Scott Rocklage and Expansions Therapeutics

Scott Rocklage received his Bachelor’s degree in chemistry from the University of California Berkeley.

He later went on to earn his Ph.D. in Chemistry from the Massachusetts Institute of Technology. Rocklage is currently a managing partner of 5AM Ventures. 5AM, under Rocklage’s direction, helps finance medical research.

Expansions Therapeutics, which is funded by 5AM Ventures, as well as Kleiner Perkins, Sanofi Ventures, and Novartis Venture Fund, works to find cures for genetic disorders. One disorder in particular known as myotonic dystrophy type I, or DM1, is one of the main causes of muscular dystrophy in adults.

5AM Ventures, Kleiner Perkins, Sanofi Ventures, Novartis Venture Fund, along with Alexandria Venture Investments and RA Capital Management provided 55.3 million for Expansions Therapeutics to fight myotonic dystrophy type I. Read more: Scott Rocklage | Crunchbase and Scott Rocklage | Bloomberg

Because it is a genetic disorder, whole families can be harmed by myotonic dystrophy disorder. There is not currently an effective treatment for the disease, but Expansions Therapeutics hopes to change that.

Expansions Therapeutics has discovered that RNA plays a role in the spread of DM1. RNA molecules build DNA by converting proteins. DM1 happens when these RNAs reach levels of toxicity inside cells. Learn more about Scott Rocklage: and

When this happens, important systems in the body, like the heart, the hormonal system, the gastrointestinal system, respiratory system, and the nervous system begin to break down.

Expansion Therapeutics is working on medicine that interacts with RNA on a molecular level. They hope that a cure for myotonic dystrophy type I will pave the way for cures for a number of deadly hereditary diseases.

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