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ROME Therapeutics Demonstrates Efficacy of its Novel LINE-1 Reverse Transcriptase Inhibitor in Translational Models of Autoimmune Disease

Company to share preclinical data from lead program in autoimmune disease at American College of Rheumatology (ACR) Convergence 2022   

BOSTON, Mass. – November 10, 2022 – ROME Therapeutics, a biotechnology company harnessing the power of the dark genome to develop breakthrough medicines for cancer and autoimmune diseases, today announced it will share preclinical data from its lead program, a LINE-1 reverse transcriptase inhibitor, in a poster presentation at American College of Rheumatology (ACR) Convergence 2022, taking place November 10 – 14, 2022, in Philadelphia.

LINE-1 reverse transcriptase is an endogenous reverse transcriptase (eRT) encoded by repetitive sequences in the human genome. eRTs convert RNA into DNA in the cytosol, triggering a viral mimicry response via nucleic acid-sensors, such as cGAS, resulting in activation of Type 1 interferon and NFkB pathways. Since excess activation of these pathways can lead to autoimmune disease, eRT inhibitors have great potential to improve pathological outcomes.

Through rational structure-based drug design, ROME identified novel, selective and potent inhibitors of LINE-1 reverse transcriptase. The data show that ROME’s compound inhibited a Type 1 interferon response in multiple in vivo and in vitro models of autoimmune disease.

“Endogenous reverse transcriptases encoded by repetitive sequences in the human genome are increasingly implicated in many difficult-to-treat diseases, including autoimmune disease. At ROME we have identified first-of-their-kind selective inhibitors of LINE-1 endogenous reverse transcriptase, a target that triggers inflammatory responses,” said Dennis Zaller, Ph.D., Chief Scientific Officer of ROME. “We are excited to share new preclinical data demonstrating that our LINE-1 reverse transcriptase inhibitor effectively blocks inflammatory Type 1 interferon responses that drive many intractable autoimmune diseases. This demonstrates the promise of therapeutic targets within the dark genome, and we look forward to advancing this program with the goal of offering a new treatment option for diseases with great unmet need.”

Details of the poster presentation are as follows:

Abstract # 1698

Title: LINE1 Reverse Transcriptase Inhibitors Abrogate Type 1 Interferon Responses

Presenter: Daniel Rios, Principal Scientist, ROME Therapeutics

Session: Innate Immunity Poster: Basic and Translational Science

Date: Monday, November 14, 2022

Time: 1:00-3:00 p.m. ET

About ROME

ROME Therapeutics is developing novel therapies for cancer and autoimmune diseases by illuminating the role of the dark genome in human health and disease. Leveraging the company’s unprecedented data sciences platform, ROME has built a deep pipeline of therapies targeting the dark genome. To lead this exploration, ROME has assembled a team of world-class leaders across oncology, immunology, virology and machine learning. ROME is based in Boston, Mass. For more information, please visit www.rometx.com.

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