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Speakers

  • Julius Brennecke

    Julius Brennecke

    Academic Role: Senior Scientist, Institute of Molecular Biotechnology

  • Howard Chang

    Howard Chang

    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 have 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 CRISPER/Cas9 nuclease to develop a genetic drive targeting female fertility genes that spread into caged mosquito populations. HEG and CRISPER/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

    Michelle Hastings, PhD, is the Director of the Center for Genetic Diseases and an Associate Professor of Cell Biology and Anatomy at the Chicago Medical School at Rosalind Franklin University of Medicine and Science. Her research focuses on understanding genetic basis of disease and testing new therapeutic approaches by modulating the process of pre-mRNA splicing to alter gene expression. Her work has resulted in the discovery of effective means of targeting splicing with antisense molecules for the potential treatment of a number of neurodegenerative diseases including Batten disease, Usher syndrome, cystic fibrosis, Alzheimer’s and Parkinson’s disease. Dr. Hastings’ studies on Usher syndrome led to the first demonstration that hearing and balance can be recovered in mice with a mutation that causes congenital deafness in humans, laying the groundwork for developing a treatment for Usher in humans. Her recent work has demonstrated that antisense technology can modulate gene expression pathways associated with Alzheimer’s disease to mitigate learning and memory deficits in mouse models of the disease. A major focus of the lab currently is on developing approaches to treat different forms of Batten disease using antisense technology. Dr. Hastings holds a number of patents and has been supported by the National Institutes of Health and numerous Foundation grants. She was recognized as a 2019 Researcher to Know by the Illinois Science and Technology Consortium.

  • Katherine High

    Katherine High

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

    Dr. Katherine A. High is a physician-scientist with a longstanding interest in gene therapy for genetic disease. As an Investigator of the Howard Hughes Medical Institute at the Children's Hospital of Philadelphia (CHOP) and a faculty member of the University of Pennsylvania School of Medicine, she carried out pioneering basic and clinical studies in gene therapy for hemophilia and for a rare form of retinal dystrophy caused by mutations in the RPE65 gene. As Co-founder, President and Head of R&D at Spark Therapeutics, she led the approval of the first gene therapy for a genetic disease in the United States. Spark has received FDA breakthrough therapy designations for three different therapeutics, including for retinal dystrophy due to RPE65 mutations, and for hemophilia B and hemophilia A.  Dr. High is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, and is a Fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London). In addition to her operational role, Dr. High serves as a member of the Board of Directors of Spark, and also serves on the Board of Directors of CRISPR Therapeutics.

  • 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.

  • Art Levin

    Art Levin

    Academic Role: Chief Scientific Officer at Avidity Biosciences

    Art Levin has an unparalleled track record and reputation in the field of nucleic acid-based therapeutics. He serves as the Chief Scientific Officer at Avidity Biosciences. Previously he held that position at miRagen Therapeutics. Prior to that he held senior drug development roles at Ionis (formerly ISIS) Pharmaceuticals and Santaris Pharma.

    He has played key roles in the development of numerous of oligonucleotides including the first approved antisense drugs, and the first microRNA-targeted therapeutic in clinical trials. He has a combined three decades of experience in all aspects of drug development from discovery through drug registration, both in large pharma and biotech companies. Dr. Levin has published over 60 scientific articles and several of the most cited reviews in the field. He is a key opinion leader in the field and is on the scientific advisory boards of multiple institutions. Art received a doctorate in toxicology from the University of Rochester, and a bachelor’s degree in biology from Muhlenberg College.

     

  • 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

    Michael Rosbash is a Professor of Biology and the Peter Gruber Professor of Neuroscience at Brandeis University. He is also an Investigator of the Howard Hughes Medical Institute. Rosbash went to the Newton public schools in greater Boston and then to Caltech, graduating in 1965 with a B.S. in Chemistry. He spent the 1965-1966 academic year in Paris as a Fulbright Scholar in the lab of Marianne Grunberg-Monago and then entered the Ph.D. program at MIT in the fall of 1966. Rosbash worked there in the lab of Sheldon Penman and received a Ph.D. in Biophysics in 1970. After a brief stint at the University of St. Andrews, he was a post-doc in the lab of John Bishop in the Department of Genetics at the University of Edinburgh from 1971-1974. Rosbash joined the faculty of Brandeis University in 1974 and was promoted to Professor of Biology in 1986. He became a Howard Hughes Medical Institute Investigator in 1989. 

     Rosbash has made fundamental contributions to our understanding of the post-transcriptional regulation of gene expression, especially RNA metabolism in yeast. He is best known however for his work in Drosophila that illuminated our current understanding of the molecular mechanisms that underlie circadian rhythms, the intrinsic clock that controls the cyclic behaviors of all animals. These same molecules, molecular machines and biological principles not only control Drosophila circadian clocks but also the ubiquitous process of circadian rhythmicity throughout the animal kingdom. This circadian clock also controls much of cell physiology and metabolism, again in all animals - from humans to Drosophila (fruit flies).

    Rosbash and his Brandeis colleague Jeff Hall as well as Mike Young of the Rockefeller University have received numerous awards for their circadian work, including most recently the 2017 Nobel Prize in Physiology or Medicine. They previously received the Shaw Prize in Life Science and Medicine (2013), the Wiley Prize in Biomedical Sciences (2013), the Massry Prize (2012), the Canada Gairdner International Award (2012), the Louisa Gross Horwitz Prize for Outstanding Basic Research (2011), and the Peter and Patricia Gruber Foundation Neuroscience Prize (2009). Rosbash also received the Caltech Distinguished Alumni Award (2001), and he is a Member of the National Academy of Sciences, a Fellow of the American Association for the Advancement of Sciences and a Fellow of the American Academy of Arts and Sciences.

               

     

  • 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

    Jonathan K. Watts is Associate Professor at the RNA Therapeutics Institute of UMass Medical School (Worcester, MA, USA).  He completed his training at Dalhousie University (BSc), McGill University (PhD) and UT Southwestern Medical Center (Postdoctoral).  After establishing his independent laboratory at the University of Southampton, UK, he moved to the RNA Therapeutics Institute in 2015.  He has received over 20 awards including the 2013 OTS Young Investigator Award, the 2015 Vice Chancellor’s award for teaching, and the 2018 Angel Award for ALS research.
    Current work in the Watts lab is focused on the optimization of ASO chemistry for use in the CNS and lung, and on the chemistry of guides and donors for genome editing.  The Watts group works on both platform technology and disease applications, and has contributed to the development of two drugs that have reached patients on a compassionate use basis. Jon is on the Board of Directors of the Oligonucleotide Therapeutics Society since 2015. 

  • 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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