RNA-driven disorders are not widely known diseases, but they are devastating to the families who have the damaged gene that causes them. One of the worst RNA-driven disorders is myotonic dystrophy type 1 or DM1.
DM1 is the main cause of adult-onset muscular dystrophy. Matthew D. Disney, Ph.D. is a medical researcher who has dedicated his career to finding a cure to RNA-driven diseases. Read more: Scott Rocklage | Crunchbase and Scott Rocklage | Bloomberg
He is the main medical researcher at the firm Expansion Therapeutics, which is dedicated to finding drugs that target DM1 and other RNA-driven diseases. Disney and Expansion Therapeutics are dedicating themselves to the cause, but they need money to finance the research of Disney and his team.
RNA is transcribed by DNA. That’s normal. However, some people and families have a genetic defect that causes the DNA to repeat the transcription of the RNA, so it just keeps accumulating in the cells of the victims.
When it reaches a certain level, it’s toxic to the cells, and causes problems in muscles, the heart, the gastrointestinal tract and other body systems. There are currently no effective cures for this type of disease. These are also known as expansion repeat disorders.
Fortunately for the families suffering from DM1, Dr. Scott Rocklage is also devoted to finding a cure for DM1. Dr. Rocklage joined 5AM Ventures in 2003 as a Venture Partner. In 2004 he became Managing Partner.
He and 5AM Ventures led an effort with other venture capital firms to raise Series A financing for Expansion Therapeutics. Along with Novartis Venture Fund, Sanofi Ventures, Kleiner Perkins, Alexandria Venture Investments and RA Capital Management, Dr. Rocklage raised $55.3 million to fund Expansion Therapeutics and Disney’s research into developing a small molecule drug to cure DM1.
Dr. Rocklage has been in the fields of healthcare and medicine for over 30 years. He obtained approval from the Food and Drug Administration for three drugs: Omniscan, Cubicin and Teslascan. He has designed clinical drugs for many other drugs. As an inventor, he has over 30 patents. He has more than 100 peer-reviewed scientific publications.
Dr. Rocklage also has a lot of experience running medical research firms. He served as Board Chairman for Novira, Relypsa and Achaogen.