At RaNeDis Pharmaceuticals we are focused on the treatment and management of rare and neglected diseases. RaNeDis Pharmaceuticals is a development stage pharmaceutical company engaged in the development of a novel formulation of an HDAC inhibitor with enhanced brain exposure for the treatment of rare genetic, metabolic diseases.
RaNeDis Pharmaceuticals’ technology was licensed from the University of Notre Dame.
The Company is developing a novel formulation of an HDAC inhibitor, RND-001, that combines an HDAC inhibitor with other agents that improve its ability both to stay longer in the bloodstream and to access the primary target organ, the brain. This formulation has resulted in dramatic improvements of function and survival in a mouse transgenic model of Niemann-Pick Type C disease. Additional research also suggests that this formulation is able to increase the expression of the genes involved in other rare metabolic diseases such as Gaucher’s Disease, GM-1 gangliosidosis, and Krabbe’s Disease. The Company is currently assessing the activity of RND-001 in models of several other monogenetic disorders.