supervise international IP litigation strategies, providing validity and infringement opinions, licensing and coordinating due diligence exercises. Chambers 2015 ranked Beatriz as a leading IP individual "noted for her profound understanding of the life sciences sector. Clients appreciate the sheer depth of her knowledge and her collegiate approach to cases".
Beatriz is a member of the Cellular Therapy and Regenerative Medicine Advisory Committee at the BioIndustry Association. For the past eight years Beatriz has coordinated a series of legal lectures for the University of Cambridge Masters in Bioscience Enterprise and lectures on the course.
Division Head, Lund Stem Cell Center, Lund University, Sweden
Graduate School of Innovation Management, Tokyo Institute of Technology, Japan
Executive Director, Genetics Policy Institute USA
Vice President, Advanced Therapy Delivery, GSK, UK
Jan is Vice President, Advanced Therapy Delivery at GSK and head of Advanced Therapy Delivery. In this role Jan has responsibility for building GSK’s CMC/supply platform for cell and gene therapies, from new technology development through to product commercialization. Prior to establishing the Advanced Therapy Delivery group in 2011 Jan had responsibility for CMC development and commericalisation of a number of biopharmaceutical products, and since joining GSK in 1997 has held a number of roles in both R&D and manufacturing organizations in new technology and new product introduction. Jan holds a Masters in Chemistry and PhD in Biological Chemistry from the University of Oxford.
ADRIAN J THRASHER
Programme Lead, UCL Institute of Child Health, UK
Adrian Thrasher is the Programme Head of the Infection, Immunity, Inflammation and Physiological Medicine Academic Programme at ICH and has a long standing research and clinical interest in development and application of gene therapy. He is Director of the Clinical Gene Therapy Programme, and Theme Leader of the Gene Stem and Cellular Therapies theme of the Biomedical Research Centre, at ICH/GOSH. Adrian is PI on several clinical trials for immunodeficiency and is director of the clinical gene therapy GMP facility, managing a team of trial coordinators, clinical scientists, and quality systems personnel.
GM Cell BioProcessing, GE Healthcare, USA
CEO, BioTime, USA
Mike is the CEO of BioTime, Inc. (NYSE MKT: BTX) and its subsidiaries OrthoCyte Corporation, ReCyte Therapeutics, BioTime Asia, and ES Cell International. The Companies are focused on developing an array of research and therapeutic products using human embryonic stem cell technology.
He received his PhD from Baylor College of Medicine in 1989 concentrating on the biology of cellular aging. He has focused his academic and business career on the application of developmental biology to the age-related degenerative disease. He was the Founder of Geron Corporation of Menlo Park, California (Nasdaq: GERN) and from 1990 to 1998 he was a Director, and Vice President, where he initiated and managed programs in telomerase diagnostics, oligonucleotide-based telomerase inhibition as anti-tumor therapy, and the cloning and use of telomerase in telomerase-mediated therapy wherein telomerase is utilized to immortalize human cells.
From 1995 to 1998 he organized and managed the research collaboration between Geron and its academic collaborators James Thomson and John Gearhart that led to the first isolation of human embryonic stem and human embryonic germ cells. From 1998 to 2007 he was President and Chief Scientific Officer at Advanced Cell Technology, Inc. (OTCBB: ACTC) where he managed programs in animal cloning, human somatic cell nuclear transfer, retinal differentiation, and ACTCellerate, a technology for the multiplex derivation and characterization of clonal human embryonic progenitor cell lines.