Orpheris is a clinical-stage neuroscience company leveraging a novel platform technology from Johns Hopkins that enables the selective delivery of therapeutic agents across the blood barrier into re-activated microglial cells.
In a number of neurodegenerative diseases such as ALS, Parkinson’s, and Alzheimer’s, re-activated microglia are shown to be responsible for the increased neuroinflammation and subsequent neuronal loss and disease progression in these patients. Orpheris has developed a rich pipeline of therapeutic candidates targeting multiple pathways believed to be responsible for driving microglia activation and disease progression. Each drug candidate is engineered with the hydroxyl dendrimer technology to deliver therapeutic payloads across the blood brain barrier into activated cells with no off-target toxicity. The hydroxyl dendrimers used in our platform rapidly distribute throughout the body after systemic administration, do not bind to tissues or proteins, are non-toxic, and are excreted intact in the urine.