Looking closely at the logo for newly launched Omega Therapeutics Inc., there's a definitely an "O" for omega. But there's more. Lurking right there alongside the "O" is also an "A," indicating quite a wide range and vision.
"If we think about how our platform and science work, that we're pathbreaking a new class of therapeutics for the future of medicine, it essentially means that we're tackling every disease that manifests itself as a result of the dysregulation of genes," Mahesh Karande, Omega's president and CEO, told BioWorld. "We realized we must think in a transformative way and we thought also of the last letter in the Greek alphabet. It's the end-all. Once you have that, we figured you can transform all of medicine."
Karande said Omega has a transformative way of looking at genomic medicine so that it can venture into treating genetic disease, immunology, inflammation, metabolic disease and oncology. He maintained that Omega is the "first to drug domains that have never been drugged before."
Omega is the latest creation from venture capital firm Flagship Pioneering. Cambridge, Mass.-based Omega's platform is designed to treat a variety of indications by controlling genomic expression while leaving nucleic acid sequences unchanged.
The company's platform is able to precisely tune insulated genomic domains, which control genomic activity, without altering nucleic acid sequences, according to Karande. Unlike other modalities where there is only the ability to tune up or down, Omega can do both, either disrupting its formation or encouraging its reformation.
"The key is that very few modalities allow you to tune," Karande said. "If you look at other modalities, or gene editing, you're cutting something out. Nobody was actually able to tune to the right level of expression that's required to treat a particular condition. We are able to target them."
Flagship's labs conducted the initial research into unleashing the human genome's native capacity, along with the Whitehead Institute for Biomedical Research, which is also in Cambridge. The initial work on epigenetics was conducted by Richard Young, a professor of biology at MIT and a member of the Whitehead Institute, and by Rudolf Jaenisch, also a biology professor at MIT and a founding member of the Whitehead Institute. They described how genomic activity is controlled by 3D closed-loop structures of DNA, now called insulated genomic domains, which contain one or more genes and their regulatory elements.
Those academic and organizational founders of the company collaborated to find that insulated genomic domains exist and that DNA is folded into those domains, which control gene expression.
"That's the work that was done and it gave us the confidence to set up the company," Karande said. "My team in the past couple of years has worked through and taken it to proof-of-concept level. We have more than 10 different genes that we have studied."
The research allowed Omega, which was formed in 2017, to achieve early proof of concept in a number of disease models, according to Tom McCauley, Omega's chief scientific officer.
"We're pushing them forward to development in the next six to nine months," McCauley told BioWorld. "We hope to knock on the door to the clinic by 2021."
McCauley was chief scientific officer of Zikani Therapeutics Inc. and at Lexington, Mass.-based Translate Bio Inc., which is developing CFTR gene modulator MRT-5005 to treat cystic fibrosis and OTC gene stimulator MRT-5201 to treat ornithine transcarbamylase deficiency. (See BioWorld, May 2, 2019.)
None of the launch's financial details were announced.
"We're not disclosing any specifics about the fund raise," Karande said. "We're not worried about funding. We have strong financial backing from Flagship."
Flagship has founded more than 100 companies and initiated 20 IPOs since 2013. Among its companies include Rubius Therapeutics Inc., which filed its IPO last year. (See BioWorld, July 19, 2018.)
Karande, formerly of Macrolide Pharmaceuticals Inc. and Novartis AG, also serves on the company's board. He said the company platform is very broad and that while the primary goal is to create therapeutics for patients, it can also optimize other therapeutics, opening the door to partnering with others, including cell therapy companies.
"That's a major thrust of partnership. If someone approaches us, we are open to partnering," he added.
As it now stands, Omega has upwards of 35 employees but "hiring significantly is our plan," Karande said.