About Skyhawk
We discover and develop small molecules that modify RNA expression
Our Mission
Our mission is to revolutionize disease treatment by developing
small molecule therapeutics that modify RNA expression.
RNA mis-expression has been found to cause a growing list of diseases, from orphan and neurological conditions to major cancers. Very few treatments are available for these challenging conditions, many of which have been considered “undruggable.”
Our founders and Scientific Advisory Board are major figures in disease biology and immunology, and launched Skyhawk to harness the power of the immune system to provide breakthrough treatments for patients.
Leadership Team
Bill Haney
Co-Founder & Chief Executive Officer
An inventor and entrepreneur, Bill Haney started his first company as a college freshman, inventing and building air pollution control systems for power plants. Since then he has started or helped start more than a dozen technology companies.​ Bill was a founding member of the national environmental advisory board for the US Environmental Protection Agency, the President's Circle for the National Academy of Sciences, has won a Humanitarian Award from Harvard Medical School, an Achievement Award from the ACLU and serves or has served on boards for Harvard, MIT, State and Federal Government agencies, and the World Resources Institute.​ Bill holds a BA from Harvard College and was a Kennedy School Fellow from 1997-2001.
Sergey Paushkin, M.D., Ph.D.
Co-Founder & Head of R & D
Sergey Paushkin, M.D., Ph.D., is a scientific co-founder of Skyhawk. He has more than 20 years of experience in the discovery and development of small molecules targeting RNA including the first approved small molecule to modulate RNA splicing, Evrysdi (risdiplam), for treatment of Spinal Muscular Atrophy (SMA). Prior to Skyhawk, Sergey was Director of Research at the SMA Foundation, where he managed the Foundation’s drug discovery & development collaborations with pharma, biotech, academic, clinical, and CRO groups to bring first-in-class therapeutics including Evrysdi, Spinraza, Zolgensma to patients. Earlier, at PTC Therapeutics, Sergey started the SMA program; there he also discovered the human tRNA splicing endonuclease complex described in his Cell and Nature publications.
Dr. Paushkin holds a medical degree from Russian State Medical University and PhD in Biochemistry from the Cardiology Research Center, Moscow. His undergraduate research was focused on the molecular bases of prion diseases. Sergey received post-doctoral training from Dr. Gydeon Dreyfuss Laboratory at HHMI/ University of Pennsylvania where he studied the SMN complex, splicing machinery, and RNA binding proteins. He authored many publications in top-tier journals including a publication in Science covering his work on the discovery of risdiplam. He is also a co-editor of the first SMA textbook.
Kathleen McCarthy
Co-Founder & Distinguished Senior Scientist
Kathleen is a leading expert in developing small molecules that target RNA-splicing. She has more than 15 years of experience in the discovery and development of small molecules targeting RNA including the first approved small molecule to modulate RNA splicing, Evrysdi (risdiplam), for treatment of Spinal Muscular Atrophy (SMA). 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 this first-ever small molecule therapeutic driving mRNA alternative splicing, to clinical trials. The drug candidate was bought by Roche in 2011 and Kathleen moved to Roche to work as a lead pre-clinical scientist. Kathleen first- and last-authored critical papers in the field, including the Science paper (2014) where the SMA small molecule targeting RNA splicing was heralded as a first-in-class small molecule therapeutic that specifically corrects exon skipping, and the 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.
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Scientific Advisory Board
Tyler Jacks, Ph.D.
Chair, Skyhawk Scientific Advisory Board | President of Break Through Cancer | Founding Director, Koch Institute for Integrative Cancer Research | Professor of Biology at MIT | HHMI Investigator
Prof. Tyler Jacks is the founding Director of the Koch Institute for Integrative Cancer Research at MIT – a National Cancer Institute (NCI)-designated Cancer Center. His lab uses cutting-edge technologies to study cancer genetics and cancer immunology, with discoveries that led to the founding of Dragonfly.​ 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. Jacks 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. Jacks served as Chair of the National Cancer Advisory Board at the National Cancer Institute (NCI), was co-chair of the White House's Cancer "Moonshot" Blue Ribbon Panel.​ was a member of the American Association for Cancer Research (AACR) Board of Directors, and was elected President of the AACR in 2009. He is the president of Break Through Cancer Foundation. Jacks has a bachelor’s degree in Biology from Harvard, and a Ph.D. from UC San Francisco.
Phillip A. Sharp, Ph.D.
Skyhawk Scientific Advisor | 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.
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 | President & CEO of 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
Rob Hershberg, M.D., Ph.D.
Skyhawk Scientific Advisory Board | Venture Partner, Frazier Life Sciences Team
Dr. Rob Hershberg began his career as an Assistant Professor at Harvard Medical School and an Associate Physician at Brigham and Women’s Hospital in Boston. Later, Dr. Hershberg co-founded VentiRx Pharmaceuticals and, as President and Chief Executive Officer, led the company through its transformational partnership with Celgene. Dr. Hershberg joined Celgene in 2014 to lead their efforts in Immuno-Oncology, was promoted to Chief Scientific Officer in 2016, and was subsequently Executive Vice President and Head of Business Development & Global Alliances and served as a member of the Executive Committee until the acquisition of Celgene by Bristol-Myers Squibb in 2019. Rob is currently a Venture Partner on the Frazier Life Sciences team. He completed his undergraduate and medical degrees at the University of California, Los Angeles and received his Ph.D. at the Salk Institute for Biological Studies.
Jeannie T. Lee, M.D., Ph.D.
SSkyhawk 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.
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.
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.
Ed Wild
Skyhawk Scientific Advisory Board Professor, Professor of Neurology, University College London
Prod Wild is a Professor of neurology at University College London, Associate Director of UCL’s world-leading Huntington’s Disease Centre, and a Consultant Neurologist at the National Hospital for Neurology & Neurosurgery in London’s Queen Square. He is a leading expert in clinical care, therapeutic development, clinical trial design and conduct, and biomarkers for Huntington’s disease. He pioneered the study of mutant huntingtin and neurofilment light proteins to empower therapeutic development in HD and is the chief investigator of the HDClarity study, the largest ever study of cerebrospinal fluid in HD. He has authored over 200 peer-reviewed scientific publications and received the Huntington’s Disease Society of America Researcher of the Year Award, and Huntington Society of Canada Community Leadership Award. He co-founded HDBuzz, the leading source of accessible, impartial research news for the HD community.
Amanda Garner, Ph.D.
Skyhawk Scientific Advisory Board Professor, University of Michigan
Amanda L. Garner is the Charles Walgreen, Jr. Professor in the Department of Medicinal Chemistry and Director of the Interdepartmental Program in Medicinal Chemistry at the University of Michigan. She received her Ph.D. in Chemistry from the University of Pittsburgh and completed NIH-funded postdoctoral studies at The Scripps Research Institute. Dr. Garner began her independent career in 2013 at the University of Michigan and her research integrates chemical biology, medicinal chemistry, and molecular and cellular biology approaches for early-stage drug discovery efforts with a primary focus on validating new therapeutic targets in RNA biology. For her research accomplishments, she was awarded Catalyst Awards from the Dr. Ralph and Marian Falk Medical Research Trust in 2016 and 2022, named a Rogel Scholar from the Rogel Cancer Center in 2022, awarded the David W. Robertson Award from the ACS Division of Medicinal Chemistry in 2022, and Ono Pharma Breakthrough Science Initiative Award in 2023.
Our Partnerships
Skyhawk’s Pharma collaborations have contributed biology insights, commercial perspectives and upfront payments of over $525 million to develop targeted drug candidates – driving growth in Skyhawk’s platform.
Our Investors
Skyhawk is financed with the capital of leading long-term investors whose focus is on the
long-term value that comes from doing high quality, patient-focused drug development..
