Rosana Kapeller’s Rome Therapeutics has pulled in $77 million to target vast, untapped parts of the genome. With VCs ranging from Section 32 to Sanofi Ventures now behind it, Rome is working to hustle autoimmune and cancer drugs toward the clinic.
Rome’s big idea is to look beyond the 2% of the genome that codes directly for proteins and seek out targets in repeating sequences that make up 60% of human DNA. Initially dismissed as “junk” DNA, research has revealed non-coding regions of the genome play roles in processes such as the modulation of the innate immune response. Rome is working to realize the therapeutic potential of the emerging knowledge of the long stretches of DNA.
Since breaking cover with $50 million in series A funds last year, Rome has identified targets derived from the repeatome. Rome has chemical matter for its first program and is moving it into preclinical tests. Armed with the $77 million in series B money, Rome will work to take its lead program into the clinic in the next few years.
“We’re going to continue to accelerate our leading programs into the clinic, expanding the pipeline and building what we call the repeatomics platform. It's very much going to add a new layer to precision medicine,” Rome CEO Rosana Kapeller, M.D., Ph.D., said.
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A mix of tech and biotech VCs have come together to bankroll Rome’s work, reflecting its position at the intersection of the two industries. Section 32, the VC shop set up by GV founder Bill Maris, led the series B round with assists from fellow new backers Sanofi Ventures, Casdin Capital, Andreessen Horowitz and Alexandria Venture Investments. Existing backers including Arch Ventures and GV also contributed.
Rome will use the money to advance autoimmune and cancer programs while continuing to expand its data science platform. Under the leadership of Menachem Fromer, who Rome hired from Verily earlier this year, Rome has put together the platform to “mine enormous volumes of data,” Kapeller said.
As Rome has grown from a few people to a still-expanding team of 25, it has advanced both the data platform and its targets. The process has revealed challenges to working on targets away from the familiar coding parts of the genome.
When developing traditional therapies, researchers can knock out a coding section of the genome to understand what might happen when the target is inhibited. That is possible because small-molecule targets are typically encoded by two alleles. The vast stretches of repeating DNA require a different approach to target validation.
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“One of the challenges that we have is to use the current genetic tools to modulate the activity of these targets. We rationally understood that, but one of the big challenges in the beginning was how to validate those targets when there are hundreds of copies of them,” Kapeller said.
Kapeller also highlighted the predominance of short-read sequencing as an issue. Short reads work well in many contexts, enabling Illumina to dominate the sequencing market, but they are a poor fit for the identification of active and inactive repeats. “Long-read sequence sequencing is really going to be very impactful for us,” Kapeller said.