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Speakers

  • Howard Chang

    Howard Chang, MD, PhD

    Academic Role: Professor, Stanford University; Investigator, HHMI

    Howard Y. Chang M.D., Ph.D. is Director of the Center for Personal Dynamic Regulomes and the Virginia and D.K. Ludwig Professor of Cancer Genomics at Stanford University. He is a Howard Hughes Medical Institute Investigator; he is also Professor of Dermatology and of Genetics at Stanford University School of Medicine. Chang earned a Ph.D. in Biology from MIT, M.D. from Harvard Medical School, and completed Dermatology residency and postdoctoral training at Stanford University. His research addresses how large sets of genes are turned on or off together, which is important in normal development, cancer, and aging. Chang discovered a new class of genes, termed long noncoding RNAs, can control gene activity throughout the genome, illuminating a new layer of biological regulation. He invented ATAC-seq and other new methods for defining DNA regulatory elements genome-wide and in single cells. The long term goal of his research is to decipher the regulatory information in the genome to benefit human health. 

    Dr. Chang’s honors include the NAS Award for Molecular Biology, Outstanding Investigator Award of the National Cancer Institute, Paul Marks Prize for Cancer Research, Judson Daland Prize of the American Philosophical Society, and the Vilcek Prize for Creative Promise. He is a Member of the National Academy of Medicine, American Society for Clinical Investigation and Academia Sinica. His work was honored by the journal Cell as a Landmark paper over the last 40 years and by Science as “Insight of the decade”.

  • Paula Cohen

    Paula Cohen

    Academic Role: Professor; Associate Vice Provost for Life Sciences, Cornell University

  • Andrea Crisanti

    Andrea Crisanti

    Academic Role: Professor, Imperial College of London

    Andrea Crisanti is professor of molecular parasitology at Imperial College, graduated in Medicine at the University of Rome "la Sapienza' and carried his doctoral work at the Basel Institute for Immunology. Prof. Crisanti has also been appointed full professor of Microbiology and Clinical Microbiology at the University of Perugia where until recently has coordinated the Centre of Functional Genomics. He has pioneered the molecular biology of the human malaria vector Anopheles gambiae and has made a number of important scientific contributions that advanced the genetic and molecular knowledge of the malaria parasite and its mosquito vector. These include: i) the identification of the molecules involved in mosquito gut meal digestion; ii) the characterization of key genes involved in plasmodium molecular motor; iii) the development of gene transfer technology for anopheline mosquitoes; iv) the establishment of gene drive technology in mosquitoes; and v) the development of synthetic sex distorter producing male only progeny. The development of gene drive technology promises to overcome many of the roadblocks that so far have hampered the eradication of malaria in resource poor countries. Crisanti’s group has utilized this technology to spread genetic modifications impairing mosquito reproductive capability either targeting genes involved in female fertility of inducing male sex bias in the progeny. This works using a class of engineered enzymes that cut unique DNA sequences known as homing endonucleases (HEG) at defined chromosomal locations. During meiosis the cleavage site is repaired by homologous recombination using as template the allele carrying the HEG thereby increasing the frequency of the genetic modification in the progeny. Crisanti’s lab demonstrated that the activity of HEG is not due to intrinsic biological properties of the enzymes. Any endonuclease (TALEN or Zinc finger) placed in the right chromosomal location with the correct specificity for the corresponding site on the unmodified allele can function as drive. This provided the rationale to exploit the CRISPR/Cas9 nuclease to develop a genetic drive targeting female fertility genes that spread into caged mosquito populations. HEG and CRISPR/cas9 nuclease could also be used to manipulate the sex ratio of mosquitoes. This has been achieved by introducing nucleases that selectively shreds the X chromosome during sperm cell formation thereby generating a mosquito line that produces male gametes only. 

  • Kate Fitzgerald

    Kate Fitzgerald

    Academic Role: Principal Investigator, UMass Medical School

    Dr. Fitzgerald received her B.Sc. in Biochemistry in 1995 from University College Cork, Ireland, and her Ph.D. in 1999 from Trinity College Dublin, Ireland. She was a post-doctoral fellow in the Department of Biochemistry at Trinity College Dublin working with Luke O’Neill (1999-2002).  She joined the Division of Infectious Disease at the University of Massachusetts Medical Schoolas a Wellcome Trust Fellow in 2002 and joined the faculty in 2004. She is currently Professor of Medicine, Director of the Program in Innate Immunity and The Worcester Foundation Chair inBiomedical Sciences.
     
  • Myriam Gorospe

    Myriam Gorospe

    Academic Role: Chief, Laboratory of Genetics and Genomics NIA, NIH

    Dr. Gorospe received her Ph.D. in Molecular and Developmental Biology from the State University of New York at Albany in 1993. She then joined the National Institute on Aging (NIA) for post-doctoral training and has been a Principal Investigator and head of the RNA Regulation Section since 1998. Her group studies post-transcriptional gene regulation in mammalian models of cellular stress, cell division, senescence, and aging. Her research program investigates the influence of RNA-binding proteins (RBPs) and microRNAs on the expression ofgene products involved in these processes.

  • Michelle Hastings

    Michelle Hastings

    Academic Role: Associate Professor, Rosalind Franklin University of Medicine and Science

  • Katherine High

    Katherine High

    Academic Role: President and Head of Research and Development, Spark Therapeutics

    Dr. Kathy High, an accomplished hematologist with a longstanding interest in gene therapy forgenetic disease, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to adeno-associated viral (AAV) vectors in a variety of target tissues. Kathy’s work has evolved to encompass clinical translation of potential gene therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia (CHOP), Kathy assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application. As president at Spark Therapeutics, she has led the development and received regulatory approval of the first gene therapy for a genetic disease approved in both the U.S. and EU. Spark has shown human proof-of-concept of its leading gene therapy platform in both the retina and the liver, and has received breakthrough therapy designations for three different therapeutics across two different tissue targets, the eye and the liver. In addition to her operational role, Kathy serves as a member of the Board of Directors of Spark. Kathy was a long-time member of the faculty at the University of Pennsylvania and of the medical staff at CHOP, where she was also an Investigator of the Howard Hughes Medical Institute. She served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy (ASGCT). She received her A.B. in chemistry from Harvard University, an M.D. from the University of North Carolina School of Medicine, a business certification from the University of North Carolina Business School Management Institute for Hospital Administrators and an M.A. from the University of Pennsylvania.

  • Scott Johnson

    Scott Johnson

    Academic Role: Chief Medical Advisor, The Medicines Company

    T. Scott Johnson is Chief Medical Advisor at The Medicines Company. Scott is responsible for the medical affairs function of the business development team that identifies business opportunities based on changes in the medical and healthcare environment. During the past four years, he has been responsible for the licensing or acquisition of a number of products including inclisiran. Scott was a co-founder of MDCO and was a Director from September 1996 until July 2009 when he joined management on a full-time basis.

 Previously, Scott was a founding General Partner at JSB Partners, L.P., an investment bank with a focus on mergers and acquisitions, private financings and corporate alliances within the healthcare sector. From September 1991 to July 1999, Scott served as a founder and managing director of MPM Capital, LLC, a venture capital firm.

 Scott holds a Bachelor of Science in Chemistry (Phi Beta Kappa) and an MD (Alpha Omega Alpha) from the University of Alabama. His academic career included faculty positions at Harvard Medical School, the University of Colorado Medical School, and the University of South Alabama. He is Board Certified in Internal Medicine, Pulmonary and Critical Care Medicine and Sleep Disorders Medicine.

  • Kathy Niakan

    Kathy Niakan

    Academic Role: Group Leader, Francis Crick Institute

    Kathy Niakan is a Group Leader at the Francis Crick Institute in London where her laboratory investigates mechanisms that direct how the first cell types become specialized in their fate and function in human embryos. Her laboratory was the first to use CRISPR/Cas9 genome editing techniques to study gene function in human embryos and discovered the role of a key gene in human embryo development. Research equipment and objects from her lab have been exhibited at the Science Museum in London and are part of the permanent collection. In collaboration with Mary Herbert and Doug Turnbull at Newcastle University, work from her laboratory was provided as evidence to the UK Department of Health ahead of a vote in 2015 in the UK House of Commons and House of Lords to change regulation allowing the clinical use of mitochondrial replacement therapy. Kathy is a Blavatnik Award UK Finalist in Life Sciences and was named Time Magazine’s 100 Most Influential People. Kathy obtained a B.Sc. and B.A. from University of Washington, a PhD at University of California, Los Angeles and undertook postdoctoral training at Harvard University. She was a Next Generation Research Fellow at University of Cambridge.

     

  • Michael Rosbash

    Michael Rosbash

    Academic Role: Peter Gruber Endowed Chair in Neuroscience, Professor of Biology, HHMI, Brandeis University, 2017 Nobel Prize in Physiology or Medicine

  • Amy Wagers

    Amy Wagers

    Academic Role: Co-Chair, Harvard Department of Stem Cell and Regenerative Biology Harvard University

    Amy Wagers is the Forst Family Professor of Stem Cell and Regenerative Biology at Harvard University, Senior Investigator in the Section on Islet Cell and Regenerative Biology at the Joslin Diabetes Center, an HHMI Early Career Scientist, and a member of the Paul F. Glenn Laboratories for the Biological Mechanisms of Aging at Harvard Medical School. Dr. Wagers received her PhD in Immunology and Microbial Pathogenesis from Northwestern University, and completed postdoctoral training in stem cell biology at Stanford University. Dr.Wagers’ research seeks to understand how changes in stem cell activity impact tissue homeostasis and repair throughout life. Work from her lab provides evidence for the existence of a conserved systemic regulatory axis that modulates tissue maintenance and regeneration across a wide variety of tissues that vary significantly in their intrinsic repair capacity, and her ongoing studies have begun to identify the molecules responsible for age-variant regulation of regenerative potential. Dr. Wagers has authored more than 100 primary research and review articles, andher work has been recognized by awards from the Burroughs Wellcome Fund, Beckman Foundation, WM Keck Foundation, and Glenn Foundation, and National Institutes of Health. In 2013, she received the New York Stem Cell Foundation’s Robertson Prize for outstanding achievement in translational stem cell research.

  • Jonathan Watts

    Jonathan Watts

    Academic Role: Associate Professor, RNA Therapeutics Institute and Department of Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School

  • Timothy Yu

    Timothy Yu

    Academic Role: Neurologist and Principal Investigator, Division of Genetics and Genomics, Boston Children's Hospital and Harvard Medical School

    Dr. Yu is a neurologist and researcher at Boston Children’s Hospital. He completed his MD-PhD at UCSF studying circuit development in C. elegans with Cori Bargmann, neurology residency at Massachusetts General Hospital and Brigham and Women's Hospital, and a postdoctoral fellowship in human geneticswith Christopher Walsh. He is an Assistant Professor at Harvard Medical School and an Associate Member of the Broad Institute of MIT and Harvard. An early pioneer inhigh throughput sequencing, he developed some of the first methods for genome-scale sequencing and wrote one of the very first bioinformatic pipelines for large-scale interpretation of human genomic variation. He has identified or contributed to the identification of over a dozen new human disease genes and co-founded a pediatric genomic diagnostic company. His lab applies diverse skills in genetics, neurobiology, and bioinformatics to study neurodevelopmental disorders and advance genomic medicine. Current projects range from computational analyses of tens of thousands of individuals with autism to identify disease genes, to investigations of genome sequencing for newborn screening and neonatal ICU care, to the development of rapid-turnaround, N-of-1 personalized therapies for rare pediatric disorders.

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