ROME Therapeutics

ROME Therapeutics is a biotechnology company based in the Boston/Cambridge, Massachusetts area that develops medicines targeting the so-called dark genome — stretches of DNA that do not encode traditional proteins. This portion of the genome consists largely of repetitive elements, collectively termed the repeatome, from which the company takes its name. Reported repeat families include long interspersed elements (LINEs, ~21%), short interspersed elements (SINEs, ~15%, including Alu elements), human endogenous retroviruses (HERVs, ~9%), and simple and tandem sequence repeats.

The company's lead drug candidate is an inhibitor of LINE-1 reverse transcriptase (RT), a viral-like protein encoded by the LINE-1 element. Activity of LINE-1 RT triggers innate immune responses implicated in autoimmune disease; because it is described as expressed in diseased but not healthy cells, ROME argues that inhibiting it could suppress pathogenic inflammation without broadly impairing responses to infection. The company intends to develop the candidate for serious autoimmune conditions including lupus, taking it through early clinical trials for safety and dose determination plus proof-of-mechanism studies. Beyond the lead program, ROME reports several earlier first-in-class programs across autoimmune disease, cancer and neurodegeneration.

ROME's disclosed leadership includes co-founder, President and CEO Rosana Kapeller, MD, PhD, previously an executive at Nimbus Therapeutics and GV's first entrepreneur-in-residence, and board chair Jeff Hatfield. Other named executives include a chief scientific officer, SVPs of chemistry, strategy and operations, and corporate development, an EVP of early development, a VP of finance, and a director of data science.

Founding story

ROME was founded by Rosana Kapeller, MD, PhD, who developed the concept while serving as GV's first entrepreneur-in-residence, following her earlier work at Nimbus Therapeutics. The founding premise was that the innate immune system's reaction to abnormally activated genomic repeats — such as LINE-1 — contributes to disease, and that technologies newly available in the prior decade (long-read sequencing and machine learning) made this part of the genome tractable for drug discovery. Additional sources name Benjamin Greenbaum and David Ting as co-founders alongside Kapeller.

Business model

Venture-funded, pre-commercial drug discovery and development company; no revenue-generating products are described in the sources. The company funds internal research and clinical development from equity financings, with strategic corporate venture arms of several pharmaceutical companies among its investors.

Traction

Approximately $199 million raised across three rounds; a nominated first-in-class autoimmune development candidate progressing toward early clinical trials; multiple additional discovery programs across autoimmune disease, cancer and neurodegeneration; and a multidisciplinary team spanning oncology, immunology, virology and machine learning.

Latest developments

In September 2023 ROME closed an oversubscribed $72 million Series B extension, bringing total Series B proceeds to $149 million and expanding the syndicate with Johnson & Johnson Innovation-JJDC, Bristol Myers Squibb, Eurofarma Ventures, Luma Group, Mirae Asset Capital, and family offices Raycap and Sigmas Group, alongside existing investors. Proceeds were earmarked for advancing the LINE-1 RT inhibitor into early clinical trials, further pipeline and platform work, and positioning the company as a clinical-stage business.