Editorial Advisory Board
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
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 500 journal articles and has applied for or received over 250 national and international patents.
Head of Centre for Cardiovascular Science, Gustav Born Chair of Vascular Biology, University of Edinburgh, 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.
Chief Commercial Officer, FloDesign Sonics
With more than 15 years’ experience in the Regenerative Medicine sector, Nina has held Business Development roles at the Cell Therapy Catapult (UK), and the University of Edinburgh, and worked as a Life Science Consultant for a wide range of Regenerative Medicine businesses. Most recently, she was the Head of Lonza’s Autologous Cell Therapy business, with manufacturing sites in the US and Europe. As part of this role, she was also in charge of leading the partnership with Octane Biotech, with the aim of establishing the Cocoon™ as a novel manufacturing technology to remove bottlenecks and enable commercially viable services for patient-scale therapies.
Director, Cell Therapy Processing/Advanced Cell Therapy Labs, Yale University, USA
Alexey received his medical education and was certified as a general surgeon in Russia. After completing medical school, he received his PhD in transplantation/pathology in 2003. His thesis study was focused on the impact of cell transplantation on the early stages of atherosclerosis. Alexey then moved to the US to undertake his postdoctoral training. Over 5 years he was a postdoctoral fellow in two laboratories – Thomas Jefferson University and Children’s Hospital of Philadelphia. During this period he gained expertise in immunology, hematology, stem cell biology and published a number of scientific papers. In 2011, he started to work as a cell processing technologist at the University of Pennsylvania and over the next 3 years he received training in clinical cell processing in a GMP cell manufacturing facility. Currently, Alexey is working at Yale University (Yale-New Haven Hospital) as Facility Director of the Clinical Cell Therapy Processing and Advanced Cell Therapy laboratories. His expertise is in clinical manufacturing of cellular products for clinical trials, including product and process development, cell processing and culture, operations of academic GMP facility and compliance with regulations. He is also an Associate Research Scientist at the Department of Laboratory Medicine at Yale University.
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).
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.
Dean of the Faculty of Health Sciences, Simon Fraser University, Canada
Dr. Bubela was appointed Dean of the Faculty of Health Sciences at Simon Fraser University in September 2017. Prior to that appointment, she was Professor and Associate Dean (Research) in the School of Public Health and Adjunct Professor in Alberta Business at the University of Alberta (UofA), Canada. She joined the faculty of the UofA 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, focused on 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. It addresses barriers to the development and 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 governmental 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, BioCanRx, 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 over 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.
President & CEO, Director, RepliCel Life Sciences, Canada
Lee Buckler 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. Mr. Buckler has a Bachelor’s Degree in Education and a Law Degree. After law school, he did a one year judicial clerkship with the B.C. Supreme Court and was a practicing attorney for three years at Edwards, Kenny & Bray. Mr. Buckler 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. Mr. Buckler serves on numerous industry conference advisory boards, is an advisory board member for BioCision, Phacilitate Cell & Gene Therapy and RoosterBio, and is on the Board of Directors for Hemostemix.
Head of the Hematopoietic Innovative Therapies Division, CIEMAT and the Centre for Biomedical Research on Rare Diseases (CIBERER)
Juan Bueren is the Head of the Hematopoietic Innovative Therapies Division at the CIEMAT and the Centre for Biomedical Research on Rare Diseases (CIBERER). Since 2004 is also the Coordinator of the Advanced Therapies Unit of the Fundación Jiménez Díaz and CIEMAT. Awards of Appreciation and Distinguished Service Award from the Fanconi Anemia Research Foundation (FARF). Former President of the Spanish Society for Gene and Cell Therapy (2011-2013). Board member of the European Society for Gene and Cell Therapy. Member of the Hematologic and Immunologic Gene and Cell Therapy Committee from the American Society for Gene and Cell Therapy. Author of 125 papers in international journals on topics related with hematopoietic stem cells, gene therapy and cell reprogramming.
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.
CSO, BrainVectis, France
NATHALIE CARTIER-LACAVE is founder and CSO of BrainVectis.
Research Director at INSERM, where she leads the group “Biotherapies of neurodegenerative diseases”, Nathalie is president of the European Society of Gene and Cell Therapy (ESGCT).
Her research led to the first gene therapy clinical trial in humans, using HIV as a gene transfer vector (Science 2009). Thanks to this trial, four children suffering from adrenoleukodystrophy were cured.
She has been interested in the link between cholesterol and neurodegenerative diseases for 10 years.
Professor of Hematology at Paris Descartes University, Head of the Department of Biotherapy at Hospital Necker-Enfants Malades, AP-HP and Chief of Research of the Laboratory of Human Lympho-hematopoiesis U1163 Inserm in the Institut Imagine in Paris
Marina is a Professor of Hematology at Paris Descartes University, Head of the Department of Biotherapy at Hospital Necker-Enfants Malades, AP-HP and Chief of Research of the Laboratory of Human Lympho-hematopoiesis U1163 Inserm in the Institut Imagine in Paris. She also serves as Coordinator of the Biotherapy Clinical Investigation Center at Necker-Enfants Malades, hospital from AP-HP, Greater Paris University Hospitals and Inserm. She completed her residency at the University of Padua in Italy and obtained her PhD in blood cell biology at Paris VII University.
Pr. Cavazzana has made seminal discoveries that have led to clinical trials with lentiviral vector gene modified cells for treatment of nonmalignant hematologic diseases. She has spearheaded translational and clinical advances achieved in HSC gene therapy for multiple genetic immune, hematopoietic, and metabolic disorders. She is well known for her work to design, lead, and implement a clinical trial of retroviral gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare, inherited disorder of the immune system. In 2002, Pr. Cavazzana reported the first benefit in a human gene therapy trial when she demonstrated reconstitution of the immune system in young boys with X-SCID. When it was discovered that two of these boys developed leukemia from the therapy, Pr. Cavazzana coordinated the efforts of a highly skilled group of clinicians, virologists, molecular biologists, and regulators to develop novel therapies. In addition to this work, in 2017 she published the proof of principle that using a lentiviral vector expressing an anti-sickling beta chain into the hematopoietic stem cells provides a stable clinical benefit to a patient with sickle cell disease.
Pr. Cavazzana is the author of more than 240 publications in peer-reviewed journals and was awarded two European Research Council grants (2011 and 2016). Her work was rewarded by the American Society of Hematology (Award on Clinical Research in Gene Therapy in 1999), by the French Academy of Sciences (Special Medical Award in 2000 and Jean-Pierre Lecocq Award on Gene Therapy in 2004). She was awarded with the Matmut Prize for medical innovation in 2008, the Outstanding Achievement Award of the European Society of Gene and Cell Therapy in 2009, the title of Officier de l’Ordre National de la Légion d’honneur in 2011, and the Irène Joliot-Curie “Scientific Woman of the Year” award in 2012 by the French Academy of Sciences, French Ministry of Education and Research and EADS group corporate foundation. In 2016, she was awarded the French National Medicine Academy Prize.
She currently serves as a member of UNAIDS Science Panel and has previously served as Chairwoman of the Scientific Committee for Strategies and Evaluation of Clinical Research Protocols (INSERM). She has also served as a member of the Blood editorial board.
Vice President, Regulatory CMC, Orchard Therapeutics
Michael is an expert in the human therapeutic biotechnology and biologics fields in areas including Regulatory Affairs, Quality, Compliance, and Manufacturing. He has provided leadership for companies developing recombinant proteins, monoclonal antibodies, cellular, and genetic therapies primarily for the treatment of Cancer and Rare Diseases including Amgen, Dendreon, and Juno Therapeutics. Michael is currently the Vice President of Regulatory CMC for Orchard Therapeutics.
Programme Director – Science Advice and Research, National Institute for Health and Care Excellence (NICE)
Nick had a 20-year career in analytical science, process technology and general management in the chemical, pharmaceutical and contract laboratory industries prior to joining NICE in 2010 as the associate director responsible for establishing and managing the Diagnostics Assessment Programme. In 2014 Nick was appointed to his current role where he oversees NICE Scientific Advice, the Science Policy and Research programme and NICE’s input to the European Network for Health Technology Assessment (EUnetHTA). Nick has broad scientific and policy interests relating to the evaluation of technologies and interventions to support the development of clinical, public health and social care guidance. His experience includes consideration of HTA issues arising from the availability of novel new products such as cell and gene therapies and work on methods issues relating to the evaluation of antimicrobials. Nick was also the co-chair of the evaluation and commissioning subgroup of the UK regenerative medicine expert group and led NICE’s contribution to a project on the assessment and appraisal of regenerative medicines that reported in 2016.
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.
Chief Technology Officer, BlueRock Therapeutics, USA
Robert Deans, Ph.D., serves as BlueRock’s CTO, where he is responsible for implementing BlueRock’s novel induced pluripotent stem cell production platform. He has more than 25 years of experience in stem cell therapeutics, including hematopoietic as well as mesenchymal stem cell (MSC) cells and gene therapy approaches, as well as translational science and global regulatory expertise. Dr. Deans previously served as CSO of Rubius Therapeutics, where he was responsible for development of a gene-engineered red cell therapeutics platform. Prior to Rubius, he was an EVP at Athersys, where he helped advance numerous adult adherent stem cell therapeutics into late-stage clinical development. Dr. Deans also served as VP of research at Osiris Therapeutics, where he developed Prochymal, which achieved clinical approval in some geographies. Before that he was director of R&D at Baxter Healthcare where he developed biologics, including a clinical-stage retroviral gene therapy. He holds a B.S. from the Massachusetts Institute of Technology and a Ph.D. in microbiology from the University of Michigan. Dr. Deans founded and chaired the International Society for Cellular Therapy’s Commercialization Committee and chaired the Science and Technology Committee of the Alliance for Regenerative Medicine.
Founder and Chief Scientific Officer, Executive Vice President of Research and Development, Nohla Therapeutics
Colleen Delaney is the Founder, Chief Scientific Officer and EVP of Research and Development at Nohla Therapeutics. She is also a Professor at the UW Department of Pediatrics, Division of Pediatric Hematology/Oncology, and was a Member and recipient of the Madeline Dabney Adams Endowed Chair of the Fred Hutch Clinical Research Division. In addition, Colleen is a member on the Advisory Council on Blood Stem Cell Transplantation of the Health Resources and Services Administration. She also serves on the Boards of Directors of the American Society of Bone Marrow Transplant and the Cord Blood Association. In 2006, she established and became the Director of the Cord Blood Transplant Program at the Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, which has grown to be one of the busiest such programs in the country and the coordinating center of several multicenter clinical trials. She is the sponsor-investigator of three Investigational New Drug FDA files and has been the Principal Investigator of nine ongoing and completed clinical trials and several ancillary studies.
Chief Technical Officer, Semma Therapeutics
David is the Chief Technical Officer for Semma Therapeutics. He has over 28 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, T-cells and regenerative medicine products for clinical applications. He has been instrumental in the creation of seven 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. David 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. David is a member of the NIH recombinant DNA advisory committee (RAC) and an independent consultant to the Biotechnology industry.
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).
University Lecturer in the Immunobiology of Stem Cells and Oxford Martin Senior Fellow, Oxford University, UK
Paul Fairchild was Co-Director of the Oxford Stem Cell Institute from 2008-2015, which was funded by the Oxford Martin School during this period. He remains connected with the School through his role as an Oxford Martin Senior Fellow.
Paul’s research addresses the immunological barriers to stem cell therapies.
After obtaining a first class degree and an award for top graduate in the Biological Sciences, Paul Fairchild began his research career in Oxford, where he studied for a DPhil within the Nuffield Department of Surgery, focussing on the immune response to organ allografts. After spending five years as a Post Doctoral Fellow in the Department of Pathology, University of Cambridge, he returned to Oxford where he is currently a lecturer and RCUK Academic Fellow within the Sir William Dunn School of Pathology. Here he has applied his immunological training to the emerging field of cell replacement therapy and regenerative medicine to investigate the immune response to tissues differentiated from embryonic stem cells, the rejection of which threatens to undermine the success of regenerative medicine in the future. He has developed strategies which may one day promote the indefinite survival of stem cell-derived grafts and is currently collaborating with Geron Corporation to bring such technology to the clinic.
Head of the Research Dept. Cell and Gene Therapy, University of Hamburg, Germany
Prof. Boris Fehse heads the Research Unit Cell and Gene Therapy at the University Medical Center Hamburg-Eppendorf and is one of the authors of the genome surgery analysis published by the BBAW in 2015.
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
Dr Richard Harbottle is currently an Independent Group Leader of Gene Therapy Research in the section of Molecular Medicine at Deutsches Krebsforschungszentrum (DKFZ). The Cancer Research Centre based in Germany. Previously Richard was at Imperial College London where his key research focused on the development of DNA technologies for gene therapy, with a particular focus on the application of scaffold/matrix attachment region (S/MAR) vectors.
His group utilises state-of-the-art techniques in molecular and cellular biology and bio-imaging with an emphasis in gene delivery to pre-clinical model systems. A particular focus of his group is the development of genetically marked tumour models, which can be used to investigate the molecular mechanisms of tumour formation as well as simplifying the validation of therapeutic intervention.
Richard is a also a founding member of the British Society for Gene Therapy and served as an elected member of its executive board since the inception of the society in 2003 until 2010. He is an active member of the society and currently contributes as a co-opted member of the executive. He is also an active member and participant of the American and European gene therapy societies and the American Association of Cancer Research.
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.
VP, Process Development, Evox Therapeutics
Bo has over 30 years’ experience in the biotechnology industry. He has expertise in the development of CMC for a wide range of protein, viral vector and cell therapy medicinal products.
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, HairClone, UK
Paul is a recognised pioneer in the field of Regenerative Medicine and has over 25 years’ experience in the US and UK in the commercialisation of various Regenerative Medicine and Cell Therapy products in both public and private Companies. Over the years, he has built a successful track record in all aspects of the business, from carrying out translational research, in-licensing technologies from Universities and out-licensing products to a variety of HealthCare Companies, designing, obtaining regulatory approval and running clinical trials in the US, Canada, UK and Europe, designing, building and operating GMP facilities, founding biotech companies, and launching, marketing and selling cell based products in the US and UK. When at Organogenesis Inc he was the lead inventor on patents for the first multi-cell therapy approved by the FDA and was involved in all stages of the development of this product from the bench, through the clinical trial process, development of a licensing and marketing partnership and the eventual approval and launch in the US. This product has now treated well over half a million patients and is the largest selling manufactured cell therapy in the world. After Organogenesis Paul returned to the UK and was the sole founder of Intercytex. He served as CEO for the first 5 years and under his leadership the company grew rapidly, attracted significant VC investment from Europe, the US and Asia. During this period Intercytex developed several products that reached the clinic, including the world’s first cell therapy clinical trial to induce new hair formation and carried out with Bessam and Nilofer Farjo. Paul has numerous patents and publications in the field and is often invited to speak at International Conferences and sit on a variety of panels, grant funding bodies and regulatory committees. He is co-editor of the Journal of Regenerative Medicine, a co-Director of the Doctoral Training Centre in Regenerative Medicine at Manchester University and Industrial Director of Regener8.
Principal, Sven Kili Consulting Ltd. UK
Dr Sven Kili, is Principal at Sven Kili Consulting Ltd. Where he provides specialist strategic consulting services to innovative Cell & Gene Therapy companies. His clients include small and medium sized companies from formation through to clinical development and commercialisation. He was previously the Head of Development for the Cell and Gene Therapy division of GSK Rare Diseases where he led teams developing and commercialising exvivo Gene Therapies for a variety of rare genetic disorders including Strimvelis®, the first ex-vivo gene therapy to be approved for children with ADA-SCID; Wiskott – Aldrich syndrome (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, approval and commercialisation activities of the first combined ATMP approval in the EU for MACI®. He and his team also prepared and submitted Advanced Therapy regulatory filings for Australia and the US, including health technology assessments and he was responsible for late stage developments for Carticel® and Epicel® in the US. Before joining Genzyme, Sven led the cell therapy activities and oversaw all UK & Irish regulatory functions and was the QPPV for pharmacovigilance for the Geistlich Pharma. 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 is on the board of CCRM in Canada; Xintela – a Swedish Stem Cell company, the SAB for LGC Corporation and is the chair of the CGTAC as part of the UK BIA and a Board member of the Standards Co-ordinating Body for Regenerative Medicine. Most recently, Sven was appointed as Chair of the UCL course “Masters in Manufacture and Commercialisation of Stem Cell and Gene Therapies” steering committee. Additionally, he still maintains his clinical skills in the UK NHS and serves as an ATLS Instructor in his spare time.
Co-Founder & VP, Discovery Research, Carisma Therapeutics
Michael is a co-inventor of the CAR Macrophage technology and a scientific co-founder of Carisma Therapeutics Inc. In his role as VP of Discovery Research, he oversees the research & discovery efforts of the company.
Michael developed CAR Macrophages during his doctoral thesis under the co-mentorship of Saar Gill and Carl June at the University of Pennsylvania. Michael’s scientific expertise is in the intersection of immunology, synthetic biology, cancer immunotherapy, and translational pharmacology.
Michael previously earned a Doctor of Pharmacy degree from the University of Sciences in Philadelphia, and a PhD in Pharmacology from the University of Pennsylvania.
Chief Scientific Officer, Co-Founder, SwanBio Therapeutics
Karen Kozarsky, Ph.D., has 25 years of experience in gene therapy, with a primary focus on the development of gene therapy for inherited rare diseases utilizing adeno-associated virus (AAV) vectors.
Dr. Kozarsky has deep experience evaluating potential therapeutic opportunities, identifying new areas, and developing products from the earliest preclinical stages through IND. She has been involved in the development of multiple gene therapy products that are in clinical trials.
Previously, Dr. Kozarsky was President of Vector BioPartners, VP of R&D at REGENXBIO Inc., and Head, Gene Therapy in the GlaxoSmithKline Biopharmaceutical Center of Excellence for Drug Discovery. Prior to that, she was a Research Assistant Professor at the University of Pennsylvania in the Institute for Human Gene Therapy, and completed postdoctoral fellowships at the University of Michigan in gene therapy and in immunology.
Dr. Kozarsky was named by the Alliance for Regenerative Medicine as the first co-Chair of the Gene Therapy Section to support policies to advance novel gene therapies, and has been a committee member for the American Society of Gene & Cell Therapy.
Dr. Kozarsky received a Ph.D. in Biology from the Massachusetts Institute of Technology and a B.A. in Biology from Amherst College.
Director, Carolinas Cord Blood Bank, Duke University, USA
Dr Joanne Kurtzberg is an internationally renowned expert in pediatric hemtaolgoy/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Dr Kurtzberg pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT). Over the last two decades she has established an internationally known pediatric transplant program at Duke University, which treats children with cancer, blood disorders, immune deficiencies, hemoglobinopathies and inherited metabolic diseases. In 2010 Joanne established the Julian Robertson Cell and Translational Therapy Program (CT2) at Duke. CT2 focuses on translational studies from bench to bedside with a focus on bringing cellular therpaies in regenerative medicine to the clinic. Recent areas of investigation in CT2 include the use of autologous cord blood in children with neonatal brain injury and cerebral palsy, as well as preclinical studies manufacturing oligodendrocyte-like cells from cord blood to treat patients with acquired and genetic brain diseases. Studies of autologous bone marrow ALDHbright cells in adults with stroke, radiation-induced brain injury are also underway. Plans are ongoing to extend this work to the use of autologous cord blood in the study of young children with autism specrum disorder.
CHRISTINE LE BEC
Head of CMC Gene Therapy, Sensorion
Christine Le Bec joined Genethon in 1997 as a scientist and currently heads the CMC Analytical Department. She is responsible for the analytical activities in the characterization and release testing of gene therapy products at early stage development, stability studies, and interface with CMO for method transfer and validation, analytical/QC testing. She also assists in the response to CMC questions from regulatory agencies query. She has a strong expertise in the development and qualification of analytical methods based on biochemical, biophysical and cell based assays to assess identity, potency, impurity profile, and safety. Before joining Genethon, she obtained her PhD in Bio-Organic Chemistry from Université Pierre et Marie Curie (Paris VI) in 1993. She worked as a postdoctoral researcher at Thomas Jefferson University (Philadelphia, US) and then at Institut Pasteur (Paris, France) in the field of synthesis, structural analysis and in vitro evaluation of antisense DNA as therapeutic agents for cancer and AIDS.
Scientific Program Manager, Trans-NIH Tissue Chip for Drug Screening Program, National Center for Advancing Translational Sciences (NCATS), NIH
Lucie Low is the scientific program manager of the trans-NIH Tissue Chip for Drug Screening program at the National Center for Advancing Translational Sciences (NCATS). She also serves as the NIH-NASA liaison point of contact, facilitating collaborations between NIH and NASA on areas of overlapping agency interest. Prior to joining NCATS in 2016, Low was an intramural research fellow at the National Center for Complementary and Integrative Health (NCCIH), researching the complex interactions between pain, emotion and cognition. She obtained her undergraduate degree from Magdalen College, Oxford University, and her Masters and PhD degrees in Neuroscience from University College London in the UK. She undertook postdoctoral training at McGill University in Montreal, Canada, before joining the US National Institutes of Health in 2012. She has authored over 20 peer-reviewed manuscripts on the neuroscience of pain and tissue chips.
Senior Vice President, Manufacturing and Supply Chain, Adaptimmune
John Lunger is Senior Vice President, Manufacturing and Supply Chain at Adaptimmune, having joined the company in March of 2017. John is responsible for clinical manufacturing and global supply of Adaptimmune’s autologous T-cell therapy products. From 2015 through 2016 John was Head of Supply Chain and Commercial Product Supply at Merrimack Pharmaceuticals where he led clinical and commercial supply chain as well as the cross functional supply team for Onivyde, Merrimack’s first commercial product launched in October of 2015. Earlier in his career, he held various senior manufacturing, operational, and strategy roles with VWR International, Pfizer, and Wyeth Pharmaceuticals. In his nearly 10 years with Wyeth he had a number of leadership positions, including operations and supply chain strategy, supply management, procurement and strategic sourcing, business systems implementation, generic pharmaceutical business management, marketing, and site operations management in a pharmaceutical manufacturing plant in Ireland. John began his career serving as a nuclear trained officer on a U.S. Navy submarine, strategic supply chain consulting with Accenture, and business development with a start-up procurement services company.
John holds a Bachelor of Science degree (with distinction) in Ocean Engineering from the U.S. Naval Academy and an MBA in economics and operations management from the University of Chicago’s Booth School of Business.
TAMARA J. LASKOWSKI
Senior Research Scientist – Immunotherapy, Allison Group Department of Immunology, MD Anderson Cancer Center
Dr. Tamara J. Laskowski received her doctorate degree in Human Molecular Genetics and Immunology from University of Texas Health Science Center where her work focused on genome editing of patient-derived stem cells to correct genetic mutations causative of Wiskott-Aldrich Syndrome, an immunodeficiency disorder that results in severe impairments to the immune system.
Subsequently, Tamara joined Dr. Laurence J.N. Cooper’s laboratory at MD Anderson Cancer Center as a fellow. Her work focused on engineering stem cells with the goal of generating off-the-shelf NK and T-cell immunotherapies for targeting solid tumor malignancies. Though Tamara remains as a collaborator in the Cooper laboratory, her work has led her to transition to Dr. James Allison’s Immunotherapy Platform at MD Anderson. In her new role as Senior Research Scientist, Dr. Laskowski’s work primarily involves immune-monitoring of patients undergoing clinical trials in Immunotherapy and development of novel immunoassays. Dr. Laskowski also shares an interest in technology innovation, and has developed multi-plex assays for testing therapies against solid tumors. In 2017, this work led to an invitation to participate in the National Science Foundation (NSF) Innovation Corps, an exclusive program through which she was trained to develop strategies for expanding the economic and societal benefits of innovative ideas which have commercialization potential. She was the sole recipient of an award for outstanding performance upon completion of the program.
In addition to her research work, Dr. Laskowski participates in several educational programs at MD Anderson. She is a lecturer at MD Anderson’s School of Health Professions and also serves as a scientific communications coach teaching students and fellows how to effectively communicate science and medicine with scientific and lay audiences.
CSO and founder, InMuneBio, 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. Mark has been a Chief Scientific Officer and Chief Manufacturing Officer since the formation of the InMuneBio in September 2015. Since February 2009, he has also been Director of Cellular Therapy at the Royal Free London NHS Foundation Trust. He received his PhD in clinical immunology from London Hospital Medical College, University of London in 1992 and is a qualified immunopathologist.
CEO, Avrobio, USA
Geoff is an experienced CEO with proven success leading innovative businesses. While CEO of Organogenesis Inc., the company treated 1 million patients with living cell therapies, received the first FDA CBER allogeneic cell-therapy approval and achieved an unparalleled position within regenerative medicine. Geoff was founding CEO of eGenesis, applying CRISPR Cas-9 gene editing to xenotransplantation. Geoff spent 11 years at Novartis in senior leadership positions within the Global Transplantation & Immunology franchise. Past activities include Chairman of the Board of MassBio, Chairman of the Board of the Alliance of Regenerative Medicine, and Advisory Council to the Health Policy Commission for Massachusetts. Through his public and private enterprise work, Geoff is dedicated to applying innovation to reshape and improve the quality of healthcare.
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.
Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania
Dr. Jan Joseph (Jos) Melenhorst obtained his PhD at the LUMC (Department of Hematology) on the pathogenesis of Aplastic Anemia. In 1998 he moved to Bethesda, Maryland, where he did his research ‐ first as a postdoc, later as a staff scientist ‐ in the laboratory of Dr. John Barrett at the National Institutes of Health, on the immunobiology of marrow failure syndromes, leukemic disorders, and allogeneic stem cell transplantation. In 2012 he was recruited by Dr. Bruce Levine and Dr. Carl June to the University of Pennsylvania, first as Deputy Director of their clinical manufacturing (cGMP) facility. After a year he was promoted to Director of Product Development & Correlative Sciences. In this role, he was at the cusp of the first ever CAR T cell therapy approved by FDA: Kymriah. Dr. Melenhorst is interested in understanding and improving the anti-tumor efficacy and safety of adoptively transferred chimeric antigen receptor-modified T cells through correlative, mechanistic, and functional genomics approaches.
Professor of Clinical Cell and Gene Therapy, 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.
ATMP & Vaccine Scientific Programme Manager, Translational Medicine & Drug Development, EATRIS
Dr David Morrow is the ATMP and Vaccine Scientific Program Manager at EATRIS C&S. David received a BSc (Hons) in Molecular Biology from University College Dublin in 2001 and a PhD in Vascular Biology from Dublin City University in 2006. This was followed by an American Heart Association Postdoctoral Fellowship at the University of Rochester Medical Center, NY from 2006-08 which resulted in an American Heart Association Young Investigator Award in 2008. From 2009-2015 he was an NIH/American Heart funded Principal investigator heading multiple projects in Cardiovascular disease and Cancer. David also holds an MBA in health science management and has worked as a consultant and in technology transfer in lifesciences. David has recently authored papers and being invited as guest speaker at panel discussions at international conferences discussing the major bottlenecks in ATMP development.
Senior Director, Viral Vector and Gene Editing Process Development, Juno Therapeutics, a Celgene Company
John joined Celgene in 2018 and serves as the Senior Director of Viral Vector and Gene Editing Process Development. Prior to joining Celgene, John was the Vice President of Process Development at AGC Biologics (formerly CMC Biologics) where his organization was responsible for all cell line development, upstream and downstream process development, analytical and formulation development, and technology transfer and technical manufacturing support. Prior to AGC Biologics, John held director-level positions at Amgen, focused on process development and characterization, clinical and commercial technology transfer and process validation. John has a Ph.D. in chemical and biological engineering from the University of Wisconsin-Madison and a bachelors of chemical engineering from the University of Delaware.
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.
Director, AMBR-Consulting; Former Senior Quality Assessor for Biologics MHRA; Former Member, Committee for Advanced Therapies (CAT), EMA
Christiane has spent 12+ years in medicines, tissue, cell and gene therapy regulation. For the last 8 years she has worked at the MHRA, the UK’s medicine regulator, where she has assessed all classes of biological medicines at both UK and EU level and acted as the UK representative at the European Medicines Agency’s (EMA) committee for advanced therapies (CAT). Christiane was the Rapporteur or Co-Rapporteur for several advanced therapy medicinal products (ATMPs) and many other biological product applications. She acted as the Rapporteur for the EMA Gene Therapy Guideline and was a drafting group member for other CAT guidelines. As acting Manager for the Biologicals Unit, Christiane was actively involved in the MHRA’s Brexit preparations and has in-depth knowledge of the MHRA no-deal provisions.
Prior to joining the MHRA, Christiane worked for the UK competent authority responsible for the European Tissues and Cells Directive.
Christiane received a Ph.D. in molecular developmental neurobiology from Kings College London and has spent several years researching and publishing in the area of cancer, embryonic development and neurobiology. Before joining the public sector, Christiane has also taken a law degree.
Christiane recently left the public sector to become an independent regulatory consultant.
Senior Vice President, Head of Technical Operations, Sarepta Therapeutics
Palani has over twenty-five years of pharmaceutical experience and continues to contribute to commercial registration and clinical development of numerous candidates in small molecule, biologics, ADCs and cell therapy. He currently serves as the head of Technical Operations at Sarepta Therapeutics and focuses on small molecule and antisense based RNA medicines. He is currently leading Sarepta’s gene therapy CMC and Manufacturing team and is responsible for all stages of development from research through commercial. Prior to Sarepta, he held increasing leadership positions and recently led biologics and new modality development at Takeda Pharmaceuticals with responsibility for global development of drug candidates in variety of modalities including cell and gene therapy, microbiomes and antibody drug conjugates, etc. Before Takeda, he spent a number of years each at Millennium Pharmaceuticals, Biogen, Gilead Sciences and Par Pharmaceuticals. Palani completed executive leadership education at Oxford University and Insead, post-doctoral work at University of California Riverside and Virginia Commonwealth University Richmond. He received his PhD from Indian Institute of Technology Kanpur.
ATMP Research coordinator, Prometheus, Division of Skeletal Tissue Engineering Skeletal Biology and Engineering Research Center, KU Leuven, Belgium
Ioannis is a Chemical Engineer by training (graduated from the University of Patras, Greece)having obtained his PhD at Department of Biochemical Engineering, University College London funded by the National Scholarship Foundation of Greece. Subsequently he joined KU Leuven where he obtained funding to initiate an autonomous research track and is currently ATMP Research coordinator, within Prometheus – the KU Leuven division of skeletal tissue Engineering. He is active in addressing bioprocessing/translational challenges that would allow scalable and robust manufacturing of adult progenitor/stem cells, required for moving ATMPs for skeletal healing into the clinic. His task is to promote interfaces with industrial partners aiming at valorising research solutions generated within the platform along the entire ATMP bioprocess pipeline and has initiated several collaborations at the academia/industry interface. His research aims are to: Design and engineer progressively complex yet autonomous skeletal 3D living implants adopting breakthroughs in organoid technologies (Tissue engineered ATMPs); Develop a panel of metrics to enable quantitative definition of biologic events ensuring activation and maintenance of mechanism of action; Link therapeutic functionality upon implantation to the patient to in vitro quality attributes of skeletal ATMPs; Develop automated devices that can contribute to a cell therapy industry 4.0 equivalent. Integrate in silico derived tools for a self-regulated/adaptive manufacture of next generation living implants.
MARIA DEL PILAR REDONDO
Site Head, Takeda Madrid, Cell Therapy Technology Center
Chief Medical Officer / Chief Development Officer, Medigene, Germany
Dr. Kai Pinkernell is responsible for the clinical advancement of Medigene’s immunotherapy platforms since February 2016. He has acquired long-term expertise in worldwide clinical development, clinical marketing and clinical sales as well as automation and GMP production of cellular therapies. Prior to joining Medigene, he held leading positions at Miltenyi Biotech GmbH, Bergisch Gladbach, Germany, most recently as Global Head of Clinical Business and Head of Clinical Development. Previously, Dr. Pinkernell was with Cytori Therapeutics Inc., San Diego, USA, as Senior Director of Regenerative Cell Technology. He studied medicine and received his MD from the Westfaelische-Wilhems University in Muenster, Germany. Furthermore, Dr. Pinkernell holds a Master of Business Administration from the Marshall School of Business at the University of Southern California, Los Angeles, USA.
Fellow, National Institute of Standards and Technology (NIST)
Dr. Plant was recently awarded the title of NIST Fellow, and previously served as Chief of the Biosystems and Biomaterials Division at National Institute of Standards and Technology, an agency of the U.S. Department of Commerce. She has served in the White House Office of Science and Technology Policy and on National Science and Technology Council committees, co-founded the ASTM International Committee F04.46 on Standards for Cell Signaling, and is currently an ex officio member of the NIBIB National Advisory Council for Biomedical Imaging and Bioengineering. She is a Fellow of the American Institute for Medical and Biological Engineering, and an American Association for the Advancement of Science Fellow. Her research interests focus on robust quantification of cell response through quantitative cell imaging, and theoretical approaches for prediction of complex biological response.
CARLOS R. PLATA SALAMAN
Chief Scientific Officer and Chief Medical Officer, Esteve
Dr. Plata-Salamán leads Esteve’s Scientific and Medical Organization responsible for the discovery and development of first-in-class NMEs and ATMPs as well as the regulatory and medical units including Drug Discovery, Preclinical Development, Clinical Investigation, Drug Safety & Pharmacovigilance, Medical Affairs, Regulatory Affairs, Scientific Quality Assurance, Global Pharmaceutical Quality System, Portfolio & Project Management, and Intellectual Property.
Prior to Esteve, Dr. Plata-Salamán was the Head of Global External R&D at Eli Lilly and Company where he designed and implemented a new worldwide organization that among its responsibilities was the due diligence of internal Lilly projects and external partnership, licensing and acquisition opportunities (molecules, technologies, companies) across all therapeutic areas and at any stage of development.
At Johnson & Johnson, Dr. Plata-Salamán was Head of Drug Discovery Unit, and Core Member of the Executive Neuroscience Franchise Team and Global Neurology, Psychiatry and Pain Product Teams; he was recipient of the Standards of Leadership Award.
Prior to Johnson & Johnson, Dr. Plata-Salamán was a Tenured Full Professor of Neuroscience and Molecular Biology at the University of Delaware. He is the author of 380 publications and inventor in 200 patents & patent applications worldwide, has chaired Committees on Promotion and Tenure, and has served as reviewer for Granting Agencies and on Study Sections/Panels, Editorial and Advisory Boards. Dr. Plata-Salamán is recipient of various Research, Academic & Scholar Recognitions, First-Class Honors for Clinical Work, and was invited to present at a Nobel Conference in Stockholm sponsored by the Nobel Foundation.
Dr. Plata-Salamán received his BSc from the English College in Guadalajara, his MD from the University of Guadalajara Medical School in México, and his DSc in Medical Sciences from Kyushu University Faculty of Medicine in Japan.
Senior Lecturer in Bioprocessing of Regenerative, Cellular and Gene Therapies, Dept. of Biochemical Engineering, Faculty of Engineering Science, University College London
Dr Qasim Rafiq is an Associate Professor at University College London in Cell and Gene Therapy Bioprocess Engineering. He is a multidisciplinary engineer working at the life science, engineering and commercial interfaces with a research focus on the bioprocessing, automation and biomanufacture of cell and gene-based therapies. He has specific interest in addressing the large-scale manufacturing challenges and enhancing process and product understanding to enable successful translation from the laboratory to the clinic.
Qasim currently leads a research portfolio of > £2.5M as Principal Investigator and leads the Cell and Gene Therapy Bioprocess Engineering group at UCL which includes 4 post-doctoral researchers and 7 PhD students. Projects focus on addressing both upstream and downstream process development challenges for the production of patient-specific and universal donor ATMPs for a range of clinical applications. Product applications include stem cells (mesenchymal, haematopoietic and pluripotent), extracellular vesicles and gene-modified T-cells.
He is also Programme Director of a new MSc programme launching in September 2020 entitled “Manufacture and Commercialisation of Stem Cell and Gene Therapies”.
Prior to joining UCL, he was an Assistant Professor at Aston University where he established the Bioprocess Engineering Group. He completed his PhD at Loughborough University investigating the scalable manufacture of human mesenchymal stem cells in bioreactors where he was the first to demonstrate the litre-scale production of hMSCs on microcarriers in stirred-tank bioreactors.
He is both a Chartered Engineer (CEng) and Chartered Scientist (CSci) and sits on multiple scientific and engineering committees including the IChemE Biochemical Engineering Subject Interest Group, British Standards Institute Biotechnology Committee and the BIA’s Cell and Gene Therapy Advisory Committee.
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.
Director of Biopharmaceuticals and ATMP, NDA Group AB
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 previousuly served as Adjunct Professor of Biochemistry for the University of Helsinki.
Paula worked as a senior researcher at Finnish Medicines Agency, Finland. 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, Arnold & Porter, UK
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.
Graduate School of Innovation Management, Tokyo Institute of Technology, Japan
DR STEFAN SCHEDING
Professor and Senior Consultant in Hematology, Division Head, Lund Stem Cell Center, Lund University
Stefan Scheding, MD, is a professor and senior consultant in hematology at the University Hospital in Lund, Sweden. He has a long-standing experience in stem cell research and transplantation as well as a track record of establishing and leading clinical stem cell transplantation laboratories. Currently, he is heading the “Bone Marrow Stem Cell and Cellular Therapies” research group at the renowned Lund Stem Cell Center, and he is acting head of the Stem Cell Center division. Dr. Scheding is representing the Southern Swedish Medical Region in the Swedish Cell Therapy Project (Vävnadsrådet) and he is currently the vice chair of the board and WP leader in the Swedish National ATMP Consortium, CAMP.
Executive Director, Genetics Policy Institute USA
Bernard Siegel is the founder and Executive Director of Genetics Policy Institute (GPI), a nonprofit organization with offices in Palm Beach, Florida and Palo Alto, California. Mr. Siegel received his undergraduate and law degrees from the University of Miami (BA 1972, JD 1975). He is a member of the Florida Bar since 1975. He first became involved with the scientific community when, in 2002, he filed a landmark legal case seeking a guardian for an alleged human clone, a case that was widely credited for exposing the clone as a sham. That experience inspired Mr. Siegel to trade his 30-year courtroom career to found GPI, which leads a global “Pro-Cures Movement”. Mr. Siegel is a frequent speaker and consultant on the subjects of stem cells, public policy, patient advocacy and the societal implications of longevity. Mr. Siegel has served on the governing boards for the Coalition for Advancement of Medical Research (CAMR), Americans for Cures Foundation, and the Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine. He has also served on committees for the Alliance for Regenerative Medicine (ARM) and the International Society for Stem Cell Research (ISSCR). He serves on Advisory Panels for the New England Regional Spinal Cord Injury Center at Boston medical Center and Sabrina Cohen Foundation for Stem Cell Research. He is a member of the American Society for Bioethics + Humanities and the International Society for Cell Therapy (ISCT).
Senior Associate Scientist, Vector/Cellular Process Development, bluebird bio
Nolan graduated from the University of Massachusetts, Amherst with a BS in Biochemistry and Molecular Biology where his research areas of focus were on molecular cloning and RNAi in trypanosomes in the Klingbeil lab. Over the past ten years he has gained extensive experience working in both Upstream and Downstream Process Development roles, getting his start in the area of enzyme replacement therapies before moving into the gene therapy field at bluebird bio. Working in the Vector Process Development group, most of his time has been spent developing suspension-based lentiviral vector production systems for commercial manufacturing. This includes early to late stage process development at the miniature and bench-scales, nanoparticle analysis and characterization, as well as process scale-up and technology transfers. Nolan was recently awarded an Outstanding Poster Award at ASGCT 2019 for his work adapting a transient transfection process onto the Sartorius Stedim ambr® 15 system.
When not focusing on vector process development, Nolan enjoys life on the south shore of the Boston area with his wife and their dog.
Chief Development Officer, Freeline Therapeutics, UK
Jan Thirkettle is Chief Development Officer of Freeline. Previously he was VP, Cell & Gene Therapy Platform at GSK where he built GSK’s Chemistry, Manufacturing and Control (CMC)/supply capabilities in the cell & gene therapy field, enabling the submission of a Marketing Authorisation Application (MAA) for an ex vivo gene therapy for ADA-SCID in 2015 and establishment of a portfolio of technologies and programmes. Prior to this, Jan was responsible for CMC and supply of a number of late/launch stage biopharmaceuticals. He holds an MA in Chemistry and a PhD in Biological Chemistry from Oxford University.
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.
PROFESSOR MONDHER TOUMI
Public Health Department – Research Unit EA 3279, Aix Marseille University
GM Cell BioProcessing, GE Healthcare, USA
Phil is General Manager of GE Healthcare’s cell therapy business, an initiative funded in part by GE Healthymagination, a $6 billion strategy to revolutionize the world’s health by improving the quality, access and affordability of care. Prior to joining GE, Phil was Head of Innovation for Lonza’s Pharmaceutical division, leading a group of research scientists, process development engineers, and commercial strategists to drive new technology initiatives focused on cell, protein, and viral therapeutic manufacturing.
Phil’s career has included a number of senior innovation, business and market development roles at Becton Dickinson, Invitrogen, and Life Technologies, as well as two start-up biotechnology companies in the Washington, DC area.
Phil received his Ph.D. in Biochemistry and Molecular Biology from Georgetown University Medical Center and subsequently held an IRTA fellowship at the National Cancer Institute in the Laboratory of Molecular Oncology.
DR XIUYAN WANG
Associate Director, Michael G.Harris Cell Therapy and Cell Engineering Facility, Associate Laboratory Member, Department of Molecular Pharmacology, Memorial Sloan Kettering Cancer Center
Dr. Wang was trained by Dr. Adolfo García-Sastre and received her Ph.D. in Biomedical Sciences from the Mount Sinai School of Medicine in New York. She joined Dr. Peter Cresswell’s laboratory in the Department of Immunology at Yale University as a Howard Hughes Medical Institute fellow, and later as a Cancer Research Institute postdoctoral associate. Dr. Wang joined Memorial Sloan Kettering Cancer Center in 2007, and is currently the Associate Director of the Michael G Harris Cell Therapy and Cell Engineering Facility, and an Associate Lab Member in the Department of Molecular Pharmacology. Dr. Wang is in charge of the R&D, process development and manufacturing. She has overseen the successful manufacturing of all the CAR T cell products used in 13 phase I/II clinical trials, over 20 batches of cGMP grade viral vectors, as well as tech transfers to multiple industrial partners. She served in the translational science and product development committee in the American Society of Gene and Cell Therapy (ASGCT). She is currently a member for the National Heart, Lung and Blood Institute (NHLBI) independent external expert panel and a committee member for the Alliance of Regenerate Medicine (ARM) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL).
CEO, AgeX Therapeutics, USA
Mike served as BioTime’s CEO from 2007-2015, and is currently Co-CEO and member of the Board of Directors since 2002. Dr. West also serves as a Director of BioTime’s subsidiary company Asterias Biotherapeutics. From 1998 to 2007, Dr. West served as CEO, President, and Chief Scientific Officer of Advanced Cell Technology, Inc. (now Ocata Therapeutics), a company engaged in developing human stem cell technology for use in regenerative medicine. Prior to that, he was founder, officer, and board member of Geron Corporation (Nasdaq: GERN).
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.
VP, Information Management and Facilities Engineering, Adaptimmune
Charles Wilfong is Vice President of Information Management and Facilities Engineering functions at Adaptimmune, where he is responsible for global strategies and operations. He has 23 years experience across the pharmaceutical, biotechnology and engineering industries supporting Research, Clinical, Manufacturing and Commercial functions. Charles has led several large cross-functional change management initiatives to digitize core business process and make better use of data and analytics. He has a Bachelor of Architectural Engineering from the Pennsylvania State University, and a Master of Business Administration and Master of Public and International Affairs from University of Pittsburgh.
CAR T Technical Commercialization Lead, Cell Therapy Development & Operations (CTDO), Celgene
Chris Wiwi currently serves as the Technical Commercialization Team lead for bb2121, Celgene’s 1st in class anti-BCMA CAR T product for patients with relapsed refractory multiple myeloma. His team is responsible for ensuring a successful global launch of bb2121, in collaboration with Celgene’s partner, bluebird bio.
Chris has worked on the bb2121 program since its inception, in both functional leadership and team leadership roles. Most recently, he served as the CMC team lead, where he was responsible for all CMC aspects of bb2121 from IND phase to global registration. Under Chris’ leadership, the bb2121 team initiated clinical development in the United States, Europe, Canada and Japan, and recently completed enrollment for bb2121’s global registration study, KarMMa-1.
Previously, Chris was head of Analytical Research & Development for Celgene’s cell therapy organization, where he built and led the analytical development, validation and QC teams that were responsible for the advancement of multiple novel cellular therapy and biomaterials candidates from research to clinical manufacturing.
Chris is a recognized leader in the cell therapy field internally and externally.
He is a graduate of Celgene’s accelerated leadership program (Catalyst) reserved for top talent across the organization. Chris is very active in coaching and mentoring staff and was recently nominated for Celgene’s Mentor of the Year award.
Kyle and Josephine Morrow Chair and Professor in Microbiology, Baylor College of Medicine
Lynn Zechiedrich developed minivectors to study DNA, the enzymes that act on DNA, and the antibiotic and anticancer drugs that inhibit these enzymes. Minivectors also proved to be excellent gene therapy delivery vectors. Among other honors, she won a New Investigator Award from the Burroughs Wellcome Fund, a Curtis Hankamer Research Award, and funding from the Human Frontier Science Program. She is a Fellow of the National Academy of Inventors. She was Baylor College of Medicine’s BRASS Mentor of the Year in 2013. She holds two issued U.S. patents and three issued foreign patents that are licensed to Twister Biotech, Inc., a company she founded in 2011, and has multiple patents pending. She has published more than 60 articles and book chapters and given over 170 invited talks. She served on numerous grant review committees, reviews for 40 different peer-reviewed journals, ranging from mathematics and physics to microbiology and gene therapy, and serves on multiple editorial boards.