Editas' $120 Million Haul Brings New Sources of Funding to Gene Editing Therapies

By Bio-IT World Staff

August 10, 2015 | Editas, a young Cambridge-based company forging ahead with therapies based on the technology of CRISPR gene editing, has more than doubled its financing with a $120 million funding round announced this morning. The impressive haul marks the first influx of money for CRISPR development to come from generalist investors who do not specialize in biotech and the life sciences. Leading the round, which includes over a dozen investors, is a brand new firm, bng0, making its first investment ― with backing from Bill Gates and Boris Nikolic, a science advisor to the Bill and Melinda Gates Foundation.

With biotech money flowing freely, it’s no surprise that new sources of funding are finding their way to CRISPR. The technology, derived from a DNA-cutting protein that bacteria use to fight off viruses, provides a radically simple way to edit the genetic code, opening up new paths to treat rare genetic disorders, cancers, and viral diseases like HIV. And while the sums that Editas has been able to raise might have seemed jaw-dropping when the company was formed two years ago, recent funding rounds at biotech companies like Moderna and Juno Therapeutics have raised the bar for the industry.

It is notable, however, that bng0 and other investment partners have lined up behind Editas, and not one of its competitors in the CRISPR therapy space, Intellia Therapeutics or CRISPR Therapeutics. All three companies have compelling scientific talent and at least tentative lines to large pharma partners, and analysts could make persuasive cases for any of them as natural leaders if and when gene editing reaches the clinic.

An advantage for Editas may be that, at least in public, it has been far more explicit about how exactly it will develop CRISPR from an exciting scientific tool into a safe and effective drug. Editas is the only CRISPR therapy company to spell out a lead program, targeting a form of inherited blindness caused by defects in the gene CEP290. (Disorders of the eye are very popular targets for all forms of gene therapy, because it’s much easier to deliver genetic payloads to the eye than to more insulated organs.) Editas has also specified at least two vehicles it is trying out for getting CRISPR complexes into cells, arguably the biggest bottleneck for the technology. One strategy involves an intricate lipid package, while the other takes advantage of the natural diversity of CRISPR in bacteria to fit a CRISPR system into the tiny adeno associated virus, a classic vector for gene therapies. Put together, these developments make it much easier to say what sort of therapy Editas will be trying to get to clinical trials.

A deal between Editas and Juno also strongly suggests that the company will have a stake in the red-hot field of immunotherapy, editing patients’ own immune cells to fight cancer.

Editas says that its latest funding round puts it on track to operate through 2018, while hiring new scientists to address problems like CRISPR’s tendency to make extra cuts in the genome far from the targeted sites. In the past, big investments and partnerships in CRISPR have tended to pile on top of each other, and it would not be surprising if Intellia and CRISPR Therapeutics now find themselves in a good position to attract new funding of their own.