On January 23, 2018 Skyhawk Therapeutics, Inc. ("Skyhawk"), a new private company focused on the discovery and development of small molecule therapeutics that correct RNA expression, reported the close of $8 million in seed funding led by major family and biotech investors, including Tim Disney, the Duke of Bedford, Alexandria Venture Investments, and other undisclosed private investors (Press release, Skyhawk Therapeutics, JAN 23, 2018, https://www.prnewswire.com/news-releases/skyhawk-therapeutics-launches-with-8-million-in-seed-financing-to-advance-its-novel-platform-and-portfolio-of-small-molecules-that-correct-rna-expression-300586378.html [SID1234551690]). Proceeds of the financing will advance Skyhawk’s portfolio of specific and selective small molecules that initially target RNA exon skipping, which drives a set of 50+ diseases that include both broad-based neurological conditions and previously "undruggable" oncogenes in cancer.

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"We are pleased be working with leading investors to advance our mission of revolutionizing disease treatment with small molecules that correct RNA expression," said founding investor and executive chairman of Skyhawk’s board of directors, Bill Haney. "The Skyhawk team has spent the last 12 months building an expansive intellectual property base covering its unique drug discovery tools and small molecule library portfolio. The company’s first drug candidate targets a set of exon-skipping-driven cancer indications, and we are focusing our energy on driving it into the clinic in 2019."

Initially, Skyhawk is targeting diseases driven by a type of RNA mis-splicing called "exon skipping," where key regions on the RNA are left out during the RNA splicing process. Skyhawk’s proprietary technology enables the rational design of small molecules that target specific binding pocket regions on RNA, using both sequence and structural specificity, at particular moments in the RNA splicing process. By doing so, they reverse the mis-splicing and treat the disease.

"The ability to use small molecule therapeutics to selectively target RNA and correct RNA expression is potentially transformative," said Catherine Nuccio of Alexandria Venture Investments. "The technology has now moved beyond proof of concept and is applicable in a range of diseases for the benefit of a broad base of patients. We are excited to seed and support the team that helped to develop the initial technology and its mechanisms."

There are 50+ known diseases driven by exon skipping, from broad based neurological conditions to major cancers, and hundreds more driven by similar forms of RNA mis-expression.

Skyhawk Founders and Board of Directors
Skyhawk founders and founding board of directors include:

William Haney, Skyhawk co-founder and executive chairman of the Skyhawk board of directors, and co-founder and CEO of Dragonfly Therapeutics. Bill is currently CEO of Dragonfly Therapeutics, a Boston-based biotech that develops novel first-in-class therapeutics targeted at natural killer (NK) cells and other cells of the innate immune system. He is an investor and entrepreneur, having started or helped start more than a dozen technology companies.
Kathleen McCarthy, Skyhawk co-founder and CEO. Kathleen is a leading expert in developing small molecules to target RNA splicing. As a pre-clinical scientist at the Spinal Muscular Atrophy (SMA) Foundation in New York and at Roche in Basel, Switzerland, Kathleen helped bring the first-ever small molecule therapeutic targeting mRNA-protein interactions to clinical trials in SMA. She is a co-inventor and co-author on numerous patents and publications describing small molecules that specifically and selectively target RNA expression.
Kevin Koch, Skyhawk founding board member, venture partner with OrbiMed Advisors, LLC and President and CEO of Edgewise Therapeutics. Dr. Koch was formerly SVP of Drug Discovery: Chemical and Molecular Therapeutics at Biogen, where he managed global drug discovery and immunology. He was the co-founder of Array BioPharma, where he oversaw the invention of over 20 clinical development candidates, and has held senior positions at Amgen Inc. and Pfizer Central Research.
Andrew Boyd, Skyhawk founding board member and 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 at the forefront of technology development, and has been a leading investor in firms such as Uber, Snapchat and SpaceX.
Andrew Bedford, Skyhawk founding board member, technology investor and Duke of Bedford, England. Andrew currently manages a broad portfolio of London properties, and, as the 15th Duke of Bedford, England, serves as a British nobleman and peer. He is an investor and board member in numerous companies with 30+ years’ experience in international real estate and technology.
Skyhawk Founding Scientific Advisory Board
Skyhawk’s founding scientific advisory board (SAB) is comprised of scientists with expertise in RNA-based disease and neurological conditions, mechanisms of RNA splicing, alternative splicing regulation in cancer, DNA replication and RNA structural biology. Members include:

Friedrich Metzger, founding chairman of the Skyhawk scientific advisory board and former Head of Discovery Rare Diseases at Roche. Dr. Metzger is a leading scientist in RNA-based disease and neurological conditions. At Roche, Dr. Metzger spent 15 years running drug discovery programs for Alzheimer’s, ALS, Parkinson’s, Duchenne Muscular Dystrophy and Spinal Muscular Atrophy (SMA). He is the senior author on the Science and Nature papers describing the first small molecules that could specifically and selectively target RNA expression.
Jacqueline A. Lees, founding member of the Skyhawk scientific advisory board, Associate Director of the Koch Institute for Integrative Cancer Research, and Professor of Biology at MIT. Dr. Lees is a leading scientist in cancer cell cycle regulation and alternative splicing regulation in cancer. Her research focuses on metastasis and personalized medicine, and the genetic causes of cancer.
Adrian Krainer, founding member of the Skyhawk scientific advisory board, member of the American Academy of Arts & Sciences, and Professor at Cold Spring Harbor Laboratory. Dr. Krainer is a world expert on the role of mRNA splicing proteins in cancer, and neurological and rare diseases. His lab is leading the understanding of mechanisms of RNA splicing, and the means by which faulty splicing can be corrected.
Steven Bell, founding member of the Skyhawk scientific advisory board, Investigator at Howard Hughes Medical Institute, member of the National Academy of Sciences, and Professor of Biology at MIT. Dr. Bell is a leading scientist in the study of DNA replication, with an emphasis on how assembly of enzymes is regulated during the cell cycle to ensure genomic maintenance.
Frédéric Allain, founding member of the Skyhawk scientific advisory board and Professor/Head of the Institute of Molecular Biology and Biophysics at ETH, Zurich, Switzerland. Dr. Allain is a leading scientist in the role of RNA in disease and the use of NMR technology to elucidate the structure of protein RNA complexes. He and his team are expert in the use of biophysical tools to study protein RNA interactions and mRNA splicing regulation, with a focus on the segment of genetic diseases that originate from post-transcriptional misregulation of gene expression often caused by splicing, RNA editing or translation defects.
"Skyhawk’s founding team has unique depth of experience in this growing area of small molecule therapeutic development." said Tim Disney, founding investor in Skyhawk. "To support their mission we put together a long-term, science-focused group of investors, who share their humility in the face of this great opportunity, and a strong interest on getting drugs to patients, quickly."