EPI01 is in clinical trials for Sickle Cell Anemia

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We Want Patients and Families to Live Longer & Have Good Quality of Life

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For Us, it’s Personal

We want Sickle Cell Disease and Beta-Thalassemia patients, and those who touch their lives, to feel safe and confident in treating these life threatening conditions. We are developing new gene control medicines that turn ON genes.

EpiDestiny’s first product EPI01 is a Gene Control Therapy under clinical investigation to improve hemoglobin in Sickle Cell Disease.

Gene Control Therapy is a transformative treatment paradigm. We are the leader in gene control clinical research.  We use small molecules to turn ON healthy genes in diseased human cells, redirecting their cell fate “destiny” towards normality.

"Gene control therapy works by turning ON or OFF genes to unleash healthy genes to cure diseases"

EPI01 Turns ON Fetal Hemoglobin at the DNA level to Modulate Sickle Cell Anemia

Our innovative therapies target transcriptional OFF proteins to turn ON gene expression in a cell.  Restoration of normal gene expression by small molecule gene control promises to be a powerful approach to treat a broad range of genetic diseases. 

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EPI01 for Sickle Cell Disease

EPI01 is in Human Clinical Studies

Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study.

Click here for EPI01 PLOS Medicine publication

EPI01 is being investigated in clinical trials in adult patients with sickle cell disease.

EpiDestiny anticipates initiating a phase 2 trial with EPI01 in adult patients with sickle cell disease.

Please visit clinicaltrials.gov for more information.
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