Background: RAN translation is the abnormal translation of the body’s native RNA into toxic proteins that occurs in diseases caused by repeat expansion mutations. This results in “jumbled or misread proteins” that can have devastating consequences.
Rationale: Since the discovery of RAN translation in 2011 by Dr. Ranum, the Company’s Chief Scientific Advisor and Co-founder, RAN proteins have been identified in nine different expansion mutation diseases including the most common genetic form of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), myotonic dystrophy, and Huntington’s disease. To date, there are no effective therapies for these disorders despite substantial research efforts by the scientific and medical communities.
Research Details: The Company is working with the University of Florida to transfer technology developed in the Ranum lab to the Company to develop therapies for C9orf72 ALS/FTD and other repeat expansion disorders.