Orpheris is focused on the treatment of neuroinflammatory diseases that result in death or severe disabilities, including childhood cerebral adrenoleukodystrophy (ccALD), Parkinson's disease, amyotrophic lateral sclerosis (ALS), Huntington's disease, Rett syndrome and autism spectrum disorders.

Orpheris' patented hydroxyl dendrimer technology selectively targets activated microglia implicated in a number of brain diseases. Controlling neuroinflammation by targeting activated or neurotoxic microglia with OP-101 is a potent weapon against CNS disorders. OP-101 is the company's first potential drug candidate utilizing hydroxyl dendrimer technology to deliver therapeutic doses of an approved anti-inflammatory drug across the blood brain barrier to reduce neuroinflammation.

The selective targeting of OP-101 to the activated microglia in the brain, after systemic administration, enables intracellular concentrations of an approved anti-inflammatory, anti-oxidant agent not previously possible. Pre-clinical data has demonstrated that OP-101 may reduce disease progression by arresting the neuroinflammatory cascade, which is driven by oxidative stress and the release of proinflammatory cytokines. Shutting down this process converts the microglia from an activated, neurotoxic state to a resident neuroprotective state.