Gates backs a $120M breakout round for CRISPR/Cas9 pioneers at Editas

Editas CEO Katrine Bosley

Editas quickly emerged as one of the first biotech upstarts with potentially game-changing gene editing technology in its hands. Now it's rounded up a $120 million megaround to speed its way through the next two years of research, setting up its lead clinical programs for human testing with a gang of investors that includes legendary software magnate Bill Gates and leaving the biotech poised to jump into the IPO queue at the time of its choosing.

The new money, which comes from a mix of public and private outfits that are familiar crossover players in the current boom market for IPO, lands on top of a big $43 million round that got the game started in late 2013. But there was at least one twist.

The round was led by Boris Nikolic, who created bng0 to solely invest in Editas. Nikolic has close ties to Gates, who has discussed his own personal fascination with gene editing tech, and the biotech's execs say that Gates and other members of wealthy clans jumped in for the ride. Then comes a long lineup: Deerfield Management, Viking Global Investors, Fidelity Management & Research, money managed by T. Rowe Price, Google Ventures, Jennison Associates on behalf of certain clients, Khosla Ventures, EcoR1 Capital, Casdin Capital, Omega Funds, Cowen Private Investments and Alexandria Venture Investments. The company's founding investors, Flagship Ventures, Polaris Partners and Third Rock Ventures, as well as Partners Innovation Fund also stayed in the game.

Generally that kind of lineup and timing would suggest that Editas' S-1 filing for an IPO is in the spell-check phase. But the executive team at the biotech--a 2014 Fierce 15 company--says only that they're taking one step at a time and will get around to the IPO as the stars align. The big step now is focusing on groundbreaking human studies.

"The financing really does show tremendous belief with genome editing," says Editas CEO Katrine Bosley, providing a strong financial foundation that will enable multiple programs over time to move into the clinic to "make that promise a reality."

When the mainstream pubs talk about gene editing, the conversation usually turns quickly to Hollywood scenarios, with discussions about tailor-made embryos that would sound like the stuff of scifi fantasies if it hadn't already been tried once by an academic crew in China. But Editas' CEO is an experienced, savvy biotech chief (Bosley negotiated the $925 million deal to sell Avila to Celgene in 2012) with a much more realistic, near-term game plan aimed at new drug development.

At the front of the pipeline you'll find a program for Leber congenital amaurosis (LCA10). A form of the disease is caused by a mutation in the CEP290 gene that leads to blindness. Using guide RNAs paired with the Staphylococcus aureus Cas9 protein--a gene editing scalpel that's small enough to be delivered with an adeno-associated virus (AAV) vector--scientists at Editas were able to correct the mutation in patient fibroblast cells. The team has also worked on sickle cell anemia, another target in gene therapy, though Bosley is reluctant to get very specific about the lineup past LCA10.

Editas created an IP portfolio that started with work done by Feng Zhang, an investigator at the Broad Institute. Zhang in turn was linked with the other pioneers in this field, specifically Jennifer Doudna and Emmanuelle Charpentier, who went on to found companies of their own in gene editing. All the companies--Intellia, now partnered with Novartis ($NVS), Caribou and CRISPR Therapeutics--have been lining up cash and deals, with Editas partnering alongside Novartis rival Juno Therapeutics ($JUNO) in a landmark pact centered on new and improved CAR-Ts and TCRs, which use engineered T cells to attack cancer cells.

Editas CFO Andrew Hack

"This isn't a date," says CFO Andrew Hack. "This is a marriage in CAR-Ts. And who do you want to be married to?"

Considering Celgene's ($CELG) recent billion-dollar tie-up, he answers himself, Juno would appear the obvious answer.

Just like Juno and Novartis, Editas' competition has challenged the Cambridge-based biotech on some of the intellectual property patents filed by Feng Zhang. Bosley counters that this is still a new and developing tech that's improving all the time, trusting in a wide variety of IP sources for the company's foundation. It might also be useful to note that these patent battles have sometimes been resolved without giving away the store, as Novartis demonstrated when it gave Juno a single-digit revenue stream to settle their dispute.

Special Report: FierceBiotech's 2014 Fierce 15 - Editas Medicine

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