Scott Rocklage is Fighting for the Cure

Expansion Therapeutics is a pioneering biotech firm on the forefront of fighting myotonic dystrophy type 1 (DM1), the main cause of muscular dystrophy that begins in adulthood. Expansion Therapeutics specializes in developing drugs that target RNA-driven diseases. RNA is a natural molecule transcribed by DNA. However, sometimes it accumulates in cells until it reaches toxic levels. DM1 is one of those diseases.

It’s caused by a defective gene that causes the DNA to repeat, leading to the excess production of RNA. When they accumulate, they become toxic. That leads to problems in the heart, respiratory system, hormones, muscles, central nervous system and gastrointestinal tract. Currently there are no drugs that effectively cure or treat this devastating disease. Learn more about Scott Rocklage: and

However, Expansion Therapeutics has the benefit of the laboratory work and research performed by Matthew D. Disney, Ph.D, of The Scripps Research Institute. Because it’s caused by genetic defects, DM1 can affect entire families, and it gets worse with each generation. According to the Chairman of the Board of Expansion Therapeutics as well as its founding investor, Disney is the main scientific researcher. Read more: Scott Rocklage | Bloomberg and Scott Rocklage | Crunchbase

Disney says repeat RNA diseases such as DM1 can now be targeted with small molecule drugs. He has been working on developing a solution for RNA-driven disorders for over a dozen years. He swears he and his dedicated team will not stop until they develop a cure. They have a plan, and now, thanks to the funding Expansion Therapeutic just received, they can carry through on their plan.

Scott Rocklage of 5AM Ventures is making sure Disney and Expansion Therapeutics has the funding it needs to carry on this important and vital work. With his leadership, 5AM Ventures, Novartis Venture Fund, Kleiner Perkins and Sanofi Ventures took on the initiative to raise $55.3 million of Series A financing for Expansion Therapeutics to develop a cure for DM1. RA Capital Management and Alexandria Venture Investments also helped raise this funding.

The managing partner of 5AM Ventures, Scott Rocklage has great experience in the medical and healthcare field. He started his career with a Ph.D in Chemistry from the Massachusetts Institute of Technology. He joined 5AM Ventures as a Venture Partner in 2003.

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