CRISPR Therapeutics

CRISPR CEO Rodger Novak

CRISPR Therapeutics
Revolutionary new gene editing tech has inspired a prominent startup, along with a spat over who controls key patents

CEO: Rodger Novak
Based: Basel/Cambridge, MA
Founded: 2014
Clinical focus: Gene editing
Company website

The scoop: Out of all the great expectations now centered in the biotech field, perhaps none of them exceed the glossy rep enjoyed by gene editing. New CRISPR-Cas9 tech developed primarily by UC Berkeley's Jennifer Doudna and Emmanuelle Charpentier offers the ability to surgically repair a gene, a potentially revolutionary approach to editing out diseases. Sci-fi enthusiasts were quick to seize on the notion of designer babies, but the initial clinical focus will concentrate heavily on practical new therapies that can be tested in human studies. And of all the upstarts in the field, CRISPR Therapeutics has one of the best scientific pedigrees, looking to Charpentier as its scientific founder.

What makes CRISPR Therapeutics Fierce: Great expectations aren't cheap to fulfill. But CRISPR Therapeutics hasn't had to work on a shoestring budget. GlaxoSmithKline ($GSK) and Celgene ($CELG) led the way on a $64 million raise in April, putting their deep pockets to work backing a fast-growing organization. CEO Rodger Novak explained to FierceBiotech that the company will have a presence in Basel and London, but you can expect to see the bulk of its staff in Cambridge, MA.

The initial focus has been on launching a slate of new research projects, with an eye to determining which should be kept in-house and which can be partnered more effectively.

The company has been growing fast. A small team of about a half dozen staffers has been transformed into a group that's now 30 strong, with plans to move into permanent space at the end of the year with room enough to double that roster, says Novak. Around the same time at the end of this year the company expects it will be ready to take the wraps off the lead program, and Novak says you can expect CRISPR Therapeutics as well as the other gene editing leaders to announce new collaborations.

The last big round theoretically could pave the way to an IPO, the CEO acknowledges, but the most important task right now is to reveal how this technology will perform in practical, early-stage research. In the meantime, perhaps other nucleases will pop up that may be useful for particular editing applications, but he's happy that at this stage Cas9 has achieved a prominent and widely studied position in the field and remains the backbone of their R&D effort. That's not likely to change anytime soon.

In the meantime, there's an ongoing dispute over the patents at the foundation of this new biotech field. Doudna and the CRISPR Therapeutics group have been pressing a claim against patents filed by MIT's Feng Zhang, who had signed on with a rival team at Editas (a 2014 Fierce 15 company).

However that turns out, all these companies are scrambling to recruit staffers and get into the clinic in the race to provide some hard data on how gene editing can make a big difference in human health. And CRISPR Therapeutics has a very good shot at breaking radically new ground. 

Investors: SR One, Celgene ($CELG), New Enterprise Associates, Abingworth and founding investor Versant Ventures.

For more:
As patent battle brews, Celgene and GSK lead $64M raise for CRISPR Therapeutics
CRISPR-Cas9 pioneer starts recruiting R&D group in Cambridge hub

-- John Carroll (email | Twitter)

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