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 activated microglial cells. In a number of neurodegenerative diseases such as ALS, Parkinson's, and Alzheimer's, activated microglia are shown to be responsible for the increased neuroinflammation and subsequent neuronal loss and disease progression in these patients.
Orpheris have 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.
* Preclinical POC in Rett KO model translates to other diseases
Microglia are the resident central nervous system (CNS) macrophage, and can be rapidly activated to the pro-inflammatory state in response to a wide range of CNS insults and diseases. Chronic activation of microglia causes the release of pro-inflammatory agents and triggers a cascade of events, which can harm healthy neurons and other CNS cells. Neuroinflammation caused by activated microglia is present in the majority of neurological diseases and suppression of the activated microglia is currently recognized as a potent therapeutic target.
Orpheris' technology platform consists of hydroxyl dendrimers that specifically target activated microglia and reactive astrocytes associated with neuroinflammation resulting in demyelination and neuronal loss. 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.
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