Our gene control platform consists of two fundamental pillars: identifying novel gene control targets linked to genomically defined patient populations, and drugging gene control targets.
Identifying Novel Gene Control Targets
Starting from human tissue samples, we compare diseased cells to normal cells and analyze the cells of different patient subsets within a disease to systematically and efficiently identify alterations in gene expression programs that represent optimal points of therapeutic intervention and associated biomarkers for patient selection.
To home in on the cell’s gene expression program, we use genomic tools to locate super-enhancers, which are highly specialized regions of non-coding DNA that drive increased expression of the genes crucial to the function of a given cell. Analysis of super-enhancers and their associated genes provides critical insights into changes in gene expression programs that contribute to disease.
We have invested significant resources in our tissue processing, genomics and computational biology capabilities to industrialize the analysis of gene expression programs. We have amassed one of the largest known datasets of gene expression programs across many diseases and cell types. Through those efforts, we have identified multiple novel drug targets and biomarkers in oncology, immuno-oncology and autoimmune diseases. Our long-term goal is to analyze gene expression programs in a wide range of serious diseases where we believe currently underserved patients can benefit from gene control medicines.
Drugging Gene Control Targets
Our internal drug discovery efforts are focused on small molecule chemistry to target transcription factors, transcriptional kinases and other transcriptional and regulatory proteins. Because these specialized proteins play a central role in gene expression, we believe they are among the most promising and high-potential gene control targets for therapeutic intervention. However, these proteins have historically been difficult to drug.
To overcome the challenges that have prevented others from systematically developing gene control medicines, we have built a combination of expertise, tools and capabilities that we believe gives us cutting-edge insights into drugging transcriptional and regulatory proteins. We have made significant investments in developing our capabilities in biochemistry, structural biology and medicinal chemistry to characterize the structure and function of transcriptional and regulatory proteins. We have also developed a sophisticated suite of proprietary assays to measure the biochemical, biophysical, cellular and genomic activity of known and novel compounds against these gene control targets.
We are building a pipeline of gene control medicines through two distinct approaches: our internal drug discovery efforts, and externally focused efforts to link existing drugs to novel genomically defined patient populations identified through our platform. These externally focused efforts could enable us to identify drugs that we could in-license, acquire or use as starting points for our own drug discovery programs to accelerate our development path.