Scott Rocklage Is Fighting For A Cure

Managing partner of 5AM Venture, Scott Rocklage, announced that the company has joined in a $55.3 million in a Series A round of funding for Expansion Therapeutics. Novartis Venture Fund, Kleiner Perkins, Sanofi Ventures, along with 5AM Venture, were all participants in this round of funding.


Expansion Therapeutics will be using this money to fight the diseases it has in its portfolio. Specifically, they are looking for treatments for myotonic dystrophy type 1. Also known as DM1, myotonic dystrophy type 1 is the leading cause of muscular dystrophy in all adults.


What Is Myotonic Dystrophy

The condition of myotonic dystrophy type 1 occurs when the production of RNAs reach toxic levels in the cells. This is when muscles, the heart, the hormonal system, the gastrointestinal system, the respiratory system, and the central nervous system start to become defective.


In the United States muscular dystrophy affects 1 in every 5,600 to 7,700 males age 5-24. Worldwide that number is 1 in every 8,000.


Currently there is no effective cure for DM1 which is why Rocklage and Expansion Therapeutics’ work is so important.


Expansion Therapeutics’ Approach

Expansion Therapeutics are working on medications that fight the RNA in this type of illness. They focus on illnesses that are expansion repeat disorders. These are illnesses that are hereditary and do not respond to conventional treatments. In the case of DM1, this illness can affect entire families, The disease can be passed down from generation to generation.


More About Scott Rocklage

Scott Rocklage earned a Bachelor of Science degree in chemistry from the University of Berkeley. He then went on to the Massachusetts Institute of Technology where he received his PhD.


With over 30 years of healthcare management experience, Dr. Rocklage bring a wealth of knowledge to 5AM Ventures. He was named managing partner of the firm in 2003. Learn more:

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