As Fulcrum advances its lead programs in Fragile X syndrome (FXS) and facioscapulohumeral muscular dystrophy (FSHD) it is establishing CNS and muscular dystrophy franchises.  

The Scientific Advisory Board brings expertise in disease, biology, and physiology in order to advance new programs through drug discovery and development and to deliver on the promise of genomic medicine. 


Peter L. Jones, Ph.D.

Mick Hitchcock Endowed Chair of Medical Biochemistry and Associate Professor of Pharmacology at the Center for Molecular Medicine, University of Nevada, Reno School of Medicine

Peter L. Jones is an expert in epigenetic mechanisms of gene regulation with a focus on muscle development and disease.  Peter is a leader in understanding the epigenetic disruption and pathogenic mechanisms underlying Facioscapulohumeral Muscular Dystrophy (FSHD). Peter received his Bachelor’s degree in Microbiology from Miami University (Ohio) and his Ph.D. in Genetics and Molecular Biology from Emory University. He completed his postdoctoral training 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. Peter currently serves as an Associate Professor of Pharmacology at the Center for Molecular Medicine at the University of Nevada. Previously in his career, Peter served as an affiliate investigator with the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center for FSHD and was a 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 in the Department of Cell and Developmental Biology.  Most recently, Peter was an Associate Professor in the Departments of Cell and Developmental Biology and Neurology at the University of Massachusetts Medical School.  

 

Leslie Leinwand, Ph.D.

Professor of the Department of Molecular, Cellular and Developmental Biology and Chief Scientific Officer of the BioFrontiers Institute at the University of Colorado, Boulder; Howard Hughes Medical Investigator Professor

Leslie Leinwand is a renowned muscle physiologist with broad expertise into the genetic underpinnings of heart and skeletal muscle defects.  Leslie received her Bachelor’s degree from Cornell University and her Ph.D. at Yale University, and completed her postdoctoral training at Rockefeller University. She was Chair of the Department of Molecular, Cellular and Developmental Biology at the University of Colorado. She 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. Her 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 the Chief Scientific Officer of the BioFrontiers Institute at the University of Colorado and a Howard Hughes Medical Investigator Professor. Leinwand has authored more than 300 scientific publications and founded three biotechnology companies, Myogen Inc., Hiberna, Inc. and MyoKardia, Inc. Myogen, 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.

 

Steve Warren, Ph.D.

Chairman of the Department of Human Genetics and Professor of Biochemistry and Pediatrics at Emory University

Steve Warren has made seminal discoveries related to FXS and its potential treatment. Steve led an international group that first isolated the FMR1 gene responsible for Fragile X syndrome in 1991, and is now a leader in the effort to identify new genomically focused therapeutic approaches to treating this condition. 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. 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.  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 Science, the National Academy of Medicine, and the National Academy of Arts and Sciences.

 

Marius Wernig, M.D., Ph.D.

Associate Professor of Pathology and Chemical and Systems Biology and Institute for Stem Biology and Regenerative Medicine, 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 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. 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.