Jennifer Doudna, Emmanuelle Charpentier - Caribou Biosciences, CRISPR Therapeutics

The two scientists' work on gene editing takes the biopharma world by storm

Names: Jennifer Doudna, Emmanuelle Charpentier
Title: Founders, Caribou Biosciences and CRISPR Therapeutics

Scientists rarely make their way into the industry limelight. Years spent in the lab patiently laboring toward a breakthrough often involves painstaking effort building on the work of others. And even the most brilliant investigators tend to be on the sidelines when the plaudits are handed out to the clinical-stage developers and marketing gurus that capitalize on their advances.

Jennifer Doudna and her colleague Emmanuelle Charpentier seem determined to buck that state of affairs. Both are at the cutting edge of the development and commercialization of gene editing. And in cofounding two of the leading companies in the field, they're helping to inspire a new generation of academic entrepreneurs step up with their own therapeutic advances, promoting a tech vision which can only benefit the biotech world.

Doudna, a UC Berkeley structural biologist and Howard Hughes Medical Institute investigator, did some of the pioneering work on gene editing with Charpentier. The two were awarded 2015 Breakthrough Prizes in Life Sciences, while Doudna also took home the Lurie Prize in the Biomedical Sciences in 2014. Doudna launched Caribou and was one of the original founders of Editas before splitting away and backing the upstart Intellia.

That change in camps highlighted what would become a showdown for control of the patents behind the gene editing technology, which revolves around a new and improved method for DNA splicing in editing out diseases. MIT's Feng Zhang signed on and stayed on with the Editas group, which is now defending its patents against a challenge from the University of California. And UC represents both Doudna and Charpentier, who helped found the newly financed biotech CRISPR Therapeutics, which is setting up its research group in Cambridge, MA.

With the ecosystem for biotech and drug developing flourishing around the globe, new technologies like this are quickly finding major backers. In Intellia's case, Novartis ($NVS) stepped in, looking to keep a tech edge that had already been honed in CAR-T. CRISPR Therapeutics, meanwhile, just earned a $64 million Series A, which gained the backing of Celgene ($CELG) and GlaxoSmithKline's ($GSK) venture arm. And the big companies are helping to bankroll an accelerated effort in the clinic, which promises to help shave years off the time it takes to demonstrate a new technology's potential.

That new sense of urgency gives scientists a much more central role in the industry, one that should command a significant slice of equity in what's being discovered in the lab. And that can help change lives and inspire more company creation--one of the most influential trends in biotech.

-- John Carroll (email | Twitter)

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
Novartis steps in to help bankroll CRISPR-Cas9 pioneer Caribou

Jennifer Doudna, Emmanuelle Charpentier - Caribou Biosciences, CRISPR Therapeutics