Physciocrine Pathways

Our scientists were the first to identify the Resokine pathway and we are harnessing this pathway and its association with homeostasis in skeletal muscle to develop Resolaris, our first clinical product candidate, as a first-in-class therapeutic for patients with rare myopathies with an immune component, or RMICs, for which there are limited or no approved treatments.

We have shown that stimulation of the Resokine pathway by Resolaris alters immune responses and the expression or release of immune-related proteins from cells in response to inflammation. We selected immuno-modulation as our initial area of focus for the following reasons:

  • We believe immunology plays a significant role in most diseases, including genetic diseases
  • A number of Physiocrines have been shown to be differentially expressed in immune cells
  • A large number of Physiocrine pathways appear to relate to immunology, as at least seven different tRNA synthetase proteins are associated with certain immune-driven diseases
  • Approximately 100 Physiocrines have demonstrated activity in various cell-based assays related to immunological pathways

Our initial therapeutic efforts target severe, rare disease indications in which patients suffer from the immune-related consequences of their genetic disease because:

  • Our scientific understanding of Physiocrines as immuno-modulators intersected with multiple rare diseases
  • We believe patients with rare genetic diseases often face challenges related to the responses of their immune systems to changes in tissues that are caused by their genetic mutations
  • We believe the pathological immuno-phenotypes in rare diseases present an opportunity for us to therapeutically intervene with greater impact