Expansion Therapeutics is a biotech startup based in San Diego, California. It recently held its Series A financing round in which it raised $55.3 million from six venture investment firms. The goal of this company is to provide treatments for genetic disorders. The first disorder it is developing a drug for is myotonic dystrophy type 1, or DM1. DM1 has been shown to be the main cause of adults developing muscular dystrophy.
The four main venture investment firms to have invested in Expansion Therapeutics during this round were Kleiner Perkins, Novartis Venture Fund, 5AM Ventures, and Sanofi Ventures. Two other companies participated to a lesser extent which were Alexandria Venture Investments and RA Capital Management.
RNAs (ribonucleic acid) are molecules which exist in all living cells. Their purpose is to carry instructions from DNA which leads to the synthesis of proteins. In certain people the amount of RNAs can reach a toxic level and this results in DM1 occurring. Once this happens ths person’s heart, muscles, and bodily systems start to become defective and they develop muscular dystrophy.
DM1 can be passed down generation to generation with each subsequent generation having worse symptoms. There has yet to be any good treatment for those who have DM1. Expansion Therapeutics is looking to address this and treat illnesses where too much RNA is created. Their work is a continuation of the research that was developed in the lab of Matthew Disney, Ph.D.
At 5AM Ventures, Scott Rocklage, Ph.D. is the managing partner. He has been a venture investor for the past 15 years. Prior to that he was an executive in the pharmaceutical industry for several years. He started out in the healthcare industry as a researcher and developer. He earned his Ph.D. at the Massachusetts Institute of Technology and continues to live in the greater Boston, MA region.
In addition to investing in healthcare firms, Scott Rocklage is also the chairman of the board for three different firms which are Novira, Relypsa, and Achaogen. He has been on other companies boards in the past like Variation Biotechnologies, Inc. and Epirus Biopharmaceuticals. Learn more: https://www.twst.com/bio/scott-m-rocklage/