The Science of Reprogramming Cells

We are smart enough to know all the genes in a cell. We believe we can be smart enough to tell the cell what to do.

EPI01 For Sickle Cell Disease and Thalassemia

Why are we testing EPI01, a fetal hemoglobin inducer for clinical use in sickle cell disease and thalassemia?

What did Our Clinical Researchers Do and Find?

  • EPI01 technology was investigated in patients with severe, symptomatic sickle cell disease who had not derived benefit from the standard treatment of hydroxyurea.
  • Oral EPI01, as an investigational therapy, was safe and well-tolerated by the patients.
  • Oral EPI01 demonstrated targeted depletion of DNMT1 and produced large increases in fetal hemoglobin and increased numbers of healthy red blood cells in patients.
  • Investigations are continuing to further document its long-term effectiveness and safety.

What do the Present EPI01 “Proof of Principle” Clinical Findings Mean?

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