RobertMDS Patient

Developing tamibarotene for patients with
RARA gene overexpression in hematological malignancies


June 25, 2024
Virtual Medical Expert Event

"The Opportunity for Tamibarotene to Transform the Care of Newly Diagnosed Higher-Risk Myelodysplastic Syndrome with RARA Gene Overexpression"


AnneOvarian Cancer Survivor

Expressing the Power of Small Molecules in a Big Way

By controlling gene expression with our small molecule therapies, we aim to make a profound difference in the lives of patients and their families. We are currently developing tamibarotene for genomically defined subsets of MDS and AML patients.

View Our Programs

Our Programs


A selective oral RARα agonist in clinical development for genomically defined subsets of patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia.

View Tamibarotene Program


A novel oral form of arsenic trioxide previously in clinical development for patients with acute promyelocytic leukemia.

View SY-2101 Program


A highly selective and potent oral CDK7 inhibitor previously in clinical development for pancreatic cancer.

View SY-5609 Program

We Understand How the Expression of Genes Is Controlled

Regulatory regions of the genome control the expression of genes, turning them on, off, up or down, like dimmer switches and determining how a cell behaves. Because gene expression is fundamental to the function of all cells, alterations in these regulatory regions contribute to virtually all diseases. Until recently, however, scientists lacked the tools to study these regulatory regions, rendering them largely unexploited for drug discovery and development.

Our gene control platform has led us to discover several targets for small molecule drugs, with the aim to address certain cancers that have eluded other genomics-based approaches.

Learn about our Gene Control Platform