Therapeutic Areas

Our Gene Circuitry PlatformTM is the foundation from which we are building a broad therapeutic area-focused portfolio.

Using the insights from our platform, we are creating tissue-specific transcriptional machinery cell maps. Each map serves as its own therapeutic area discovery engine, revealing dozens, sometimes even hundreds of disease-solving opportunities.

We began by generating a liver cell map and mapping is underway to create tissue-based cell maps of the brain, kidney, heart, immune cells, and muscle.

Liver

Discovery Programs:
  • Non-Alcoholic Steatohepatitis (NASH)
  • Phenylketonuria (PKU)
  • Urea Cycle Defects
  • Progressive Familial Intrahepatic Cholestasis (PFIC)

Brain

Discovery Programs:
  • Undisclosed

Heart

Discovery Programs:
  • Undisclosed

Muscle

Discovery Programs:
  • Undisclosed

Immune

Discovery Programs:
  • Undisclosed

Blood

Discovery Program:
  • Diamond-Blackfan Anemia

Kidney

Discovery Programs:
  • Focal segmental glomerulosclerosis
  • Undisclosed

Our goal is to decipher the transcriptional machinery and signaling networks controlling gene expression for cell types central to disease, ultimately delivering druggable targets for a multitude of undruggable diseases.

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