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 non-toxic, Gene Control Therapy 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.  

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 non-toxic small molecule gene control promises to be a powerful approach to treat a broad range of genetic diseases.  

EPI01 for Sickle Cell Disease

EPI01 is in Human Clinical Studies

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 3 pivotal trial with EPI01 in adult patients with sickle cell disease in 2018.

  Please visit clinicaltrials.gov for more information


EPI01 Inventors at 2016 American Society of Hematology

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