An international biotech tackles CRISPR gene editing tech with $25M bankroll

A hybrid international biotech upstart with deep U.S. academic roots, a company base in Basel and a plan to grow its research operations in London has landed a $25 million A round to launch another effort at gene editing using CRISPR/Cas9 technology--one of the hottest emerging fields in drug development.

Versant Ventures is bankrolling CRISPR Therapeutics all by itself, for now. CEO Rodger Novak, who had headed a large research team at Sanofi ($SNY) earlier in his career, tells FierceBiotech that the biotech plans to grow a staff of about 20 to 25 employees in the next two years, but he's staying mum for now about which disease targets are at the top of their list.

CRISPR is at least the second biotech to get launched with this technology, closely following Cambridge, MA-based Editas Medicine, which is backed by Polaris, Third Rock and Flagship after recruiting some top scientific talent. CRISPR, though, has some prestigious academic backers of its own, including the Nobel Prize-winning Craig Mello, co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School, and Emmanuelle Charpentier, a professor at the Helmholtz Centre for Infection Research and Hannover Medical School in Germany and the Laboratory for Molecular Infection Medicine at Umeå University in Sweden.

CRISPR-Cas9 uses the Cas9 enzyme to perform surgery, in a manner of speaking, on DNA within the genome to target genetic diseases. And it's billed as a significant step up from the older gene editing technologies. Sangamo's zinc finger approach, for example, has been touted for its ability to tailor T cells, offering a new approach that was used recently to make a group of patients resistant to the HIV virus by mimicking a mutation in the CCR5 gene which was known to spur a high level of immunity.

It's early days, says Novak, but the plan is to move one or two programs into the clinic over the next three years as they build a team with development talent in the U.K. while relying on some virtual staffers to help round out the roster of employees.

"We're interested in strategic relationships with pharma," says Novak, adding that he's been happy making the shift from a large pharma group to a dedicated biotech operation. "I've had some great colleagues," he says, "but I'm for smaller organizations."

The company in-licensed the work that was done by Charpentier, who expects to see plenty of new progress in this area among her academic colleagues.

"CRISPR-Cas9 has truly democratized genome editing, with many areas of research being transformed due to the technology's ease of use and broad applicability," said Dr. Charpentier in a statement. "The collective efforts of the broader academic research community are in turn driving tremendous progress in the field."

CRISPR Therapeutics is also highlighting a new global trend in biotech, a field which knows no boundaries other than the ones we ascribe to it arbitrarily. The money for this work can flow to wherever it can be done the best, allowing more entrepreneurs to think and act globally.

- here's the press release