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Biotech Developing Genome-Adjusting Disease Treatments

DNA analysis graphic

(Gerd Altmann, Pixabay)

23 Sept. 2019. A new biotechnology enterprise is creating disease treatments that address regions in the genome precisely regulating expression of proteins. Omega Therapeutics Inc. in Cambridge, Massachusetts emerged from stealth mode today, announced by Flagship Pioneering, its founder and incubation site.

Omega Therapeutics says it’s developing precise therapies that adjust the expression of genes, without editing their sequences in the genome. The company’s technology targets regions of the genome it calls insulated genomic domains. These three-dimensional genomic regions act as self-contained closed-loop controllers of DNA that Omega calls GenomeStats, which operate like thermostats to control temperature. The company says its therapies, called Omega Controllers, can precisely tune the genome’s activities to express proteins addressing the root causes of many diseases.

Omega’s work is based on research by biologists Richard Young and Rudolf Jaenisch at the Whitehead Institute affiliated with Massachusetts Institute of Technology. Young and Jaenisch study mechanisms in the genome that regulate gene expression. Among their discoveries are 3-D arrangements in the genome they call neighborhoods that regulate gene expression. In a 2016 paper published in the journal Science, Young, Jaenish and colleagues describe how in cancer-causing genes, these neighborhoods can be disrupted to bring them under the control of regulatory elements in the genome.

Omega Therapeutics says humans have some 15,000 insulated genomic domains — what Young and Jaenisch earlier called neighborhoods — that it identified and characterized, providing targets for its Omega controllers. The controllers are designed to bind to targeted insulated genomic domains, in specific ways to boost or restrict their protein expressions. The company expects to create candidate treatments through preclinical stages for rare genetic diseases, immunology, inflammation, metabolic diseases, and cancer.

Omega was founded two years ago by David Berry, a general partner at Flagship Pioneering, and Noubar Afeyan, CEO of Flagship Pioneering, who continues as Omega’s board chair. Berry and Young are members of Omega’s board, and Young is also a scientific advisor. The company incubated at Flagship Pioneering since 2017.

“By mapping the topology of the human genome and using computational biology,” says Berry in a Flagship statement, “we have been able to identify the correct genomic target for a therapeutic influence. Omega’s proprietary platform technology allows us to develop therapeutics for disease control, and potentially impact the way we treat and manage a broad range of illnesses.”

Flagship Pioneering, also in Cambridge, is both an incubator of new science-based enterprise, as well as venture financier, although its investments in Omega Therapeutics are not disclosed. Flagship’s process takes promising ideas from the lab, and creates prototype companies, then new companies built around those ideas. Once the new companies reach growth stage, they’re spun out to attract outside investors. Flagship says it creates six to eight new companies each year, and 20 of its growth-stage companies since 2013 are now publicly traded enterprises.

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