Mytonis Dystrophy type 1 a genetic disorder that deteriorates muscles and organs until they no longer function. The disease affects about 1 in 8,000 people in the world and typically appears between the age of 20 and 30. The disorder is commonly referred to as Steinert disease. Learn more: http://medicaldailytimes.com/medical-research/scott-rocklage-leading-way-cure/3159/
Scott Rocklage and his organization, Expansion Therapeutics, have been attempting to find cures and therapies for patients with DS1. In 2018 the company announced that it had raised an additional 55 million dollars to help fund the way to helping patients.
The rare disease occurs when fragments of DNA repeat themselves in an individual. These cloned sequence of DNA are the cause of RNA increase, which in turn, due to its toxic properties in high levels begin the muscle deterioration. Rocklage and Company are working to create medicines that attack the RNA and put a stop on the damage that mytonis dystrophy has caused. There is no known cure at the moment, but everyone at Expansion Therapeutics are excited to adventure into the unknown and possibly provide relief for thousands of hurting patients.
About Scott Rocklage
Scott Rocklage was a student at the University of California, MIT, and Berkeley and is now a Managing Partner at 5AM Ventures. Rocklage joined the company in 2003 and in just two years he was awarded the Nobel Peace Prize in Chemistry.
Before joining 5AM Ventures Rocklage spent two decades with other companies producing new FDA approved medicines. He has worked with companies such as Cubist Pharmaceuticals, Nycomed Salutar, Relppsa, and Achaogen. Scott Rocklage currently resides in Boston, Massachusetts
In 2003 Dr. Scott Rocklage became a founding partner at 5AM Ventures. Today, he is a Managing Partner. 5AM Ventures provides start-up capital for businesses that develop research instruments and biopharmaceuticals, as well as financing research medical technology and life sciences research. Dr. Scott Rocklage was the CEO of Cubist Pharmaceuticals. He was both president and CEO of Nycomed Salutar.
Myotonic Dystrophy Type 1 (DM1) is a genetic condition that can lead to adults developing muscular dystrophy. DM1 affects breathing, the brain, spinal cord, hormones, endocrine system, the digestive system, and heart.
If a parent passes on the DM1 mutation the gene their child carries will be stronger than the parent’s DM1 gene. In the child’s offspring, the gene will be stronger still. As of this writing, there is no treatment for DM1.
A situation that 5AM Ventures and five other venture capital firms including RA Capital Management and Novartis Venture Fund have devoted $55.3 million to correct. The mutation in the Myotonic Dystrophy Type 1 gene is the result of malfunctioning RNA. RNA transmits messages from the DNA to cells to regulate the synthesis of proteins.
For over a decade a research team led by Dr. Matthew Disney has been researching hereditary illnesses for which no effective treatment is available. These diseases which include DM1 are called Expansion Repeat Disorders. The perpetuation of a defective portion of DNA leads to overproduction of RNAs. Toxicity results when too much RNA has built-up.
Dr. Disney and his team’s strategy involves the use of Small Molecule Medications. Small Molecule Medications control biological processes. These meds are called Expansion Therapeutics. The treatment will specifically target the RNA causing the disease.
Dr. Disney expressed optimism that help for those suffering from Expansion Repeat Disorders is not far off.
Dr. Scott Rocklage has worked in the Pharmaceutical Industry for 30 plus years. He has a BS in Chemistry from UC Berkeley. His PhD. in Chemistry is from MIT. He holds or co-holds more than 30 patents. Dr. Rocklage is responsible for the FDA approving Cubicin a treatment for skin infections and the MRI contrasting agent Omniscan.
Connct with Dr. Scott Rocklage on LinkedIn.