Computational RNA Tools For Drug Discovery

At Skyhawk Therapeutics we use the latest structural, kinetic, and computational technologies to discover small molecules targeting RNA technologies to discover small molecules targeting RNA



Skyhawk's founders are leading scientists in the development of small molecule therapeutics that correct RNA expression.


Our proprietary technology enables the rational design of small molecules that target the RNA splicing process.

We are now developing drug candidates for cancer,
neurological disease, and rare disease. 



Friedrich Metzger, Chair Skyhawk Scientific Advisory Board

Dr. Metzger is a leading scientist in RNA-based disease and neurological conditions.  As Head of Discovery Rare Diseases at Roche for 15 years, he ran drug discovery programs for Alzheimer’s, ALS, Parkinson’s, Duchenne Muscular Dystrophy & Spinal Muscular Atrophy (SMA).  Dr. Metzger scientifically supervised Roche’s SMA splicing modifier program, currently in pivotal clinical trials, and coauthored 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 and as CSO of Versameb, a regenerative medicine company. He received a PhD in Pharmacology at University of Freiburg and Tübingen, Germany.

Jacqueline A. Lees, Associate Director, Koch Institute for Integrative Cancer Research

Dr. Lees is a leading expert in cancer cell cycle regulation and alternative splicing regulation in cancer.  She is the Assistant Director at the Koch Institute for Integrative Cancer Research at MIT and a Professor of Biology there, where her research focuses on metastasis and personalized medicine, and the genetic causes of cancer. Lees has held a number of academic posts prior to her role at MIT. She has authored more than 50 peer-reviewed papers, and received her PhD at the University of London.

Adrian Krainer, Cold Spring Harbor Laboratory; American Academy of Arts & Sciences

Dr. Krainer is a world 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. Professor 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 recently 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.

Steven Bell, PhD, Professor of Biology at MIT; Investigator, Howard Hughes Medical Institute

Dr. 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.  Dr. Bell is a Professor of Biology at MIT, an investigator at the Howard Hughes Medical Institute, and a member of the National Academy of Sciences.  He has authored over 100 peer-reviewed publications, and received his PhD at the University of California, Berkeley.

Dr. Fredrich Allain, Professor, Inst. of Molecular Biology and Biophysics at ETH, Zurich, Switzerland

Dr. Allain is a leading expert in the role of RNA in disease. His work using NMR technology to elucidate the structure of protein-RNA complexes is world leading.  He and his research team are expert in the use of biophysical tools to study protein RNA interactions, with a focus on the notable segment of genetic diseases that originate from a post-transcriptional misregulation of gene expression often caused by splicing, RNA editing or translation defects. Dr. 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 PhD from Cambridge University, U.K.


Bill Haney, Skyhawk Co-Founder & Executive Chairman of the Board; Dragonfly 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, and the President's Circle for the National Academy of Sciences.  He has won a Humanitarian Award from Harvard Medical School, an Achievement Award from the ACLU, a Genesis Award and the Pare Lorenz Prize.  He’s the co-founder of World Connect, a global non-profit, and has served on boards for Harvard, MIT, the World Wildlife Fund, the World Resources Institute, and the NRDC.  He is an honors graduate from Harvard College and was a Kennedy School Fellow from 1997-2001. 

Kevin Koch, Skyhawk Board Member; Orbimed Advisors Venture Partner

Dr. Koch is a Venture Partner with OrbiMed Advisors, LLC, and the President and CEO of Edgewise Therapeutics.  He was formerly at Biogen as Senior Vice President of Drug Discovery: Chemical and Molecular Therapeutics where he managed global drug discovery and the immunology and hematology teams.  Previously, Dr. Koch served as the President, CSO, Board member and Co-Founder of Array BioPharma, where he oversaw the invention of over 20 clinical development candidates in multiple therapeutic areas. Prior to Array, Dr. Koch held senior positions at Amgen Inc. and Pfizer Central Research.  He is the co-author or inventor of multiple patents and publications, and received his PhD in Organic Chemistry from University of Rochester.

The Duke of Bedford, Skyhawk Board Member; Technology & Real Estate Investor

The Duke of Bedford is an investor and board member in numerous technology companies, and serves as a founding board member of Skyhawk.  He has 30+ years’ experience as an international real estate investor, managing a broad portfolio of London properties as well as running the Woburn Abbey, Safari Park and Golf Club, and has been a leading technology investor since 1980.  The Duke of Bedford is a founder or board member of numerous companies and nonprofit organizations, and as the 15th Duke of Bedford, England, serves as a British nobleman and peer.  He is a graduate of Harvard College.

Andrew Boyd, Skyhawk Board Member; Fidelity Head of Global Equity Capital Markets

Andrew is head of Global Equity Capital Markets at Fidelity Management & Research Company, where he leads the private equity investments across the Fidelity funds.  In this role he has been a forefront of technology development, as a leading investor in firms such as Uber, Snapchat and SpaceX.  Before joining Fidelity, Andrew was an attorney at Goodwin Proctor LLP and a partner at Davies Ward LLP. Mr. Boyd earned his bachelor of laws from the University of Windsor, and his juris doctorate degree from the University of Detroit.

Kathleen McCarthy, Skyhawk Co-Founder & Chief Executive Officer 

Kathleen is a leading expert in developing small molecules to target RNA-splicing.  She 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 targeting mRNA-protein interactions 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.

Sergey Paushkin, Skyhawk VP & Director of Biology

Dr. Paushkin has over 20 years of experience in drug discovery and development, with a keen focus on small molecule regulation of RNA processes, and molecular mechanisms of rare diseases.  He is a co-author of numerous publications in Cell, Nature and Science magazine and co-edited a textbook “Spinal Muscular Atrophy: Disease Mechanisms and Therapy” (Academic Press, Elsevier, 2017).  Most recently Dr. Paushkin was Director at the Spinal Muscular Atrophy (SMA) Foundation coordinating research efforts of pharma, biotech, academic, clinical, and CRO groups to bring first-in-class therapeutics to clinical trials for this genetic disease. The program was acquired by Roche and currently is in Phase 3 clinical trials. Dr. Paushkin received his post-doctoral training at the HHMI, University of Pennsylvania, and holds an MD degree from the Russian State Medical University and a PhD degree in Biochemistry from the Cardiology Research Center, Moscow.

Maura McCarthy, Skyhawk Director of Finance & Development

Maura manages finance and business development at Skyhawk.  She has 15 years' experience in finance, business development and marketing, beginning her career in statistical analysis at the Federal Reserve Bank.  As a venture capitalist, Maura led diligence on investments in technology, healthcare and consumer goods, and as a senior executive at Blu Homes and Dragonfly has played a central role in raising more than $250m in equity and partner agreements.  She is a co-founder of the global non-profit, World Connect, and of Blu Homes, the leading builder of premium prefabricated homes in America.  Maura earned her BA in Economics and Philosophy from Georgetown University.

Dr. Michael Luzzio, Skyhawk Director of Chemistry

Dr. Luzzio has spent 33 years working at Syntex, Glaxo-GlaxoWellcome, Pfizer, Novartis, and Biogen, where he held positions of significant scientific and management responsibility.  As a medicinal chemist, Dr. Luzzio led and participated in teams across all phases of discovery research which contributed to the successful advancement of 13 drug candidates including two commercial drugs Tykerb® and Kisqali®.  Two other candidates are currently in advanced clinical development.  Dr. Luzzio has co-authored over 100 publications and presentations in chemistry and drug discovery and is a co-inventor on 30 issued patents. He earned his PhD in Organic Chemistry from the University of Rochester, and was an NIH Postdoctoral Fellow at the University of California, Berkeley.


35 Gatehouse Drive | Waltham, MA 02451  |  | Tel: 617.858.0041

Skyhawk Therapeutics, Inc.

35 Gatehouse Drive


Waltham, MA 02451

United States of America


Tel: 617.858.0041

Skyhawk Therapeutics Europe GmbH


Basel Technology Park

Hochbergerstrasse 60C

Basel 4057


Tel: +41 (0) 61 638 23 50

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