We launched Fulcrum Therapeutics with a bold vision: To change the course of genetically defined diseases by treating them at their root cause.
Our approach to drug discovery generates significant insights into disease biology and allows us to think creatively about the best way to modulate and balance gene expression. The result: A patient-focused product engine designed to systematically identify and validate cellular drug targets that can modulate gene expression to treat the known root cause of genetically defined diseases.
Robert J. Gould, Ph.D.
President and Chief Executive Officer, Board Member
Robert J. Gould joined Fulcrum as president and CEO at the time of the company’s launch in 2016, bringing over 30 years of experience to the role. Previously, he served as president and CEO of Epizyme from 2010 to 2015. Prior to joining Epizyme, Robert served as director of novel therapeutics at Broad Institute of MIT and Harvard from 2006 to 2010. Robert spent 23 years at Merck where he held a variety of leadership positions, culminating in the role of vice president, licensing and external research. During his time at Merck, Robert was instrumental in advancing more than 20 compounds from discovery into clinical development in multiple therapeutic areas. Robert received a bachelor’s from Spring Arbor University and a Ph.D. from the University of Iowa and completed postdoctoral studies at Johns Hopkins University.
Chief Operating Officer
Bryan Stuart joined Fulcrum as chief operating officer in 2018, bringing over 20 years of experience building biopharmaceutical companies, including deal-making, capital raising and strategic and operational leadership. Most recently, he was the president and CEO of Yarra Therapeutics, a subsidiary of Array BioPharma focused on genetically defined rare diseases. Previously, Bryan served as president, then CEO of Kastle Therapeutics, a biopharmaceutical company focused on developing and commercializing therapeutics for rare diseases. Prior to Kastle, Bryan was the chief business officer of Civitas Therapeutics, where he led the business development, capital raising, commercial and portfolio management functions, including the planned IPO and company sale to Acorda (acquired Civitas day of scheduled IPO pricing). He previously led business development, corporate development and strategy at both EKR Therapeutics (acquired by Chiesi) and Ovation Pharmaceuticals (acquired by Lundbeck A/S), where he played key roles in each company’s growth and led the successful sale of both companies. Bryan began his career as a healthcare investment banker at William Blair & Company. He has an MBA from the Kellogg School at Northwestern University and a bachelor’s from the University of Illinois.
Owen Wallace, Ph.D.
Chief Scientific Officer
Owen Wallace joined Fulcrum in April 2017. He has more than two decades of experience in drug discovery and development across multiple therapeutic areas, including neuroscience, endocrine, infectious, respiratory and rare diseases. He is the co-inventor of seven clinical candidates and has overseen teams who have progressed more than 30 compounds into clinical development. Prior to joining Fulcrum, Owen served as head, global discovery chemistry at Novartis, where he was responsible for teams and projects across multiple sites in several disease areas and the chemical genetics field. Prior to Novartis, he was the site scientific leader with Eli Lilly & Company in the United Kingdom, leading a cross-functional team focusing on Alzheimer’s disease and schizophrenia. Before that, he held research positions at Bristol-Myers Squibb, where he co-initiated a program on a novel mechanism for inhibiting HIV infection that is now in Phase 3 clinical trials. Owen earned his Ph.D. in organic chemistry from Yale University.
Diego Cadavid, M.D.
Senior Vice President, Clinical Development
Diego Cadavid joined Fulcrum in September 2016. He brings more than 22 years of experience in academic and biotechnology research and drug development. Prior to joining Fulcrum, Diego was at Biogen for more than eight years, where he was senior medical director in the multiple sclerosis clinical development group and director of the Neurology Clinical Group. During much of his time at Biogen, Diego was also a consultant at the Center for Immunology and Inflammatory Diseases at the Massachusetts General Hospital in Boston. Before joining Biogen, Diego spent nine years in academia at Rutgers-New Jersey Medical School, where he was an associate professor of neurology and neuroscience and worked on basic research on spirochetal brain infections and clinical research in multiple sclerosis. Diego completed postdoctoral research in microbiology and immunology and an internship in internal medicine at the University of Texas Health Science Center in San Antonio; neurology residency training at Georgetown University Hospitals; and a neuropathology fellowship at the Armed Forces Institute of Pathology. Diego holds an M.D. from Universidad Javeriana in Bogota, Colombia and has a faculty appointment at the University of Massachusetts.
Christopher Moxham, Ph.D.
Senior Vice President, Discovery Research
Christopher (Chris) Moxham joined Fulcrum in January 2019. He has over 20 years of drug discovery experience, primarily spent at Eli Lilly and Company where he most recently served as vice president, quantitative biology. In this role and during his tenure, he helped discover multiple clinical candidates, grew Lilly’s immuno-oncology discovery capabilities and pipeline, developed critical biopharma and academic partnerships and established a state-of-the-art lab facility. Previously, he was associate vice president, oncology research at Lilly where he established and led the company’s biomarker group. Chris began his tenure at Lilly as a research scientist focused on diabetes and obesity drug discovery. He also spent two years as a senior research fellow at Merck, where he supported a portfolio of projects centered on Alzheimer’s disease and led work that resulted in a clinical candidate. Chris has a bachelor’s in biological sciences from Cornell University and a Ph.D. in molecular and cellular pharmacology from the State University of New York at Stony Brook. He also conducted postdoctoral training at SUNY Stony Brook, sponsored by the National Institutes of Health.
Pamela S. Strode
Senior Vice President, Regulatory Affairs and Quality Assurance
Pamela (Pam) Strode joined Fulcrum in September 2019. She comes to Fulcrum from Epizyme, where she served as senior vice president, regulatory affairs and quality assurance, and played a critical role in establishing the team’s regulatory affairs and quality assurance departments. At Epizyme, Pam directed multiple U.S. Fast Track and U.S./E.U. Orphan Drug Designations and also led the regulatory strategy and operations for several clinical trial applications and a New Drug Application (NDA). She brings more than 30 years of regulatory leadership for investigational and marketed products across multiple therapeutic areas, with increasing levels of responsibility at Bristol-Myers Squibb, Boehringer Ingelheim Pharmaceuticals, Inc. and Cerulean Pharma. Pam has a B.S. in microbiology from Cook College at Rutgers University and an M.S. in QA/RA from Temple University, where she also obtained a Drug Development Certificate.
Lucienne Ronco, Ph.D.
Vice President, Translational Medicine
Lucienne Ronco joined Fulcrum at the company’s launch in 2016. She brings more than 17 years of industry experience in translational medicine, drug discovery, biotechnology and pharmaceuticals. Prior to joining Fulcrum, Lucienne was a director within the Center for the Development of Therapeutics at the Broad Institute of MIT and Harvard. Prior to her work at the Broad Institute, she was president of research at Catabasis Pharmaceuticals. Previously in her career, Lucienne worked for 10 years at AstraZeneca Boston, where she held positions of increasing responsibility, including global director of translational medicine; local director for the departments of in vivo pharmacology, informatics and disease area research; and the breast disease team scientific lead. Prior to AstraZeneca, she was a senior research investigator at the Pfizer Discovery Technology Center. Lucienne holds a Ph.D. in biological chemistry from the University of California, Los Angeles and completed postdoctoral research at Harvard Medical School.
Vice President, Human Resources
Kim Hazen joined Fulcrum in September 2017 with over 25 years of experience in human resources. Most recently, she was vice president of human resources at Warp Drive Bio, where she led efforts to build a drug discovery culture and core values to match with an emphasis on elite performance. Prior to joining Fulcrum and Warp Drive Bio, Kim served as the executive director of human resources at Aegerion Pharmaceuticals and as head of human resources at the Broad Institute of MIT and Harvard. Before joining the life sciences industry, she worked on the investment side of human resources at Pioneer Investments, Fidelity Investments, Liberty Mutual and Bank of America. Kim received a bachelor’s in psychology from SUNY Potsdam and her MBA from Boston University.
Vice President, Finance and Accounting
Peter Thomson joined Fulcrum in 2018, bringing over 15 years of experience in the life sciences industry. Most recently, he was a consultant for three Third Rock Ventures portfolio companies, where he served as interim head of finance. Before that, he served as head of finance at BIND Therapeutics, where he oversaw all finance and accounting activities, developed the corporate valuation for the company and played an integral role in the company’s sale to Pfizer. Prior to BIND, he was senior director of finance at Ovascience and director of finance at Millennium: The Takeda Oncology Company. In addition, he held positions on the finance teams at Boston Scientific Corporation, Infomedics, Inc., and the Hewlett-Packard Company. Peter earned a bachelor’s from Brown University and an MBA from the University of Chicago Booth School of Business.
L. Andrew Thompson, Ph.D
Vice President, Molecular Innovation and Pharmaceutical Development
L. Andrew (Drew) Thompson joined Fulcrum shortly after launch in summer 2016 and brings more than 20 years of experience in medicinal chemistry to the molecular sciences team. Drew joined Fulcrum from Bristol-Myers Squibb, where he led both early- and full-phase drug discovery programs in both neuroscience and genetically-defined diseases. Teams under Drew’s direction nominated two IND candidates including an allosteric modulator of gamma secretase targeting Alzheimer’s disease and an NR2B-selective NAM for treatment-resistant depression. He also helped establish the discovery chemistry platforms group and served on the leadership team while guiding chemistry efforts around phenotypic screening and target identification. At Fulcrum, Drew leads the molecular sciences effort spanning both preclinical discovery and clinical chemistry, manufacturing, and controls. Drew holds a bachelor’s in chemistry from the University of North Carolina at Chapel Hill and earned his Ph.D. from the University of California, Berkeley.
Aaron Chang, Ph.D.
Executive Director, Computational Biology
Aaron Chang joined Fulcrum in 2016, bringing over 15 years of experience. He was previously executive director of the Center for Computational Biology and Bioinformatics at University of California, San Diego, where he brought together omics analysis, systems biology and cloud computing to over 100 research groups. Aaron was also an associate investigator at the Baylor Institute for Immunology Research, heading a bioinformatics group involved in translational research efforts centered on cancer vaccine and liver transplantation studies. Aaron served as associate director of computational biology at Regulus Therapeutics during its startup phase. His group there identified several key miRNA targets currently in clinical development. At Rosetta Inpharmatics (Merck), he was a member of the pathway informatics team that developed the first in-house systems biology tools for target identification. Aaron was a National Library Medicine Postdoctoral Fellow in Biomedical Informatics at the University of Washington. He has a bachelor’s in biology from the Massachusetts Institute of Technology and completed his Ph.D. in genetics at Harvard University, working on transcription factors governing hematopoiesis.
Executive Director, Strategy & Operations
Kelly Jackson joined Fulcrum in 2016 with over 15 years of experience in biotech and pharmaceutical research, discovery and development. She was previously a director in R&D program leadership and business operations at Biogen. During her tenure, she provided strategic and operational guidance for the business process portfolio and was responsible for the prioritization and collaborative execution of initiatives driving efficiency, compliance and business agility. Kelly has also served as a member of the global clinical trial operations team at Merck, leading both international clinical development projects and transformation initiatives after joining the organization as a clinical research associate. Prior to transitioning to an industry role, she served as the director of research operations at the Mood Disorders Research Program at Case Western Reserve University/University Hospitals of Cleveland. In this role, she led the clinical research infrastructure including investigator-initiated, sponsor-driven, and NIH clinical research protocols. Kelly earned her bachelor’s degree in psychology & criminal justice from Mercyhurst University and a master’s degree in clinical counseling from John Carroll University.
Mark Levin has served as a member of our board of directors since August 2015 and chairman of our board of directors since August 2016. He currently serves as a partner at Third Rock Ventures, LLC, which he co-founded in 2007. Mark served as our interim president and chief executive officer from August 2015 to July 2016 and as interim president and chief executive officer of Voyager Therapeutics, Inc., or Voyager, a biotechnology company, from June 2013 to September 2014. He has served as the chairman of the board of directors of Voyager since June 2013. Mark was formerly on the boards of publicly traded life sciences companies Eleven Biotherapeutics, Inc. from September 2008 to November 2014 and Foundation Medicine, Inc. from 2010 to June 2014.
Mark served as founding chief executive officer of Millennium Pharmaceuticals, Inc., a biopharmaceutical company since acquired by Takeda Pharmaceutical Company Limited, from 1993 to 2005.
Mark received both a bachelor’s and a master’s in chemical and biochemical engineering from Washington University in St. Louis.
Robert J. Gould, Ph.D.
President and Chief Executive Officer, Board Member
Robert J. Gould joined Fulcrum as president and CEO at the time of the company’s launch in 2016, bringing over 30 years of experience to the role. Previously, he served as president and CEO of Epizyme from 2010 to 2015. Prior to joining Epizyme, Robert served as director of novel therapeutics at Broad Institute of MIT and Harvard from 2006 to 2010. Robert spent 23 years at Merck where he held a variety of leadership positions, culminating in the role of vice president, licensing and external research. During his time at Merck, Robert was instrumental in advancing more than 20 compounds from discovery into clinical development in multiple therapeutic areas. Robert received a bachelor’s from Spring Arbor College and a Ph.D. from the University of Iowa and completed postdoctoral studies at Johns Hopkins University.
James J. Collins, Ph.D.
James J. (Jim) Collins is the Termeer Professor of Medical Engineering and Science in the Institute for Medical Engineering and Science and the department of biological engineering at the Massachusetts Institute of Technology (MIT). He is also affiliated with Broad Institute of MIT and Harvard and the Wyss Institute for Biologically Inspired Engineering. He is a renowned expert in synthetic biology and systems biology and their applications in biotechnology and medicine. He has received numerous awards, including a Rhodes Scholarship, a MacArthur Fellowship, and a National Institutes of Health Director’s Pioneer Award. Jim is an elected member of the National Academy of Sciences, the National Academy of Engineering, the National Academy of Medicine, and the American Academy of Arts and Sciences and is a charter fellow of the National Academy of Inventors.
Audit Chair, Board Member
Katina Dorton joined the Fulcrum Board of Directors in January 2020 and serves as the Chair of the Audit Committee. She previously served as Executive Vice President and Chief Financial Officer at Repare Therapeutics, an oncology company focused on DNA damage repair and synthetic lethality. Prior to that, she served as Chief Financial Officer at AVROBIO, a gene therapy company focused on rare diseases. Ms. Dorton has also served as a managing director at Morgan Stanley and Needham & Co. and was an attorney at Sullivan & Cromwell. As an investment banker, she spent more than 15 years advising companies and their boards on capital markets, fund raising, mergers and acquisitions and other strategic transactions. She is also currently on the Board of Directors at US Ecology, an environmental services company. Ms. Dorton received a bachelor’s degree from Duke University, an M.B.A. from George Washington University and a J.D. from the University of Virginia School of Law.
Alan Ezekowitz, MBChB, D.Phil
Alan Ezekowitz was president and CEO of Abide Therapeutics, a company he co-founded in 2011 until its acquisition by H. Lundbeck A/S in May 2019. Formerly, he was the senior vice president and franchise head for disease areas including bone, respiratory, immunology, muscle, urology and dermatology at Merck Research Laboratories. Prior to Merck, Alan was the Charles Wilder Professor of Pediatrics at Harvard Medical School and served as the chief of pediatric services at the Massachusetts General Hospital for Children and the Partners Healthcare System. Additionally, he directed the Laboratory of Developmental Immunology at Massachusetts General Hospital. Alan is a member of the American Society of Clinical Investigation and a fellow of the American Association for the Advancement of Science and has served on multiple National Institutes of Health subcommittees. He was honored by the establishment of the R. Alan Ezekowitz Professorship in Pediatrics at Harvard Medical School.
James (Jim) Geraghty is an industry leader with over 30 years of strategic and leadership experience, centered around biotechnology companies developing and commercializing innovative therapies. Jim served as an entrepreneur in residence at Third Rock Ventures from 2013-2016. He was previously senior vice president, North America strategy and business development at Sanofi, which he joined upon its acquisition of Genzyme. Jim spent almost 20 years at Genzyme, where his roles included senior vice president of international development, president of Genzyme Europe, and founder, president and CEO of Genzyme Transgenics. Jim serves as chairman of the boards of Idera Pharma, Pieris Pharma, and Orchard Therapeutics, and on the board of Voyager Therapeutics. He started his career in healthcare strategy consulting at Bain and Company. A graduate of the Yale Law School, he holds a master’s degree from the University of Pennsylvania and a bachelor’s from Georgetown University.
Kate Haviland serves as the chief operating officer at Blueprint Medicines, a publicly traded precision therapy biopharmaceutical company. Kate is a member of the executive leadership team responsible for the business development, alliance management, corporate affairs, commercial strategy and operations, new product planning, program management and information technology functions. Prior to joining Blueprint in 2016, Kate was the vice president of rare diseases and oncology program leadership at Idera Pharmaceuticals. In this role, she oversaw all aspects of the product development and new product planning strategy for the company’s oncology and rare disease pipeline programs.
Previously, Kate worked at Sarepta Therapeutics as the head of commercial development, at PTC Therapeutics in commercial development as the product lead for Translarna, and at Genzyme in corporate development, before moving into program management for two enzyme replacement therapies in the personalized genetic health business unit. Before joining Genzyme, Kate spent five years as a life science strategy consultant. Kate is a member of the Corporate Advisory Council for the National Tay-Sachs and Allied Disease Association, is an advisory board member at Peeled Snacks Inc., a consumer health food company, and is a member of the inaugural class of Women in Bio’s Boardroom Ready Program, the BioPharma Executive Council and the Harvard Business School Healthcare Alumni Association. Kate holds a bachelor’s degree from Wesleyan University with a double major in biochemistry and economics and an MBA from Harvard Business School.
Peter L. Jones, Ph.D.
Mick Hitchcock, Ph.D. Endowed Chair of Medical Biochemistry, University of Nevada, Reno
Peter L. Jones is an expert in epigenetic mechanisms of gene regulation with a focus on muscle development and disease. He is a leader in understanding the epigenetic disruption and pathogenic mechanisms underlying facioscapulohumeral muscular dystrophy (FSHD). Peter currently holds the Mick Hitchcock, Ph.D. Endowed Chair in Medical Biochemistry and serves as an associate professor of pharmacology at the Center for Molecular Medicine at the University of Nevada, Reno School of Medicine. In addition, he is an investigator with the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center in Seattle. Previously in his career, Peter was an associate professor at the University of Massachusetts Medical School and principal scientist at the Boston Biomedical Research Institute, a private research institute focused on fundamental questions in muscle biology. Prior to that, he was an assistant professor at the University of Illinois at Urbana-Champaign. Peter received his bachelor’s degree in microbiology from Miami University in Oxford, Ohio and his Ph.D. in genetics and molecular biology from Emory University. He completed his postdoctoral training in biochemistry and chromatin biology with Dr. Alan Wolffe in the Laboratory of Molecular Embryology at the National Institutes of Health and the Eunice Shriver National Institute of Child Health and Human Development.
Leslie Leinwand, Ph.D.
Chief Scientific Officer, BioFrontiers Institute, University of Colorado, Boulder
Leslie Leinwand is a renowned muscle physiologist with broad expertise in the genetic underpinnings of heart and skeletal muscle defects. She is the chief scientific officer of the BioFrontiers Institute at the University of Colorado and established and co-directs the University of Colorado’s Cardiovascular Research Institute (CU-CVI), a collaborative group of physicians, molecular biologists and geneticists. The team works to integrate research and clinical applications, as well as initiate effective treatment programs and preventive therapies. Leslie’s research focuses on how cardiac and skeletal muscle adapt to stimuli, particularly the pathways involved in heart enlargement in response to exercise and disease. She is a Howard Hughes Medical Institute Professor. Leslie has authored more than 350 scientific publications and founded three biotechnology companies: Myogen Inc., Hiberna, Inc. and MyoKardia, Inc. Myogen, which was a publicly traded company, was acquired by Gilead in 2006. MyoKardia, which is publicly traded, has a small molecule in clinical trials for inherited cardiomyopathy. Leslie received her bachelor’s degree from Cornell University and her Ph.D. at Yale University and completed her postdoctoral training at Rockefeller University.
H. Lee Sweeney, Ph.D.
Director, Myology Institute, University of Florida
H. Lee Sweeney is the director of the Myology Institute at University of Florida (UF), where he is also a professor of pharmacology and therapeutics. Lee is one of the world’s premier neuromuscular disease researchers, with expertise across cardiovascular disease, skeletal muscle disorders and molecular biology – specifically the molecular motors of the myosin superfamily. He is known for creating the first visualization of structural rearrangement of the myosin lever arm, as well as for his use of gene therapy to block the loss of age-related muscle size and strength in mice. Prior to joining the faculty at UF, Lee was the William Maul Measey Professor and chairman of physiology at the University of Pennsylvania, where he is now an emeritus professor of physiology. He was elected as a fellow of the American Heart Association in 2001, a fellow of the Biophysical Society in 2017, and has been the director of a Paul Wellstone Muscular Dystrophy Cooperative Center since 2005. Lee has a bachelor’s degree in biochemistry from the Massachusetts Institute of Technology, a master’s in physiology from Harvard University and a Ph.D. in physiology and biophysics from Harvard University.
Steve Warren, Ph.D.
Chairman, Department of Human Genetics, Emory University
Steve Warren has made seminal discoveries related to Fragile X syndrome (FXS) and its potential treatment. Steve led an international group that first isolated the FMR1 gene responsible for FXS in 1991 and is now a leader in the effort to identify new genomically focused therapeutic approaches to treating this condition. Steve currently serves as William Patterson Timmie Professor of Human Genetics, Charles Howard Candler Chair in Human Genetics, and the founding chair of the department of human genetics at Emory University. He also holds secondary appointments in the departments of pediatrics and biochemistry. In addition to his groundbreaking work in the field, Steve is past president of the American Society of Human Genetics and past editor-in-chief of The American Journal of Human Genetics. He is an elected member of the National Academy of Sciences, the National Academy of Medicine, and the National Academy of Arts and Sciences. Steve received his Ph.D. in human genetics from Michigan State University in 1981 and completed his postdoctoral training at the University of Illinois College of Medicine.
Marius Wernig, M.D., Ph.D.
Associate Professor of Pathology and Chemical and Systems Biology, Stanford University
Marius Wernig is an expert in the molecular mechanisms that determine specific cell fates, and his laboratory recently identified a pool of transcription factors that are sufficient to convert skin fibroblasts directly into functional neuronal cells termed induced neuronal (iN) cells. Marius currently serves as associate professor of pathology and chemical and systems biology in the Institute for Stem Biology and Regenerative Medicine at Stanford University. He earned his M.D. and Ph.D. from the Technical University of Munich. He completed clinical training in neuropathology and general pathology at the University of Bonn and was a postdoctoral fellow at the Whitehead Institute for Biomedical Research.
Rabi Tawil, M.D.
University of Rochester Medical Center
Rabi Tawil co-directs the MDA Neuromuscular Disease Clinic at the University of Rochester Medical Center (URMC) and is a professor in the department of neurology and the department of pathology and laboratory medicine at URMC. Rabi helped establish a collaborative effort of clinicians and basic researchers that led to a unified understanding of the molecular mechanisms of facioscapulohumeral muscular dystrophy (FSHD). He has also conducted translational research on the non-dystrophic myotonias, the periodic paralyses and other neuromuscular disorders. He is the URMC director of the Richard Fields Center for FSHD Research, and in collaboration with Dr. Jeffrey Statland established the FSHD Clinical Trial Research Network. Rabi is board certified by the American Board of Psychiatry and Neurology and holds over a dozen patents in methods related to the study of FSHD. Rabi holds an M.D. from the American University of Beirut Faculty of Medicine in Beirut, Lebanon and completed his residency in neurology and fellowship in neuromuscular diseases at the University of Rochester Medical Center.
Jeffrey Statland, M.D.
University of Kansas Medical Center
Jeffrey Statland is an associate professor of neurology at the University of Kansas Medical Center (KUMC), where he coordinates the facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network. Jeffrey is a renowned FSHD researcher and is a current investigator on studies of FSHD, Duchenne muscular dystrophy, spinal muscular dystrophy and myotonic atrophy. He is particularly interested in developing new disease-relevant outcome measures to assess patient-reported disease burden. Jeffrey is a member of the American Academy of Neurology as well as an ad-hoc reviewer of Neurology and several other academic journals. He received his M.D. from KUMC, where he completed residency in the department of neurology. Jeffrey also completed a fellowship in experimental therapeutics in the department of neurology at the University of Rochester Medical Center (URMC) and was a Howard Hughes Medical Institute research scholar.
Stephen Tapscott, M.D., Ph.D.
University of Washington
Stephen Tapscott is a professor in the department of neurology at the University of Washington and a member of the human biology and clinical research divisions at the Fred Hutchinson Cancer Research Center (FHCRC). As principal investigator of the Tapscott Lab, he leads research on the regulation of gene expression during cell differentiation, the role of triplet and microsatellite repeats in the genome, epigenetic regulation in cancer and the development of cellular and genetic therapies for muscular dystrophies. Stephen serves on the scientific advisory boards of the American Brain Tumor Association and the Max Planck Institute for Heart and Lung Research and is a member of the advisory council at the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Stephen earned his M.D. and Ph.D. from the University of Pennsylvania, where he also completed a medical internship and neurology residency. He completed postdoctoral research with Dr. Harold Weintraub at the FHCRC.
Baziel van Engelen, M.D., Ph.D.
Radboud University Nijmegen Medical Centre
Baziel van Engelen is a professor of neuromuscular disorders at the Radboud University Nijmegen Medical Centre (RUNMC), where he co-coordinates the facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network. He has coauthored numerous studies of myotonic dystrophies, FSHD and inflammatory myopathies. Baziel is a member of the scientific advisory board of the European Neuromuscular Center and serves on the research committee of Euro-NMD, a European reference network for the thematic grouping of rare neuromuscular diseases. He received his M.D. from RUNMC and his Ph.D. from the University of Amsterdam and completed his neurology residency at Radboud University and the University of Berlin. He later completed a research fellowship in the department of neurology at the Mayo Clinic in Rochester, Minnesota.
Silvere van der Maarel, Ph.D.
Silvere van der Maarel is a professor of medical epigenetics at Universiteit Leiden, where he is also chair of the department of human genetics. He studies the role of epigenetic changes in rare diseases, with a particular focus on facioscapulohumeral muscular dystrophy (FSHD). Silvere is a leading authority on the genomics of FSHD and has also made research contributions to other muscular dystrophies, including oculopharyngeal muscular dystrophy and limb-girdle muscular dystrophy. He is a co-founder of the Richard Fields Center for FSHD Research, an international collaboration to accelerate research on and treatment of FSHD. In 2016, he received the FSH Society Pioneer Award. Silvere has a Ph.D. in human genetics from the Radboud University Nijmegen Medical Centre. He conducted postdoctoral research at the Max Planck Institute of Molecular Genetics in Berlin, Germany.
Bradley E. Bernstein, M.D., Ph.D.
Professor of Pathology, Massachusetts General Hospital and Harvard Medical School
Bradley E. (Brad) Bernstein is a professor of pathology at Massachusetts General Hospital and Harvard Medical School, and director of the Epigenomics Program at the Broad Institute of MIT and Harvard. His research focuses on epigenetic gene regulation. His lab studies show how gene activity is controlled by noncoding regulatory elements called ‘enhancers’, and by the way the genes are packaged into chromatin. He is a leader of the National Institutes of Health (NIH)’s ENCODE project, which is mapping the locations and functions of all noncoding elements in the human genome. His work is notable for the identification of specialized chromatin structures that underlie stem cell pluripotency, enhancers associated with autoimmunity and other diseases, and specific epigenetic aberrations that lead to cancer. Brad received his bachelor’s from Yale University and his M.D. and Ph.D. from the University of Washington, before completing a residency in clinical pathology and postdoctoral research at Harvard University. Honors include an Early Career Scientist Award from the Howard Hughes Medical Institute, a Career Award in the Biomedical Sciences from the Burroughs Wellcome Fund, the NIH Director’s Pioneer Award, an American Cancer Society Professorship and the Paul Marks Prize for Cancer Research.
Michael R. Green, M.D., Ph.D.
Co-Director, The Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School
In 1990, Michael R. Green joined the Program in Molecular Medicine at the University of Massachusetts Medical School and served as the director of the Program of Gene Function and Expression from 1999 to 2014. He is also the vice provost for strategic research initiatives, chair and professor in the department of molecular, cell and cancer biology and the director of the Cancer Center. He has made pioneering contributions to the understanding of the mechanisms that regulate gene expression in eukaryotes and how alterations in gene expression contribute to cancer and other human diseases. Recently he has used transcription-based approaches and functional screens to identify new genes and regulatory pathways involved in epigenetic regulation and cancer, which have important therapeutic implications. As a young investigator he was the recipient of the Presidential Young Investigators Award (1985), the Searle Scholar Award (1986), the McKnight Neuroscience Award (1991), and was a Harvey Lecturer (1993). He is an elected member of the National Academy of Sciences, the National Academy of Medicine, the American Academy of Arts and Sciences, and the European Molecular Biology Organization. Michael received his M.D. and Ph.D. from Washington University School of Medicine in 1981, and carried out postdoctoral work at Harvard University, where he joined as a faculty member in 1984.
Rudolf Jaenisch, M.D.
Professor of Biology, MIT; Founding Member, Whitehead Institute for Biomedical Research
Rudolf Jaenisch joined the Massachusetts Institute of Technology in 1984. His central research interest is to understand epigenetic regulation of gene expression in mammalian development and disease. He is a pioneer in the understanding of how to transdifferentiate cells from one type to another. Rudolf’s work with embryonic stem cells is providing fundamental discoveries for the field of regenerative medicine. His numerous awards and honors include the Genetics Prize from the Gruber Foundation (2001), election to the National Academy of Sciences (2003), the Max Delbruck Medal (2006), the National Medal of Science (2010), the Wolf Prize in Medicine (2011) and the Otto Warburg Medal (2014). Earlier in his career, Rudolf held research positions at Princeton University, the Fox Chase Institute for Cancer Research and the Salk Institute. From 1977 to 1984, he was the head of the department of tumor biology at the Heinrich Pette Institute at the University of Hamburg. Rudolf earned his M.D. from the University of Munich in 1967 and conducted postdoctoral work in bacteriophages at the Max Planck Institute.
Jeannie T. Lee, M.D., Ph.D.
Professor of Genetics and Pathology, Harvard Medical School
Jeannie T. Lee is a professor of genetics (pathology) at Harvard Medical School, the Blavatnik Institute, and the Massachusetts General Hospital (MGH). Jeannie specializes in the study of epigenetic regulation by long noncoding RNAs and uses X-chromosome inactivation as a model system. In the Lee Lab, growing knowledge of X-inactivation mechanisms and RNA biology is being translated to treat various human diseases, including Rett, Fragile X, and CDKL5 Syndromes. She is a member of the National Academy of Sciences, the 2016 recipient of the Lurie Prize from the Foundation for the National Institutes of Health, a 2016 awardee of the Centennial Prize from the Genetics Society of America, the 2010 recipient of the Molecular Biology Prize from the National Academy of Sciences and a Fellow of the American Association for the Advancement of Science. Jeannie was also named a Distinguished Graduate of the University of Pennsylvania School of Medicine in 2013 and an Investigator of the Howard Hughes Medical Institute. From 2013-2018, she co-launched the Epigenetics Initiative at Harvard Medical School and served as its co-director. Serving on the board of directors of the Genetics Society of America (GSA), Jeannie spearheaded The All-Genetics Conference (TAGC) in 2016. As GSA’s president, Jeannie established a strategic plan and a development strategy for the society in 2018. She received her bachelor’s in biochemistry and molecular biology from Harvard University and obtained M.D.-Ph.D degrees from the University of Pennsylvania School of Medicine. Jeannie then carried out postdoctoral work at the Whitehead Institute and the Massachusetts Institute of Technology and became chief resident of clinical pathology at MGH prior to joining the faculty at Harvard Medical School. As a new investigator, she received the Basil O’Connor Scholar Award from the March of Dimes and the Pew Scholars Award.
Danny Reinberg, Ph.D.
Professor of Biochemistry and Molecular Pharmacology, New York University School of Medicine
Danny Reinberg is the Terry and Mel Karmazin Professor of Biochemistry and Molecular Pharmacology in the department of biochemistry and molecular pharmacology at New York University School of Medicine. He is also an investigator of the Howard Hughes Medical Institute. Danny is a world-recognized leader in the fields of mammalian transcriptional regulation, chromatin dynamics and epigenetic regulation. His numerous advances are detailed in over 180 publications and his key contributions include identifying the structural and functional characterization of critical components of the human gene expression machinery; discovering several chromatin remodeling factors and key insights into the mechanisms by which genes are activated and repressed in the context of chromatin; and purifying and characterizing several histone methyltransferases and chromatin modifying complexes that have shed great insight into the regulatory interplay between histone modification and gene expression. He is an elected member of the American Academy of Arts and Sciences (2012), the National Academy of Medicine (2013) and the National Academy of Sciences (2015). He has served as an American Association for the Advancement of Science Fellow (2015) and the International Blaise Pascal Chair at the Curie Institute in Paris, France (2017). He is a recipient of the Albert Einstein Distinguished Ph.D. Alumnus Award (2014). Danny received his bachelor’s in biochemistry from Catholic University in Valparaiso, Chile and a Ph.D. in molecular biology from the Albert Einstein College of Medicine.