Expansion Therapeutics is a drug discovery and development firm which is trying to come up with cures for a number of genetic disorders. One of these is called myotonic dystrophy type I, which leads to adults developing muscular dystrophy. The had a Series A financing round which did very well, resulting in this company raising $55.3 million. There were four main firms which invested in Expansion Therapeutics in this round of financing. These were 5AM Ventures, Novartis Venture Fund, Sanofi Ventures, and Kleiner Perkins. Two other companies also participated which were RA Capital Management and Alexandria Venture Investments.
It is believed by those in the medical community that muscular dystrophy occurs when RNA hit toxic levels. Once this happens muscles, heart and various systems become defective. It is a hereditary disease and each generation has worse and worse symptoms. So far no one has come up with a treatment for muscular dystrophy which Expansion Therapeutics is trying to change.
The research that Expansion Therapeutics is using to develop an effective treatment for muscular dystrophy comes from Matthew D. Disney, Ph.D.’s lab. He is also the main scientific researcher for this company. He says he has been working on developing a treatment for this disease for the past 12 years and he believes they will come up with an effective treatment regimen. The first patients who will take this company’s medicines are those who have run out of any other option.
5Am Ventures, one of the main investors in Expansion Therapeutics, is led by Scott Rocklage, Ph.D. He first became involved with this company in 2003 as a venture partner and he was elevated to the role of managing partner a year later. He has a wealth of experience in the biotech industry. He started out as a researcher and over time transitioned to executive roles for a couple of different companies. He says he focuses 5AM Ventures on biotech startups like Expansion Therapeutics. His investment firm also uses their experience to help the companies they invest in by guiding them and their research, oftentimes on these companies board. Learn more: https://www.dialdish.com/scott-rocklage-seeks-to-conquer-cancer-by-targeting-mutant-rna-molecules/