Expansion Therapeutics has been busy raising Series A financing for the purpose of fighting the genetic disorders in its portfolio. Included in these disorders is myotonic dystrophy type I. Myotonic dystrophy type I or DM1 is known as the leading cause of muscular dystrophy in adults, and the company added $55.3 million to its coffers for the purpose of fighting this disease. Symptoms are believed to present themselves when RNA reaches toxic levels.
Sanofi Ventures, Novartis Venture Fund, Kleiner Perkins and 5AM Ventures all took part in leading the financing. Alexandria Venture Investments and RA Capital Management also participated in this undertaking.
RNAs are molecules that are transferred from DNA and converted into proteins. When the production of some RNAs reaches toxic levels inside the cells, the condition of DM1 occurs. This is when the muscles, the heart, the gastrointestinal system, the hormonal system, the central nervous system and the respiratory system begin to become defective.
Entire families can be affected by DM1, and it only grows worse with each subsequent generation. Currently, the medical community does not have an effective treatment for DM1.
Expansion Therapeutics creates medications that confront the RNA in illnesses that are driven by RNA. At the forefront of the company’s concerns are illnesses that are expansion repeat disorders. Expansion repeat disorders are illnesses that are hereditary and do not respond adequately to current medical interventions. These diseases tend to have defective genes that are caused by repeats of tiny portions of the DNA. DM1 is a disease that falls under this description when the DNA repeats encourage the production of RNAs that begin to accumulate and reach the level of toxicity.
Expansion Therapeutics uses research performed at the laboratory of Matthew D. Disney, Ph.D. as a basis for its approach. Managing Partner of 5AM Ventures Scott M. Rocklage, Ph.D. issued a press release on this matter. In the press release, the Chairman of the Board of Directors of Expansion Therapeutics and founding investor stated that Mr. Disney is the main scientific researcher on the team.
Dr. Disney stated that he is gratified that the work he and his team have completed over the past 12 years have led them to the position they are in today. It is apparent that disease-related RNA can now be addressed with small molecule medications. He stated that they are right around the corner from producing treatments that are needed by those with the direst medical needs. He pledges that he and his team will never stop until they have fulfilled this need.
The research team is not the only thing that Dr. Rocklage and Expansion Therapeutics have brought together to help with their goal. They also have a plan that will advance the field of small molecule medicines that target RNA. They are eager to continue their work with the company to create medications for patients who don’t have any options.
Expansion’s RNA-targeted medications depend on science that has the potential to treat RNA-triggered illnesses, and one of those illnesses is myotonic dystrophy that cannot be realistically treated at this moment. General Partner of Kleiner Perkins Beth Seidenberg, M.D. stated that she and her company are delighted to form a partnership with a company that is going to make it possible to treat diseases that currently have no cure.
About Scott Rocklage
Scott Rocklage is the Managing Partner of 5AM Ventures. He attended the University of California, Berkeley and earned his Bachelor of Science degree in Chemistry. He received his Ph.D. in Chemistry from the Massachusetts Institute of Technology. While at MIT, he conducted research in Richard R. Schrock’s laboratory. Dr. Schrock received the Nobel Peace Prize in Chemistry in 2005.
Dr. Rocklage joined 5AM Ventures in 2003 when he became a Venture Partner. He went on to become Managing Partner in 2004.
Dr. Rocklage has more than 30 years of healthcare management experience. He is responsible for the FDA approval that the three medications Cubicin, Teslascan and Omniscan received. He has also submitted numerous other medications to clinical trials. He has been an inventor and has more than 30 U.S. patents that he has invented or co-invented. More than 100 of his publications have been peer-reviewed.
Dr. Rocklage has had several leadership roles as well. He was the Chairman and CEO of Cubist Pharmaceuticals at one time. He was also the President and the CEO of Nycomed Salutar and held various other R&D positions with this company. He also performed several R&D positions for Catalytica.
Dr. Rocklage was the Board Chairman for Relypsa, Achaogen and Novira. He is currently the Board Chairman for Cidara, Kinestral and Rennovia. He has served on other boards as well, including those of Epirus Biopharmaceuticals, Pulmatrix, Variation Biotechnologies, Inc. and WaveRX, Inc. He was the Executive Chairman of Ilypsa, Miikana and Semprus as well. He also served as the Independent Director of Zalicus, Inc. Dr. Rocklage works at the Boston, Massachusetts office of 5AM Ventures.