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 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.
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.Learn more...
Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study.
EpiDestiny anticipates initiating a phase 2 trial with EPI01 in adult patients with sickle cell disease.