Scott Rocklage Impact In The World Of Science

The disease of muscular dystrophy is beginning to lose the battle against modern medical research thanks in a big part to Scott Rocklage, PhD, who is the managing partner of 5AM Ventures. A leading venture capital company for the healthcare industry’s research, technology and drug development efforts.


Scott Rocklage became a venture partner with 5AM Ventures in 2003, in 2004 he was appointed the managing partner and has maintained that position with immense success ever since. His latest endeavors include teaming up with other venture capitalist in the healthcare industry to tackle Myotonic dystrophy I (DM1) which is the primary cause of adult muscular dystrophy. All of the venture capital companies combined have recently added $55.3 million dollars to the fight.


The lab soldiers in the fight are being lead by Expansion Therapeutics, a drug research and development company who’s recent findings have uncovered that symptoms of the chronic disease appear when toxic levels of RNA are reached. RNA molecular transfer from a patient’s DNA is converted to proteins and reaches a toxic level then Myotonic dystrophy I sets in. Causing major nervous system and other systems in the body to break down and become defective.


Lead by research from the lab of Matthew D Disney, PhD. Expansion Therapeutics is developing small molecule medications to fight the disease related RNA to arrest the growth of toxic levels. Learn more:


Currently RNA triggered disease such as myotonic dystrophy at the time have no way of being treated but this is a huge step toward overcoming that challenge and the entire Expansion Therapeutics team is determined to give both hope and treatment to those patients who have neither right now. In addition to treating toxic RNA levels with small molecule medications the research team is at the same time working to develop medicines and treatments for patients that have direct medical needs as well.


The optimism and hope the researchers are expressing can be reinforced by the fact that they have Scott Rocklage in their corner for this fight. He has been responsible for getting approval for the innovative medications Teslascan, Omniscan and Cubicin from the FDA as well as being the inventor or co-inventor of 30 United States Patents. He began his education at Cal Berkeley with a major in Chemistry and then went on to receive his PhD. in the same major at M.I.T.


Rocklage can be found operating the 5AM Ventures main office in Boston these days.