Scott Rocklage’s Role in Treating DM 1

Expansion Therapeutics is a company that is highly dedicated to the development of drugs that are used to treat RNA-mediated diseases. The group has been sponsored by a few companies in its quest to develop a drug for myotonic dystrophy type 1. These sponsor companies include Sanofi Ventures, Novartis Venture Fund, Kleiner Perkins, 5 AM Ventures, Alexandria Venture Investment, and RA Capital Management. The Managing Partner of 5 AM Ventures, Dr. Scott Rocklage is also the Chairman of the Board of Directors of Expansion Therapeutics. Learn more:


Myotonic dystrophy type 1 is also called DM 1. The disease is the leading cause of muscular dystrophy in adults. The signs and symptoms of the disease project themselves once RNA reaches toxic levels. RNAs are molecules that develop from DNA to form proteins. Production of some types of RNAs may reach toxic levels and lead to the formation of myotonic dystrophy type 1. When the disease is at its full-blown stage, body systems such as the gastrointestinal system, the respiratory system, and the central nervous system start to fail or slow down. Major organs such as the heart and muscles may begin to become defective and thus cause paralysis of other metabolic operations of the body.


Expansion Therapeutics mainly deals with the development of drugs for diseases that are called expansion repeat disorders. These are complications that are hereditary and have a slow response to the current medications on the market. The company conducts its developments in the laboratories of Dr. Matthew D. Disney. Dr. Scott Rocklage who is the founding investor of Expansion Therapeutics said in a press conference that the leading researcher in the drug development exercise is Dr. Disney.


Dr. Scott Rocklage has been the Managing Partner of 5 AM Ventures for many years. Dr. Rocklage is a graduate of some of the most reputable learning institutions in the country. He is a holder of Bachelor of Science degree in Chemistry from the University of California. He also has a Ph.D. in Chemistry from Massachusetts Institute of Technology (MIT). During his time at MIT, Mr. Scott Rocklage did his research in Dr. Richard Shrock’s laboratory. Dr. Shrock was the winner of the prestigious Nobel Prize in Chemistry in 2005.


He joined 5 AM Ventures in 2003 as a Venture Partner. In 2004, Dr. Rocklage was made the Managing Partner of the institution. He is highly experienced in the healthcare management. He was once the CEO of Cubist Pharmaceuticals. Dr. Rocklage was also the CEO and President of Nycomed Salutar at some point.