Kathleen McCarthy | Skyhawk Co-Founder |
Chief Scientific Officer
Kathleen began her career by co-developing a treatment for Spinal Muscular Atrophy (SMA) as a pre-clinical scientist at the SMA Foundation. In this role she helped bring the first-ever small molecule therapeutic driving mRNA alternative splicing, to clinical trials in SMA. This drug candidate was bought by Roche in 2011 and Kathleen moved to Roche to work as a lead pre-clinical scientist. The SMA drug is now in pivotal clinical trials. Kathleen first-authored a Nature Communications paper describing the SMA compound mechanism (2017). Prior to her work in drug discovery and development, Kathleen completed a Fulbright Fellowship at the Swiss Federal Institute of Technology, ETH, and graduated with honors in Chemistry from Wellesley College.
SCIENTIFIC ADVISORY BOARD
Tyler Jacks, Ph.D. | Chair, Skyhawk Scientific Advisory Board Director, Koch Institute for Integrative Cancer Research | Professor of Biology at MIT | HHMI Investigator
Prof. Jacks is Director of the Koch Institute for Integrative Cancer Research at MIT – a National Cancer Institute (NCI)-designated Cancer Center, where he also directs a major research laboratory carrying out cutting-edge studies in cancer genetics and cancer immunology. Prof. Jacks has pioneered the use of gene targeting technology to study cancer-associated genes and to construct mouse models of many human cancer types, closely recapitulating human disease and yielding novel insights into tumor development as well as new strategies for cancer detection and treatment. Prof Jacks has served as Chair of the National Cancer Advisory Board of the NCI, was a member of the Board of Directors of the American Association for Cancer Research (AACR), and is a past President of the AACR. He was elected to the National Academy of Sciences, the Institute of Medicine of the National Academies, the American Academy of Arts and Sciences and the inaugural class of Fellows of the AACR Academy. Prof Jacks was co-chair of the White House's Cancer "Moonshot" Blue Ribbon Panel. He has a bachelor’s degree in Biology from Harvard, and a Ph.D. from UC San Francisco, where he trained with Nobel Laureate Harold Varmus.
Frédéric Allain, Ph.D. | Skyhawk Scientific Advisory Board Professor, Institute of Molecular Biology and Biophysics at ETH, Zurich, Switzerland
Prof. Frédéric Allain is an expert in the role of RNA in disease, using NMR technology to elucidate the structure of protein-RNA complexes. He and his research team use biophysical tools to study protein RNA interactions, with a focus on the notable segment of genetic diseases that originate from a post-transcriptional mis-regulation of gene expression often caused by splicing, RNA editing or translation defects. Prof. Allain is a specialist in investigating the molecular mechanism in drug development programs for Spinal Muscular Atrophy (SMA), Myotonic Dystrophy (DM) and Amyotrophic Lateral Sclerosis (ALS). He received his Ph.D. from Cambridge University, U.K.
Steven Bell, Ph.D.| Skyhawk Scientific Advisory Board | Professor of Biology at MIT | HHMI Investigator
Prof. Steven Bell is a leading expert in the study of DNA replication, with an emphasis on how assembly of enzymes are regulated during the cell cycle to ensure genomic maintenance. He is Professor of Biology at MIT, an Investigator at the Howard Hughes Medical Institute, a member of the Koch Institute for Integrative Cancer Research. Prof. Bell is an elected member of the National Academy of Sciences. He has authored over 100 peer-reviewed publications, and received his Ph.D. at the University of California, Berkeley.
Ben Blencowe, Ph.D., FRS | Skyhawk Scientific Advisory Board | Donnelly Centre and Department of Molecular Genetics, University of Toronto
Prof. Ben Blencowe is an internationally recognized RNA biologist who has made pioneering contributions to the understanding of the molecular mechanisms controlling alternative splicing and their roles in evolution, development and disease. He holds the Banbury Chair of Medical Research and is Professor in the Donnelly Centre at the University of Toronto; he also serves as Director of the Donnelly Sequencing Centre. Prof. Ben Blencowe has received numerous awards and honours for his research excellence and was recently elected Fellow of the Royal Society (UK). He conducted his Ph.D. research at the European Molecular Biology Laboratory in Heidelberg.
Ben Ebert, M.D., Ph.D.| Skyhawk Scientific Advisory Board | Professor of Medicine at Harvard Medical School | Chair of Medical Oncology at the Dana-Farber Cancer Institute | HHMI Investigator
Dr. Ben Ebert focuses on the genetics, biology, and therapy of myeloid malignancies. His work has led to the characterization of clonal hematopoiesis as a pre-malignant state for hematologic malignancies, and elucidation of the mechanism of action of lenalidomide and related molecules that induce degradation of specific proteins. Dr. Ebert has served as president of the American Society for Clinical Investigation and is an elected member of the National Academy of Medicine and the Association of American Physicians. Dr. Ebert has a Ph.D. from Oxford University (Rhodes Scholar), a M.D. from Harvard Medical School, and did his residency in internal medicine at Massachusetts General Hospital and a fellowship in hematology/oncology at the Dana-Farber Cancer Institute.
Adrian Krainer, Ph.D. | Skyhawk Scientific Advisory Board Professor, Cold Spring Harbor Laboratory
Prof. Adrian Krainer is an expert on the role of RNA splicing proteins in cancer, neurological and rare diseases. His lab is a leader in understanding the mechanisms of RNA splicing, and the means by which faulty splicing can be corrected. Prof. Krainer has published widely on RNA splicing in Spinal Muscular Atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death in infants. He was elected to the American Academy of Arts and Sciences for his work in this field and serves as a board member and advisor for multiple companies and organizations. Professor Krainer received his PhD from Harvard University.
Jeannie T. Lee, M.D., Ph.D. | Skyhawk Scientific Advisory Board | Professor of Genetics and Pathology at Harvard Medical School, the Blavatnik Institute, and the Massachusetts General Hospital | HHMI Investigator
Prof. Jeannie Lee specializes in the study of epigenetic regulation by long noncoding RNAs and uses X-chromosome inactivation as a model system. She also translates basic knowledge to find treatments for genetic disorders and co-founded two publicly traded companies — Translate Bio and Fulcrum Therapeutics. Prof. Lee is a Member of the National Academy of Sciences, a 2018 Harrington Rare Disease Scholar, the 2016 recipient of the Lurie Prize, a 2016 recipient of the Centennial Award from the Genetics Society of America, the 2010 awardee of the Molecular Biology Prize from the National Academy of Sciences, and a Fellow of the American Association for the Advancement of Science. She received her A.B. in Biochemistry and Molecular Biology from Harvard University and obtained M.D.-Ph.D degrees from the University of Pennsylvania School of Medicine.
Jacqueline A. Lees, Ph.D. | Skyhawk Scientific Advisory Board | Associate Director, Koch Institute for Integrative Cancer Research | Professor of Biology at MIT
Prof. Jackie Lees’ research program focuses on proteins and pathways that control cellular proliferation, terminal differentiation and/or apoptosis, and are frequently deregulated in tumor cells – understanding the role of these proteins in normal cell biology and development will help establish how their deregulation contributes to tumor development and metastasis. Prof. Lees is Associate Director of the Koch Institute, Professor of Biology at MIT, and a Daniel K. Ludwig Scholar. She received her Ph.D. degree in Biochemistry from the University of London and was a postdoctoral fellow in the laboratory of Ed Harlow at Massachusetts General Hospital Cancer Center and Harvard Medical School.
Friedrich Metzger, Ph.D. | Skyhawk Scientific Advisory Board | Professor of Neurobiology at the University of Freiburg, Germany
Prof. Metzger is a leading scientist in RNA-based disease and neurological conditions. As project leader and Head of Discovery Rare Diseases at Roche for 15 years, he ran drug discovery programs for Alzheimer’s, ALS, Parkinson’s, Duchenne Muscular Dystrophy (DMD) & Spinal Muscular Atrophy (SMA). Prof. Metzger scientifically supervised Roche’s SMA splicing modifier program, currently in pivotal clinical trials, and championed two publications describing its molecular mechanism, in Nature in 2017 and Science, in 2014. He currently serves as Professor in Neurobiology at the University of Freiburg, Germany. He received a Ph.D. in Pharmacology at University of Tübingen, Germany.
Phillip A. Sharp, Ph.D. | Skyhawk Scientific Advisory Board | Institute Professor, Massachusetts Institute of Technology | Professor of Biology and Member, Koch Institute
Prof. Phillip Sharp’s research interests have centered on the molecular biology of gene expression relevant to cancer and the mechanisms of RNA splicing. Sharp’s landmark work in 1977 provided the first indications of “discontinuous genes” in mammalian cells. The discovery fundamentally changed scientists’ understanding of gene structure and earned him the 1993 Nobel Prize in Physiology or Medicine. Prof. Sharp is an elected member of the National Academy of Sciences, the Institute of Medicine, the American Academy of Arts and Sciences, the American Philosophical Society, and the Royal Society, UK. Among his many awards are the Gairdner Foundation International Award, the Lasker Basic Medical Research Award and the National Medal of Science. Prof. Sharp earned a Ph.D. in chemistry from the University of Illinois, Champaign-Urbana. He is a co-founder of Biogen and Alnylam Pharmaceuticals, Inc.
Maurice Swanson, Ph.D. | Skyhawk Scientific Advisory Board
Professor, Department of Molecular Genetics and Microbiology at the University of Florida College of Medicine | Associate Director of the Center for NeuroGenetics
Prof. Maury Swanson is an expert on the regulation of RNA alternative processing during mammalian development and how this regulation is disrupted in neurological and neuromuscular diseases, including some types of muscular dystrophy and amyotrophic lateral sclerosis (ALS). He is a Professor in the Department of Molecular Genetics and Microbiology at the University of Florida College of Medicine and Associate Director of the Center for NeuroGenetics. His lab focuses on the functions of repetitive DNA elements, particularly microsatellites or short tandem repeats (STRs), in RNA-mediated disorders. An important objective of these studies is to enhance tissue regeneration following treatment modalities designed to block the toxicity of STR RNAs. Prof. Swanson received his Ph.D. from the University of California, Berkeley.