Scott Rocklage Efforts in Finding Cure for the DM1 condition

Scott Rocklage of 5AM Ventures and several other partners such as Sanofi Ventures, Kleiner Perkins, RA Capital Management, Novartis Venture Fund, and Alexandria Venture Investments, have been lead financiers to Expansion Therapeutics, in the fight of genetic disorders. One of such a disease is Myotonic dystrophy type I (DM1), which is the main cause of adults’ muscular dystrophy. The problem is the disease’s symptoms do not present themselves until the RNA has reached toxic levels, and it’s at this point the DM1 condition occurs.

The manifestation of the DM1 condition, the heart, hormonal system, muscles, gastrointestinal system, respiratory system, and the central nervous system starts to become defective. This condition is capable of affecting the whole family as it gets worse with every subsequent generation. The medical community is yet to get DM1’s effective treatment.

RNAs are molecules transferred from DNA and changed into proteins. Expansion Therapeutics do make medications which confront the RNA in RNA driven diseases. Their major concern, however, is the expansion repeat disorders, which are hereditary illnesses, that fail to adequately respond to the current medication. Such illnesses happen to have defective genes caused by replication of tiny portions of DNA, DM1 is an example of such illnesses.

In a press release issued by Scott Rocklage, he stated that Expansion Therapeutics have their researches are done in Matthew D. Disney, Ph.D.’s laboratories, which they use as the foundation of their research on this condition, led by Mr. Disney himself, as the chief scientific researcher.

Other than Mr. Rocklage working together with the Expansion Therapeutics in the research team, there are other goals they have achieved together. In fact, they are planning on how to advance the small molecule medicines’ field that targets RNA, with the aim of creating medications for those patients with no options.

Rocklage holds a bachelors degree in Chemistry from California University. He is also a holder of a Ph.D. in Chemistry from Massachusetts Institute of Technology. Rocklage started working at 5AM Ventures in 2003, and in 2004 he was made the Managing Partner, a position he holds to date.

He has been in healthcare management for over 30 years. He is an inventor of several medications. Rocklage has been appointed for leadership roles in several boards and organizations such as Cubist Pharmaceuticals where he is the CEO, Relypsa, Achaegon and Novira’s board chair, and many other roles.

 

Connect with Dr. Scott Rocklage on LinkedIn.

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