There are approximately 200 different cell types in the human body. Despite having identical genomes, each of these cell types has a different function. For example, a skin cell functions differently from a muscle cell despite sharing the exact same DNA. What determines a cell’s type and function is the specific set of genes that is expressed, or turned ‘‘on’’ or ‘‘off,’’ in that particular cell. This coordinated activation and repression of genes, known as the cell’s gene expression program, is controlled by non-coding regions of the genome. Alterations in these non-coding regions change a cell’s gene expression program, altering its normal function and leading to disease. Because this biology is fundamental to the function of all cells, it applies across diseases, whether the cause is genetic, environmental, bacterial, viral or multi-factorial.
Although researchers have long believed that alterations in non-coding regions of DNA, which account for 98% of the genome, play a key role in driving disease, the scientific community has lacked the tools to study these regions of the genome, rendering them poorly understood. As a result, the discovery and development of targeted therapies to date has focused almost exclusively on abnormal proteins resulting from genetic alterations found in regions of DNA that code for proteins, which represent less than 2% of the entire genome.
While targeted therapies have dramatically improved the ability to treat certain cancers and other serious diseases, the identification of new drug targets by sequencing coding regions of DNA has been largely exhausted. In cancer, inhibiting abnormal proteins resulting from single genetic alterations can often lead to drug resistance and limited durable clinical benefit. Moreover, many other serious diseases continue to go unaddressed due to the limitations of current drug discovery approaches. Taken together, these factors underscore the need for fundamentally new approaches to drug discovery and development.
Building on the work of our scientific founders, we have developed what we believe is the first proprietary platform designed to systematically and efficiently analyze non-coding regions of the genome to identify alterations in gene expression programs. By doing so, we believe our gene control platform will allow us to:
- Identify a wide array of potential new drug targets across a range of diseases
- Provide a new lens for diagnosing and segmenting patients, including those with complex, multi-factorial diseases that have eluded segmentation with other genomic-based approaches
- Advance a new wave of medicines with the potential to influence multiple drivers of disease through a single target, making them less susceptible to drug resistance and providing patients with a more profound and durable benefit than many of today’s targeted therapies.