Our Gene Control Platform

Our gene control platform has served as the foundation for our clinical stage pipeline and has been used to identify targets and biomarkers for drug development with the aim of providing a profound benefit for patients with diseases that have largely eluded other genomics-based approaches.

Regulatory Genomics

Regulatory regions of the genome control the expression of genes, turning them on, off, up or down like genomic dimmer switches. Through the coordinated activation and repression of genes, these genomic dimmer switches determine how a cell behaves, whether it is a skin cell or a lung cell, whether it is diseased or normal. Our gene control platform has allowed us to systematically analyze regulatory regions of the genome, identifying a very small subset of these genomic dimmer switches, known as super-enhancers, that control the expression of genes most critical to a given cell. By comparing super-enhancers and their associated genes in healthy and diseased cells from patient tissues, we have identified disease-causing alterations in the expression of critical genes and we have homed in drug targets and patient subsets that we believe provide the best chance of providing a therapeutic benefit for patients.

Disease Biology

We have integrated our understanding of the regulatory genome with deep expertise in the biology of the diseases that we aim to treat, including cancer and genetic diseases It has also allowed us to place our novel findings into the broader context of the disease, weaving together our insights with what is already known about the disease to further inform our decisions about which genes to control in which patients and how best to modulate those genes to discover and develop medicines most likely to provide a profound and durable benefit for patients with diseases that have eluded other approaches.

Small Molecule Chemistry

We have identified small molecules to selectively modulate transcription factors, transcriptional kinases and other regulatory proteins to control the expression of genes. Transcription factors, transcriptional kinases and other regulatory proteins represent among the most promising and high-potential gene control targets for therapeutic intervention because of the central role they play in carrying out the process by which genes are expressed. These specialized proteins have been historically difficult to drug, representing a largely untapped opportunity to bring novel and differentiated therapies to patients.