Expansion Therapeutics, Inc., a Jupiter, Florida, and San Diego, CA-based new 5AM Ventures formed private company focused on the discovery and development of ribonucleic acid (RNA) targeted small molecule medicines, closed a $55.3m Series A financing.
The round was co-led by 5AM Ventures, Kleiner Perkins Caufield Byers, Novartis Venture Fund, and Sanofi Ventures with participation from RA Capital Management and Alexandria Venture Investments.
The company intends to use the funds to advance its portfolio of small molecule drugs targeting key human disease-driving RNAs with an initial focus on expansion repeat disorders, a set of approximately 30 genetic diseases that currently have no satisfactory treatments.
Led by newly named co-founder Kevin M. Forrest, Ph.D., president and chief executive officer, Expansion Therapeutics is a drug discovery and development company pursuing the potential of small molecule medicines for RNA-mediated diseases. Based on exclusive worldwide rights to research from the laboratory of Matthew D. Disney, Ph.D., at The Scripps Research Institute, Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule binders of RNA. Through this platform, the company is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of disease indications. The initial development focus is on therapies for patients with expansion repeat diseases who currently have limited and unsatisfactory treatment options.
Following the close of the financing, the board of directors will include:
– Scott M. Rocklage, Ph.D., of 5AM Ventures and Chairman of the Expansion Board of Directors
– Matthew D. Disney, Ph.D., of The Scripps Research Institute
– Kevin M. Forrest, Ph.D., of Expansion Therapeutics
– Jason P. Hafler, Ph.D., of Sanofi Ventures
– Yujiro S. Hata, M.B.A., of Ideaya Biosciences
– Campbell Murray, M.D., M.B.A., M.P.P., of Novartis Venture Fund
– Beth Seidenberg, M.D., of Kleiner Perkins
– Andrew Levin, M.D., Ph.D., of RA Capital Management joins as a board observer
Expansion has also formed a scientific advisory board comprised of leaders in the field of RNA targeted small molecule chemistry and biology, RNA folding, and structural biology, including:
– Robert T. Batey, Ph.D., of the University Colorado, Boulder
– Dale L. Boger, Ph.D., of The Scripps Research Institute
– Ronald R. Breaker, Ph.D., of the Howard Hughes Medical Institute and Yale University
– M.G. Finn, Ph.D., of the Georgia Institute of Technology
– David H. Mathews, M.D., Ph.D., of the University of Rochester
– Michael Zuker, Ph.D., of the Rensselaer Polytechnic Institute