Scott Rockladge one of the venture captilasits in charge of the group 5AM Ventures has started to make headway in new research aimed at creating a cure for various genetic disorders.
One of these disorders, Mytonic dystrophy type I is well known for being the leading cause of muscular dystrophy in adults. His company recently donated over 50 million dollars to fight this disease. Read more: Scott Rocklage | Crunchbase and Scott Rocklage | Bloomberg
Symptoms of this disease are thought to become present after RNA levels reach a toxic threshold amount. RNA is used to convert the messages inside of DNA into usable proteins by the cell. Typically these RNA molecules are then broken down afterwards and disposed of by the body. In some cases this does not occur and RNA levels build up to toxic amounts causing issues throughout the body.
Expansion Therapeutics has created medications that help treat diseases caused by RNA. Their primary focus is on a subset of these disorders called expansion repeat disorders. These are hereditary illnesses that do not respond to typical medical treatment. These are caused by tiny defective segments of DNA. Myotonic Dystrophy type I is one such disease.
Scott Rocklage recently released a press statement from Dr. Disney which held that now disease related RNA are capable of being targeted with small molecule medications.
This represents a breakthrough in the way these diseases are treated. It is believed they are nearing a point where actual applications will be possible to treat a previously unreachable segment of the population.
These medications depend on science that has the potential to treat RNA based illnesses. Meaning that these medications will be used to treat and even cure diseases that currently have no treatment options. Learn more about Scott Rocklage: https://www.linkedin.com/in/scott-rocklage-66aa7a12a and https://ideamensch.com/scott-rocklage/
5AM Ventures has had a hand in bankrolling these projects. Headed by Scott Rocklage who has over 3- years of experience in the healthcare management industry. He has been at the helm of three medications which have been approved by the FDA Cubicin Teslascan and Omniscan.
With over 100 peer reveiwed publications Scott Rocklage is more than just another businessman with money behind his words but rather an all-new breed of investor who utilizes not just capital but his knowledge of the industry to lead his investment decisions.