A recently discovered tool that gives scientists the ability to edit DNA — the basic blueprint for life — and to rewrite the genetic codes of humans and other creatures just got a major boost toward breaking through from research to applications that might affect human health.
Editas Medicine is one of the first companies using this genome-editing tool, CRISPR-Cas9, to develop new ways to cure human disease. On August 10, the startup announced that they have raised $US120 million from a group of investors led by Dr. Boris Nikolic, the managing director of bng0 (which was formed to invest in Editas) and who was before this was a chief science and technology advisor to Bill Gates.
“The company is at the forefront of genome editing — one of the most exciting and important frontiers in science,” Nikolic said in a press release.
The massive investment is a vote of confidence in a tool that many scientists say could change everything — and in Editas Medicine’s ability to use it wisely.
Gates has been confirmed to be one of the bng0 backers, according to Forbes’s Matthew Herper.
The company was founded in 2013 with $US43 million from Flagship Ventures, Polaris Partners and Third Rock Ventures, as well as Partners Innovation Fund. Other new investors include a long list: Deerfield Management, Viking Global Investors, Fidelity Management & Research Company, funds and accounts managed by T. Rowe Price Associates, Inc., Google Ventures, Jennison Associates on behalf of certain clients, Khosla Ventures, EcoR1 Capital, Casdin Capital, Omega Funds, Cowen Private Investments and Alexandria Venture Investments.
Why it matters
This news is a major development because Editas is one of the leading startups coming up with ways to use CRISPR-Cas9 to eliminate human diseases in ways that weren’t possible in the past. Such a massive spike in the financial resources of the company could have a transformative impact on health and medicine — and of course, as Wired pointed out in a recent feature on how CRISPR will change the world, health and medicine is where the real money is.
Researchers think they will be able to cure genetic diseases with CRISPR by changing the part of a person’s genome that causes that disease. If a person has a disease caused by a gene or a specific mutation, scientists think they will be able to use this tool to rewrite or delete the parts of a genetic code that cause that illness. Right now, those ideas are still largely hypothetical: We haven’t used CRISPR to cure anything just yet.
Editas is first trying to use CRISPR to treat a cause of blindness by snipping out a genetic mutation, the company’s CEO told Forbes. After that, they may try to treat a blood condition by actually replacing a section of DNA with a new section of DNA.
The ability to make these changes is what gives CRISPR its incredible promise, but researchers haven’t yet figured out how to make this happen safely and effectively enough to do this work in humans. They can delete or even replace sections of DNA in certain lab settings without many unwanted other effects (like deleting other, desired sections of DNA), but can’t do it consistently enough yet to try this in people.
George Church, a leading Harvard geneticist and CRISPR researcher who is a scientific advisor for Editas, previously told Tech Insider in an email that he thinks researchers might eventually be able to use the tool to change genes so that people wouldn’t be vulnerable to HIV.
And that’s just the start. In a series of interviews, a number of researchers have told Tech Insider that CRISPR’s implications for human health — and for transforming the world in other ways — are almost impossible to overstate.
As Jennifer Doudna, one of the first to discover the capabilities of CRISPR, told us: “We’re basically now able to have a molecular scalpel for genomes.”
A possible wrinkle
Editas was founded by some of the first scientists to discover CRISPR, including Doudna (of the University of California, Berkeley) and Feng Zhang of the Broad Insitute of MIT and Harvard.
There’s now a patent dispute over who owns the intellectual property rights for the genetic-editing technology. Doudna and Emmanuel Charpentier published some of the first work on CRISPR and filed for a patent; Zhang filed for another patent after subsequently publishing other work and his patent was granted first. Now, Doudna has licensed her patent to another company, while Zhang’s is licensed to Editas, and Charpentier’s is licensed to yet another group.
Still, Editas’s list of scientific advisors includes some of the top researchers working with CRISPR, and various investors told Forbes they aren’t worried about how courts settle the intellectual property dispute.
The worst-case scenario is that they have to pay a royalty, but if they’re the first company to use genome editing to cure human disease, paying a royalty would be well worth it.
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