Top research institutions hand hot gene-editing tech--with a catch--to Editas

Katrine Bosley

As a startup, Editas Medicine quickly enjoyed the spotlight as a pioneer in the field of gene editing. Its founding VCs attracted a high-profile biotech CEO--Katrine Bosley--last summer, and now it also has the IP it needs from a group of leading research institutions in the forefront of developing CRISPR-Cas9 and TALENs technology.

Editas has sewn up rights to technology developed by the likes of Dr. J. Keith Joung at Mass General, Duke's Duchenne muscular dystrophy (DMD) expert Charles Gersbach and Editas founders Feng Zhang of the Broad Institute and George Church and David Liu of Harvard University, who have all contributed to a hot new field in drug research. As noted by Editas, DMD is a genetic disease, and the release points to a likely lead focus for the up-and-coming biotech.

CRISPR-Cas9 is considered the best approach to "editing" a gene by splicing in batches of DNA designed in the lab to add a trait or correct a defective gene--like deleting the CCR5 gene from blood-creating stem cells that HIV needs to infect cells. Using this technology, mutations that cause ailments such as sickle cell disease or hemophilia can possibly be repaired. And the huge potential to develop cures has spawned startups like 2014 Fierce 15 company Editas and CRISPR Therapeutics, with the pharma giant J&J ($JNJ) recently jumping into the field with Transposagen.

Editas, though, hasn't sewn up exclusive rights. The Broad Institute of MIT and Harvard and Harvard University are reserving the right to outlicense the IP for genes that Editas can't include in its R&D effort. And they're committed to keeping the technology openly available in the research community to encourage the rapid development of new therapies.

Typically, biotech startups begin operations with their IP safely in hand, making a deal like this a little late in the game. But Bosley tells FierceBiotech that she's quite pleased with the pace of progress at the company.

"In my own experience," notes Bosley in an email, "I find that the sequence of assembling the building blocks for a new company is often quite different from one company to the next. What's critical is finding all of the right pieces--the team, the science, the capital--and then melding them into a cohesive organization with a shared vision and common goals. Editas Medicine was launched just a year ago, and across these agreements with Harvard, the Broad Institute, MGH, and Duke, we've assembled an exclusive combination of rights that is unique in the field."

And she's got the fledgling biotech's sights set on the clinic in due course. "Over the past year we have been building our scientific team--we're currently over 20 employees--and have initiated our first programs internally. We anticipate that we're a couple of years from our first clinical trials, and we look forward to sharing more details with you in the coming year as we advance those programs."

As far as the Broad is concerned, this deal also fits in well with their own strategy of sparking a range of clinical programs at various biotechs.

"The Broad, MIT, and Harvard share the goal of developing innovative technologies such as CRISPR-Cas9 and promoting their translation to benefit patients," said Eric Lander, president and director of the Broad Institute. "We're committed to making these technologies broadly available for research and also ensuring that therapeutic development--bringing this technology to the clinic--has the best chance of success."

- here's the release from MIT and Harvard
- here's a release from Editas, and another, and another (PDFs)

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