XXVI Congress

118 days

12 hours

31 mins

Confirmed speakers

 

Gordon Keller earned his PhD in Immunology at the University of Alberta and completed a Post Doctoral Fellowship at the Ontario Cancer Institute in 1983. His career has taken him to Basel, Austria, Denver and New York. In 2007, Keller returned to Canada to accept the position of Director, McEwen Centre for Regenerative Medicine in Toronto. Dr. Keller is best known for his research in lineage specific differentiation of mouse and human embryonic stem cells. In December 2016, Keller was named scientific co-founder of BlueRock Therapeutics, a next-generation regenerative medicine company.

  Molly Stevens is Professor of Biomedical Materials and Regenerative Medicine & Research Director for Biomedical Material Sciences at Imperial College London. Her research focusses on designing and developing innovative bio-inspired materials for applications in regenerative medicine, tissue engineering and biosensing and has been recognised by over 20 major awards, including the 2016 Clemson Award from the Society for Biomaterials.

 
Yasuhiro Yamada is a Professor at the Institute of Medical Science, University of Tokyo, Japan. He earned PhD from Gifu University School of Medicine. He did his post-doctoral research at the Whitehead Institute, MIT. He took up a professorship in 2010 at the CiRA, Kyoto University, and then moved to the University of Tokyo in 2017. He is currently trying to use reprogramming technology to dissect the role of epigenetic regulations on cancer development.   Rick Livesey has recently been appointed as the Chair of Stem Cell Biology, UCL/Great Ormond Street Institute of Child Health, based in the newly created Zayed Centre for Rare Disease in Children. He is currently a Senior Group Leader at the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge, where he has been based since 2001. His lab uses human stem cell systems to study human forebrain development, evolution and disease, with a focus on the cellular biology of highly penetrant mutations. That work has also enabled research on therapeutic target identification and validation in neurodegeneration, as well as studies of therapeutic efficacy in human neurons and microglia.
Kristin Baldwin is a Professor of Neuroscience at the Scripps Research Institute and Adjunct Professor at UCSD. Dr. Baldwin’s research aims to develop reprogramming and genome editing techniques to produce cell types to model and treat disease. Dr. Baldwin received a B.S. from Duke University, a Ph.D. from Stanford University and performed postdoctoral studies at Columbia University. She has been named a Pew Scholar, a Kavli Fellow and an NIH Director’s Pioneer Awardee.   Asuka Morizane is Assistant Professor at the Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto. His research interests include the clinical application of stem cell-based therapy for Parkinson’s disease (PD). His group has shown that the induced pluripotent stem cell (iPSC)-derived dopamine neurons can be grafted and survive in the PD model of non-human primates with functional recovery and is currently setting up a clinical trial of cell therapy for PD with human iPSCs.
Alexander Meissner studied Medical Biotechnology at the Technical University Berlin before starting his PhD studies with Rudolf Jaenisch at the Whitehead Institute/MIT in 2002. He completed his PhD in 2006 and spent the next year and a half working with Rudolf Jaenisch and Eric Lander before starting his own lab as an assistant professor in the Department of Stem Cell and Regenerative Biology at Harvard University and as an associate member of the Broad Institute in 2008. He was promoted to associate professor in 2012 and full professor with tenure in 2015. In 2016 he has been appointed as Director and Head of the Department of Genome regulation at the Max Planck Institute for Molecular Genetics in secondary employment and changed it to his principal employment in 2017.   Isabelle André-Schmutz is a research director at INSERM and co-head of the “human lymphohematopoiesis” lab at Imagine Institute (Paris, France). Her main research focuses are deciphering human hematopoiesis and lymphopoiesis and implementation of innovative cell and gene therapy pre-clinical studies for inherited and acquired disease of the hematopoietic system. Her work has led to the development of an artificial thymus, which will be tested in a clinical trial. Her team also identified new genes involved in severe forms of inherited immune deficiencies in children and demonstrated genotoxicity of anti-viral drugs, such as AZT, used to prevent transmission of HIV during pregnancy.
Keisuke Kaji is a professor and a MRC Senior Non-Clinical Fellow in MRC Centre for Regenerative Medicine, University of Edinburgh, and interested in molecular mechanisms of reprogramming. His group previously developed a single vector reprogramming system with piggyBac transposon (Kaji, Nature, 2009, Woltjen, Nature, 2009), and identified cell surface markers to track the reprogramming process (O’Malley, Nature, 2013). The group currently aims to dissect the reprogramming mechanisms using CRISPR/Cas9 technology.   Toni Cathomen is Director of the Institute for Transfusion Medicine and Gene Therapy at the Medical Center of the University of Freiburg, Germany. His research activities focus on developing cell therapies based on induced pluripotent stem cells (iPSCs), improving the effectiveness and safety of designer nucleases (CRISPR-Cas and TALEN), and developing and manufacturing genome edited stem cell and immune cell preparations for the treatment of HIV infection, primary immunodeficiencies and cancer.
Amy Wagers is the Forst Family Professor of Stem Cell and Regenerative Biology at Harvard University and the Harvard Stem Cell Institute, and a Senior Investigator at the Joslin Diabetes Center. Her laboratory investigates how changes in stem cell activity impact tissue homeostasis and repair throughout life, and how stem cells may be harnessed for regenerative medicine using cell transplantation and gene editing approaches.   J. Keith Joung is the Desmond and Ann Heathwood Research Scholar and Pathologist at Massachusetts General Hospital and Professor of Pathology at Harvard Medical School. Dr. Joung’s research interests include the development of targeted genome editing and epigenome editing technologies. He has received numerous awards including an NIH Director’s Pioneer Award, an NIH R35 MIRA (Maximizing Investigators Research Award), and designation as a “Highly Cited Researcher” in 2016 and 2017 by Thomson Reuters/Clarivate Analytics.
Juergen Knoblich is the deputy director of the Institute of Molecular Biotechnology in Vienna. He is a developmental neuroscientist studying human brain development and psychiatric disorders. His group has developed a method for growing human brain tissue in the lab. They can recapitulate human embryonic brain development during the first trimester and analyze the developmental defects leading to neurological disorders.   Amander Clark is Professor and Chair of the Department of Molecular Cell and Developmental Biology, University of California, Los Angeles (UCLA). She is a key member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Co-Director of the UCLA Human Embryonic Stem Cell Derivation laboratory. As an independent investigator, Dr. Clark’s research is focused on germline development and epigenetic reprogramming using human pluripotent stem cells as models.
Alessio Cantore has a Ph.D. in cellular and molecular biology and is currently Project Leader at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Assistant Professor of Tissue Biology at the San Raffaele University, Milan, Italy. His research interests span between lentiviral vector engineering and the biology and translational applications of liver-directed gene transfer.   Grégoire Courtine received his PhD degree from the French institute of health (INSERM) and University of Pavia in Italy in 2003. After obtaining the Chancellor Award during his post-doctoral training at the University of California Los Angeles (UCLA), he established his own laboratory at the University of Zurich in 2008. He became Associate Professor in the Center for Neuroprosthetics at the Swiss Federal Institute of Technology, Lausanne (EPFL) in 2012. In 2014, he launched a startup that aims to translate the medical and technological advances gained over the past 15 years into a treatment to accelerate and augment functional recovery after spinal cord injury.
Eva Hedlund obtained her PhD from the Karolinska Institute and thereafter conducted postdoctoral research at UCLA and Harvard Medical School focused on CNS development and regeneration. Since 2011 she is an associate professor at the Karolinska Institute. Her laboratory focuses on elucidating mechanisms of neuronal vulnerability and resistance to identify new molecular targets for the treatment of motor neuron diseases. Her laboratory uses stem cells, genome editing, microfluidics, single cell RNA-sequencing and gene therapy   Deepak Srivastava is President of the Gladstone Institutes, Director of the Roddenberry Stem Cell Center at Gladstone, and Professor at the University of California, San Francisco. Dr. Srivastava’s laboratory discovered genetic bases for human cardiac septal and valve defects and revealed complex gene networks that regulate progenitor cells to adopt a cardiac cell fate and subsequently fashion a functioning heart. He has leveraged this knowledge to reprogram fibroblasts directly into cardiomyocyte-like cells.
Fiona Watt obtained her DPhil from the University of Oxford and carried out postdoctoral research at M.I.T., Cambridge, USA. The major research interest of her lab is in the interplay between intrinsic and extrinsic factors in the regulation of stem cell fate. Fiona is the Director of the Centre for Stem Cells & Regenerative Medicine at King’s College London and is currently on secondment as the Executive Chair of the Medical Research Council.   Robert Kotin is the Scientific Founder and Vice President of Virology and Gene Therapy at Generation Biologics. Kotin spent most of his career in the Intramural Research Program at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) as a tenure track investigator then as a tenured senior investigator and Head of the Laboratory of Molecular Biology and Gene Therapy. After leaving the NIH, Kotin joined Voyager Therapeutics as the Vice President of Virology and Gene Therapy. He earned his Bachelor of Arts degree in biology from the University of California, Santa Cruz and Ph.D. from Rutgers University and the University of Medicine and Dentistry of New Jersey (now Robert Wood Johnson Medical School) in microbiology.
David Schaffer is a Professor of Chemical and Biomolecular Engineering, Bioengineering, and Neuroscience at the University of California, Berkeley, where he also serves as the Director of the Berkeley Stem Cell Center. There, he applies engineering principles to enhance stem cell and gene therapies, work that includes novel approaches for molecular engineering and evolution of new viral vectors as well as new technologies to investigate and control stem cell fate decisions.   Marcos Timón is Head of Service of Biological Products, Advanced Therapies and Biotechnology within the Spanish Medicines Agency (AEMPS). He is the alternate member for Spain at CAT and a member of the BWP. Marcos did his PhD in Madrid and then moved to London as a postdoctoral scientist first and a research fellow later. Back to Spain he worked as CSO at Mologen Molecular Medicines, a subsidiary of Mologen AG before joining AEMPS.
Juan Ruiz is Chief Medical Officer at Abeona Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel therapies for life-threatening rare genetic diseases. He is responsible for the clinical programs of the company in Sanfilippo A and B as well as Recessive Defective Epidermolysis Bullosa. He studied Medicine and received his PhD at the University of Navarra, where he started his career in gene therapy under the supervision of Jesus Prieto. He was Medical Director at DIGNA Biotech where he developed several candidate drugs for orphan conditions, including an AAV for Acute Intermittent Porphyria.   Silvia Priori is Global Scientific Director of the ICS MAUGERI SpA SB, Full Professor of Cardiology since 2014 in the Department of Molecular Medicine at the University of Pavia (Italy), Director of Molecular Cardiology Unit at the Centro National de Investigaciones Cardiovasculares Carlos III Madrid and at the ICS MAUGERI in Pavia, Italy. She is best known for her work on genetics and pathophysiology of genetic cardiac arrhythmias and for Genetic Therapy.
Beverly Davidson is the Director of The Raymond G. Perelman Center for Cellular and Molecular Therapeutics and a Professor of Pathology and Laboratory Medicine, at the Children’s Hospital of Philadelphia and University of Pennsylvania. Her research is focused on inherited CNS diseases, with studies directed at defining the pathogenesis of these disorders and developing novel medicines for therapy, including gene replacement and gene silencing strategies for recessive and dominant CNS diseases, respectively.   Dirk Grimm is Professor of Viral Vector Technologies at Heidelberg University Hospital, Germany. For >20 years including a 6-year postdoctoral stint at Stanford University (CA, USA), he has engineered adeno-associated viral (AAV) vectors for therapeutic in vivo gene transfer. Hallmarks include the first application of DNA family shuffling for AAV capsid evolution or the discovery of RNAi-associated toxicity. Using high-throughput technologies and combinatorial RNAi/CRISPR expression, his current laboratory tailors AAV vectors for treatment of infectious or monogenic disorders. Another emerging research focus is on the use of targeted AAV vectors for direct in vivo reprogramming of somatic cells in living organisms, with the ultimate aim of in situ tissue repair and regeneration.
Hans Ovelgönne studied biology at the Wageningen agricultural university and received a PhD from Utrecht University on the molecular biology of stress proteins. Hans worked at the faculties of biology and veterinary medicine of Utrecht university, and at the departments of avian virology and mammalian virology of the ID-DLO in Lelystad. Later he joined the national institute for health and environment (RIVM) as a medicines assessor. Currently he works for the Medicines Evaluation Board (CBG), representing the CBG in the in the Committee for Advanced Therapies (CAT) and in the Scientific Advice Working Party at the EMA.   Ana Buj Bello is research director at INSERM and head of the Neuromuscular Disorders and Gene Therapy team at Genethon, France. Her research activities focus on developing AAV-based gene therapies for neuromuscular disorders, in particular congenital myopathies, with a major interest in clinical translation. Her pioneering work on gene replacement therapy for myotubular myopathy has led to the initiation of a clinical trial in patients.
Jennifer Erwin is a molecular geneticist and neuroscientist who studies how environmental and genetic variations affect the human brain in health and disease. Her research group uses a hybrid of human stem cell models, post-mortem tissue and computational approaches to interrogate the contribution of epigenetic and somatic mosaicism to brain development. She competed postdoctoral training with Fred H Gage at the Salk Institute and her PhD training with Dr. Jeannie T. Lee at Harvard.   Shin-ichi Muramatsu is a professor of neurology at Jichi Medical University in Japan. He is also a project professor of Center for Gene & Cell Therapy at The Institute of Medical Science, The University of Tokyo. Using AAV vectors, his group has been developing gene therapies for neurodegenerative diseases. His expertise has contributed to clinical trials of gene therapy for AADC deficiency. He is a founding member of Gene Therapy Research Institution Co., Ltd.