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Source: Editas.

It turns out that bacteria could hold a key to transforming how doctors treat disease -- at least that's what Editas, a privately held company that just raised $120 million from investors including Bill Gates, seems to think.

First, a bit of background
Editas was founded in 2013 through $43 million in venture funding from the likes of Flagship Ventures and Polaris Partners and the company is researching a fascinating gene-editing technique based on the immune system of bacteria.

In order to spot and destroy attackers, bacteria memorize a bit of an attacking virus' DNA in the genetic code within their immune system. Bacteria store these bits of a virus' DNA in repeating spaces located within genetic code called clustered regularly interspaced short palindromic repeats, or CRISPR. Then, bacteria pair up that bit of genetic code in the form of RNA with an enzyme known as CRISPR-associated proteins, or Cas that act like scissors, cutting up the genetic code of any invading virus to prevent it from replicating.

This defense system is so simple and effective that Editas scientists think that they can mimic it to create therapies to combat disease and conditions affecting humans, too.

Getting under way
Editas is attempting to create these new therapies by crafting a guide-RNA that targets a specific DNA sequence and then marrying that guide-RNA to Cas9, one of the most common and effective of the Cas proteins found in bacteria.

Initially, Editas targets include a rare genetic disease known as Leber congenital amaurosis, an eye disorder affecting the retina -- tissue at the back of the eye that detects light and color -- that can be caused by at least 14 different genetic mutations, including mutations to the CEP290, CRB1, GUCY2D, and RPE65 genes.  In pre-clinical research, Editas corrected a mutation to the CEP290 gene in primary patient fibroblast cells, suggesting that this approach could one day be used to correct this condition.

Editas is also researching CRISPR therapies for sickle cell anemia and exploring the use of CRISPR approaches in concert with the immune system's T-cells, something that the company is exploring further in collaboration with Juno Therapeutics.

Amassing talent
In order to turn pre-clinical CRISPR research into clinic-worthy therapies, Editas is lining up a top shelf slate of talent that's supported by some of the savviest healthcare venture capital money out there.

Editas founders include Feng Zhang, one of the most prominent names in CRISPR research, and its CEO is Katrine Bosley, a biotech veteran that was formerly CEO at Avila Therapeutics, a company she sold to Celgene for $350 million, plus potential milestones, in 2012. Also among the individuals packing the company's leadership is the senior director of molecular and cell biology, David Bumcrot, who came from RNA research pioneer Alynylam.

Editas' board of directors is also made up of a who's who of biotech venture capitalists, the most recent of whom is Boris Nikolic, the former chief scientific advisor to Bill Gates at Gates' Catalyst 3 and the Bill & Melinda Gates Foundation. It's Nikolic who ostensibly lined up Gates as an Editas investor through his newly created investment company bng0.

Looking ahead
Focusing on localized tissue, such as the retina, makes the most sense early on because CRISPR, for all its promise, still poses risks that aren't fully understood. Although CRISPR targets pretty accurately, there's still the potential for off-target genetic code to get incorrectly snipped and that could lead to all sorts of unintended complications and risks.

Ultimately, applying CRISPR to a wider swatch of diseases could make the biggest impact, but we're arguably years away from getting to that point. Hopefully, with excitement translating into bulging R&D coffers from investors like Gates, advances that lead to revolutionary new treatments will occur more quickly.

 

Todd Campbell is long Celgene. Todd owns E.B. Capital Markets, LLC. E.B. Capital's clients may have positions in the companies mentioned. The Motley Fool recommends Juno Therapeutics. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.