RobertMDS Patient

Developing tamibarotene for patients with
RARA gene overexpression in hematological malignancies


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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"


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AnneCancer Survivor

Restoring healthy gene expression and myeloid differentiation

At Syros Pharmaceuticals, we're on a mission to offer hope to patients with blood disorders through biologically targeted solutions. We are developing tamibarotene, an oral selective RARα agonist in late-stage clinical development, for higher-risk myelodysplastic syndrome patients with RARA gene overexpression. When RARA is overexpressed, cells in the bone marrow may not differentiate into healthy myeloid cells, which can lead to hematologic malignancies. However, when oral tamibarotene is administered, tamibarotene has been observed to bind to RARα, allowing for the restoration of gene expression and myeloid differentiation. With approximately 50% of MDS patients exhibiting RARA overexpression, tamibarotene has the potential to offer a targeted treatment option. Join us on this journey as we work to pave the way for a brighter future for patients with hematologic malignancies.

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Our Programs

Tamibarotene

A selective oral RARα agonist in clinical development for genomically defined subsets of patients with higher-risk myelodysplastic syndrome with RARA gene overexpression.

View Tamibarotene Program

SY-2101

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

View SY-2101 Program

SY-5609

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