Skyhawk Therapeutics Launches With $8 Million in Seed Financing to Advance Its Novel Platform and Portfolio of Small Molecules That Correct RNA Expression

WALTHAM, Mass., Jan. 23, 2018 /PRNewswire/ -- Skyhawk Therapeutics, Inc. ("Skyhawk"), a new private company focused on the discovery and development of small molecule therapeutics that correct RNA expression, today announced 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. 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.

"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."

About Skyhawk
Skyhawk Therapeutics is a drug discovery and development company focused on revolutionizing disease treatment with small molecules that correct RNA expression. Our founders are leading experts in small molecule therapeutics development for RNA-based disease. Our first drug candidates are targeted towards cancers and neurological conditions. The company is based in Waltham, Massachusetts. For more information, please visit: www.skyhawktx.com.

Media Contact:
Maura McCarthy
617.858.0041
maura@skyhawktx.com

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SOURCE Skyhawk Therapeutics, Inc.