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.
Our pipeline
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)
What’s New
First Preclinical Data of ROME’s LINE-1 RT Inhibitors in Neurodegenerative Diseases Presented
At the 3rd annual Dark Genome Symposium, ROME presented a poster showing that our proprietary LINE-1 reverse transcriptase (RT) inhibitors demonstrated neuroprotective effect on dopaminergic neuron survival in both in vitro and in vivo neurotoxin models of Parkinson’s disease and a dose-dependent decrease in α-synuclein expression, a key pathogenic driver of Parkinson’s disease.
Latest news and presentations
Risky Moves: Can blocking "jumping genes" treat diseases and aging? | Science
ROME Therapeutics Announces Publication in Nature Communications of First Large-Scale, Pan-Cancer Analysis of LINE-1 Expression Levels and Retrotransposon Activity
ROME Therapeutics Debuts First Preclinical Data of its LINE-1 Reverse Transcriptase (RT) Inhibitors in Neurodegenerative Diseases at 3rd Annual Dark Genome Symposium