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Scott Rocklage and Expansion Therapeutics Come Together to Fight DM1

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Expansion Therapeutics has a portfolio with genetic disorders in it that it has been raising financing to fight. Myotonic dystropy type 1 (DM1) is included and is the leading cause of adults developing muscular dystrophy, there has been over $55 million added to cover fighting DM1. Several corporations including 5AM Ventures, RA Capital Management, and Sanofi Ventures took part in the financing for DM1.


DNA transfers molecules of RNAa which get converted into proteins, DM1 reaching toxic levels in the cells is what cause DM1 to occur. This causes the muscles, hormonal system, respiratory system, heart and central nervous system be start becoming defective. DM1 can affect whole families and it gets worse with each generation.


There is not currently an effective way to treat DM1, Expansion Therapeutics is creating medication to confront the RNA driven illnesses. Hereditary illnesses that do not respond to current medical interventions adequately known as expansion repeat disorders are the focus of medications and research from Expansion Therapeutics. Matthew D. Disney Ph.D’s research was used as the basis to the approach.


Scott M. Rocklage, Ph.D, 5AM Ventures managing partner has issued a press release which stated the team’s main researcher is Mr. Disney. The last twelve years of research and work the team has completed are gratifying for him, and small molecule medications can be addressed with RNA related diseases. The team is close to creating treatments that will help those with direst medical needs.


Expansion Therapeutics and Dr. Rocklage have plans to advance the targeting of RNA with small molecule medicines. They are eager to continue creating medications for those who do not have options. Learn more:

May 7, 2018