A new frontier in biology
A new way to conquer disease
At ROME Therapeutics, we are illuminating the dark genome to pioneer a new approach to treating serious diseases.
For decades, drug discovery has focused on just 2% of the human genome. The remaining 98% is known as the dark genome: vast regions of underexplored territory and untapped therapeutic opportunity.
We believe the dark genome holds the next wave of therapeutic approaches and insights. We are on a mission to leverage these insights to develop breakthrough treatments for people with cancer, debilitating autoimmune diseases and beyond.
What is the dark genome?
The dark genome consists of long stretches of DNA outside of the genes that encode traditional proteins. The majority of the dark genome is comprised of repetitive elements, or genomic repeats.
Many genomic repeats originated from ancient viruses and retrotransposons that have multiplied themselves throughout our genome. In healthy cells, they are inactive. However, a growing body of evidence shows that when cells are injured or diseased, repeats are turned on — serving as a disease-specific signature that we can target therapeutically.
ROME has nominated a development candidate that represents a first-in-class approach to the treatment of autoimmune diseases.
We are advancing a deep pipeline of novel therapies informed by the insights generated by our data science platform and our deep knowledge of repeat biology.
Halting the LINE-1 lifecycle
ROME’s lead program is an inhibitor of LINE-1 reverse transcriptase, a protein produced by the virus-like genomic element LINE-1. This transposable element is normally dormant in our DNA. However, when activated, it can trigger disease-driving inflammation and genomic instability. Activation of LINE-1 has been linked to autoimmune disease, cancer, neurodegeneration and aging. Watch this video to learn more about the LINE-1 lifecycle.
MoA animation by Visual Science, 2023, and published by Baldwin et al. in Nature (doi 10.1038/s41586-023-06947-z)
Latest news and presentations
ROME Therapeutics today announced that members of the ROME Senior Leadership Team will participate in the TD Cowen 44th Annual Health Care Conference.
ROME Therapeutics Appoints Heike Keilhack, Ph.D., as Chief Scientific Officer
ROME Publishes Landmark Nature Paper Revealing First High-Resolution Structure of LINE-1 Reverse Transcriptase (RT) for Drug Discovery