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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Editorial Advisory Board

Senior Editor

Chris Mason

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

Robin Ali

ROBIN ALI

Professor of Molecular Genetics, UCL, Chief Scientific Officer, MeiraGTx

Robin Ali is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology. He is also Theme Leader for Gene Therapy at NIHR Biomedical Research Centre, Moorfields Eye Hospital and Director of the Wolfson Gene Therapy Unit, which is the UCL GMP facility for manufacturing clinical grade gene therapy vectors. He is Visiting Professor of Ophthalmology at Kellogg Eye Center, University of Michigan and Founder and Chief Scientific Officer of MeiraGTx, a UK incorporated gene therapy company with offices in London and New York. The company has secured substantial investment to develop a path to market for a number of ocular therapies licensed from UCL. The main focus of Robin Ali’s research is the development of gene and cell therapy for the treatment of retinal disorders. Since 1996, he has published over 170 peer-reviewed papers with landmark papers and preclinical proof-of-concept (POC) studies for many different ocular disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy. The results from this trial reporting an improvement in vision (NEJM, 2008; NEJM 2015) along with results from two other trials, established POC for gene therapy for inherited retinal degeneration. His group has also provided the first POC for effective transplantation of photoreceptors (Nature 2006; Nature, 2012) that has provided the basis for ES cell-derived photoreceptor transplantation, now a major programme in his laboratory (Nature Biotech 2013). Robin Ali and members of his team have received numerous prizes and awards for their work on developing new treatments for retinal degeneration, including the Pfizer/ARVO Translational Award for Ophthalmology (2010), the Alcon Research Institute Award (2009) and in 2014, Human Gene Therapy journal’s Pioneer Award (to honour the top 12 pioneers in gene therapy). In 2007 he was elected to the Academy of Medical Sciences as its youngest elected member and in 2009 elected NIHR Senior Investigator (re-elected in 2014). He is currently President of the European Society of Gene and Cell Therapy and has served on the advisory boards of a number of funding bodies including the UK Medical Research Council (Neurosciences and Mental Health Board and the Regenerative Medicine Research Committee), Research to Prevent Blindness USA and Fighting Blindness Ireland, Telethon, Italy, as well as advisory boards of several pharmaceutical and biotech companies, including Alcon/Novartis.


Tony Atala

ANTONY ATALA

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.


Andrew Baker

ANDREW BAKER

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.


NINA BAUER

Associate Director, Commercial Development, Lonza, Switzerland

Nina Bauer leads Lonza’s Autologous Cell Therapy business, with manufacturing sites in the US, Europe and Asia. As part of this role, she is also in charge of establishing novel manufacturing technologies to remove bottlenecks and enable commercially viable services for patient-scale therapies. With more than 12 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. She holds a PhD in Neuroscience from the University of Oldenburg (GER), an MBA from the University of Edinburgh (UK), and conducted post-doctoral research at the Weizman Institute (ISR), Salk Institute (USA), and the University of Edinburgh (UK).


Alexey Bersenev

ALEXEY BERSENEV

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.


Gregory Bonfiglio

GREGORY BONFIGLIO

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).


Christopher Bravery

CHRISTOPHER BRAVERY

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.


Tania Bubela

TANIA BUBELA

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.


Lee Buckler

LEE BUCKLER

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.


JUAN BUEREN

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.


Bruce Bunnell

BRUCE BUNNELL

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.


NATHALIE CARTIER-LACAVE

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.


MARINA CAVAZZANA

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.


NICK CRABB

Programme Director – Scientific Affairs, National Institute for Health and Care Excellence, UK

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 as the associate director for the Diagnostics Assessment Programme in 2010. Nick was responsible for the establishment and management of the programme. In 2014 Nick was appointed Programme Director for Scientific Affairs where he oversees NICE Scientific Advice, the Science Policy and Research programme and the NICE Office for Market Access.


Wilfried Dalemans

WILFRIED DALEMANS

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.


Editorial Board member

OLIVIER DANOS

Chief Scientific Officer, REGENXBIO Inc, USA

Dr. Olivier Danos is the Chief Scientific Officer for REGENXBIO. Prior to joining REGENXBIO, Dr. Danos was Senior Vice President, Cell and Gene Therapy at Biogen Inc. At Biogen, Dr. Danos led company efforts dedicated to identifying and developing new technologies for gene transfer and genome engineering. Dr. Danos also co-founded Lysogene, a NAV Technology Licensee focused on the development of gene therapy product candidates for the treatment of Mucopolysaccharidosis Type IIIA.
Prior to Biogen, Dr. Danos served as Senior Vice President, Molecular Medicine, Synthetic Biology and Gene Regulation at Kadmon Pharmaceuticals. Earlier in his career, Dr. Danos was Director of the Gene Therapy Consortium of the University College of London, Scientific Director at Genethon and Senior Director of Research at Somatix Therapy Corporation. Dr. Danos has directed research focused on gene therapy at the Necker – Enfants Malades Hospital in Paris, the French National Centre for Scientific Research and the Pasteur Institute in Paris.


Robert Deans

ROBERT DEANS

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.


Colleen Delaney

COLLEEN DELANEY

Founder and Chief Medical Officer, Nohla Therapeutics, USA

Colleen Delaney is the Founder and Chief Medical Officer at Nohla Therapeutics. She is also an Associate Member and recipient of the Madeline Dabney Adams Endowed Chair of the Fred Hutch Clinical Research Division and an Associate Professor at the UW Department of Pediatrics, Division of Pediatric Hematology/Oncology. 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.
Colleen is also the Principal Investigator of a research laboratory focused 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 group at Fred Hutchinson Cancer Research Center developed novel and clinically feasible methods 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 pilot study investigating the use of ex vivo expanded cord blood progenitors to augment conventional cord blood transplantation. 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.
Colleen received her MD from Harvard Medical School in 1996, and went on to complete a residency in Pediatrics at the University of California, San Francisco followed by a Fellowship in Pediatric Hematology/Oncology at Fred Hutchinson Cancer Research Center/University of Washington.


Massimo Dominici

MASSIMO DOMINICI

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).


Paul Fairchild

PAUL FAIRCHILD

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.


Boris Fehse

BORIS FEHSE

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.


Joseph Glorioso III

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 Gregory

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.


RICHARD HARBOTTLE

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.


Martin Hildebrandt

MARTIN HILDEBRANDT

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.


Armand Keating

ARMAND KEATING

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.


Paul Kemp

PAUL KEMP

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.


Sven Kili

SVEN KILI

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.


Joanne Kurtzberg

JOANNE KURTZBERG

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.


James Lawford-Davies

JAMES LAWFORD-DAVIES

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.


MICHAEL LINDEN

Co-founder, Handl Bio Partners, UK

After receiving his Ph.D. in Biochemistry and Molecular Biology at the University of Zürich, Switzerland, Prof. Linden trained in Molecular Virology at Cornell University Medical College, N.Y. After his postdoctoral training he joined the faculty at Mount Sinai School of Medicine in New York, where he used a multidisciplinary approach to understanding the molecular mechanisms and the potential applicability of site-specific genome integration by adeno-associated virus (AAV). In 2007, he moved continents to set up a new research group at King’s College London as the chair in Molecular Virology.
His contributions to the field include the biochemical and structural determination of the AAV Rep proteins, which orchestrate all aspects of the AAV life cycle, and the proposal of a mechanism of site-specific integration. During these studies Prof. Linden’s laboratory has demonstrated the potential of using embryonic stem cells for gaining insights into complex viral mechanisms.
Throughout his career, Prof. Linden has engaged in the field of AAV-mediated gene therapy including a role as the Director of the UCL Gene Therapy Consortium where he was responsible for clinical grade vector production and the support for further developments in UK based gene therapy efforts. In this context Prof. Linden was actively engaged in translational projects aimed at the development of novel therapies using AAV-based vectors.


Mark Lowdell

MARK LOWDELL

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.


Geoff MacKay

GEOFF MACKAY

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 Matthew

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.


Kirstin Matthews

KIRSTIN MATTHEWS

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.


Michael May

MICHAEL MAY

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

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.


Christian Meyer

CHRISTIAN MEYER

Chief Medical Officer, Therachon AG

Before entering his current role, Dr. Meyer was the Chief Medical Officer at uniQure B.V. where he supported Glybera, the first AAV gene therapy ever approved, and was instrumental to uniQure’s IPO on NASDAQ in 2014. He also led the clinical development of several additional AAV gene therapies for Acute Intermittent Porphyria, Sanfilippo B, Huntington’s disease and Hemophilia B, the latter of which obtained Breakthrough Therapy and PRIME designation. Prior to uniQure, he held several senior executive positions at Cardoz AB, Symphogen A/S and Zymenex A/S. Dr. Meyer began his career at Novo Nordisk A/S and held numerous academic and hospital positions before entering the pharmaceutical industry. Dr. Meyer received his Ph.D. and M.D. from the University of Copenhagen, Denmark.


Ryuichi Morishita

RYUICHI MORISHITA

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.


EMMA MORRIS

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.


KEVIN MORRIS

Professor, Center for Gene Therapy, Hematologic Malignancies and Stem Cells, City of Hope, 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. For more than a decade, Kevin Morris, Ph.D. has been one of the brightest lights in RNA research. Morris was the first to recognize unique properties of non-coding RNA; he is considered an international leader in the field. The goal of his work, in his words, is “to understand the role of non-coding RNA in life, evolution and selection.” and to use that knowledge to develop novel therapies for AIDS, cancer and other diseases. At the Center for Gene Therapy, Kevin focuses on manipulating RNA in a variety of ways to control and repress HIV-1.

Morris has led research laboratories at the renowned Scripps Research Institute in La Jolla, California as well as the University of New South Wales, Australia. His relationship with City of Hope began in 2004 with researcher and adjunct professor positions at the Beckman Research Institute of City of Hope.


Claudio Mussolino

CLAUDIO MUSSOLINO

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.


Amit Nathwani

AMIT NATHWANI

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.


IOANNIS PAPANTONIOU

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.


ROGER PEDERSEN

Wellcome Trust-MRC Cambridge Stem Cell Institute, University of Cambridge, UK

Roger received degrees in biology from Stanford (A.B, 1965) and Yale (Ph.D., 1970) and did postdoctoral work in mammalian embryology at Johns Hopkins. In 1971, he joined the University of California, San Francisco, where he studied developmental potency and fate in mammalian embryos. He moved in 2001 to the University of Cambridge, where he continued his research on human embryonic stem cells as Professor of Regenerative Medicine. The Cambridge Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute (www.stemcells.cam.ac.uk), which he co-founded, is now one of the world’s leading stem cell research centres. Prof Pedersen serves on numerous editorial boards and is a fellow of the Royal Society of Biology and of Churchill College, Cambridge. During the past 16 years Prof Pedersen’s Cambridge lab made huge strides in understanding the cytokine signalling pathways involved in maintaining human embryonic stem cell pluripotency and inducing their differentiation. More recently, his lab focused on differentiation of human pluripotent stem cells (hPSCs) into mesodermal cell types with potential applications in drug discovery, toxicity testing and cell-based therapies. This culminated in functional studies of hPSCs transplanted into gastrula stage mouse embryos to validate their developmental capacity in an organized tissue context. These findings are important because they provide compelling evidence for the functional potential of human pluripotent stem cells, both for modelling human gastrulation and for regenerative medicine.


Kai Pinkernell

KAI PINKERNELL

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.


Brock Reeve

BROCK REEVE

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.


Louise Rodino Klapac

LOUISE RODINO-KLAPAC

Vice President – Gene Therapy, Sarepta, USA

Renowned for her work in molecular genetics and gene therapy, Dr. Rodino-Klapac was head of the laboratory for gene therapy research for muscular dystrophies at Nationwide Children’s Hospital prior to joining Sarepta.

From July 2010 to June 2018, Dr. Rodino-Klapac was an Associate Professor, Department of Pediatrics, The Ohio State University College of Medicine and Principal Investigator, Center for Gene Therapy for The Research Institute at Nationwide Children’s Hospital and a faculty member of the Biomedical Sciences Graduate Program and Molecular, Cellular, and Developmental Biology Graduate Programs, The Ohio State University College of Medicine. Dr. Rodino-Klapac also served as the Ruth L. Kirschstein F32 Post-doctoral Fellow, National Institutes of Health, The Research Institute at Nationwide Children’s Hospital; and was a post-doctoral researcher, Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital.

In February 2017, Dr. Rodino-Klapac co-founded Myonexus Therapeutics, Inc., a clinical stage gene therapy biotech company focused on treatment of Limb girdle muscular dystrophy (LGMD). Dr. Rodino-Klapac serves as acting chief scientific officer of Myonexus and in early May 2018, Sarepta announced a collaboration with Myonexus to develop potentially transformative gene therapies to treat five distinct forms of LGMD.

Dr. Rodino-Klapac is the recipient of numerous awards, including the Forty Under 40 Award by Columbus Business First, and the Department of Pediatrics Outstanding Junior Faculty Award for Innovation. She’s the author of numerous publications, among them papers published in The New England Journal of Medicine, Annals of Neurology, and Pediatric Neurology.

Dr. Rodino-Klapac received her bachelor’s degree in biology from Kings College, and a Ph.D. in molecular genetics from The Ohio State University.


Michael Rudnicki

MICHAEL RUDNICKI

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.


Paula Salmikangas

PAULA SALMIKANGAS

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

BEATRIZ SAN MARTIN

Partner, Fieldfisher, 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.


STEFAN SCHEDING

Division Head, Lund Stem Cell Center, Lund University, Sweden
 
 
 


Shintaro Sengoku

SHINTARO SENGOKU

Graduate School of Innovation Management, Tokyo Institute of Technology, Japan
 
 
 


Bernie Siegel

BERNIE SIEGEL

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).


Jan Thirkettle

JAN THIRKETTLE

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 Thrasher

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.


Phil Vanek

PHIL VANEK

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.


Mike West

MIKE WEST

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.

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