Editas-MGH CRISPR Collaboration Expands the Toolbox

Partners HealthCare spinoff Editas rose to fame in 2014 when it was named to Fierce Biotech’s prestigious Fierce 15, an annual compilation of leading biotech upstarts. Since then, Editas’ technology has received worldwide acclaim as a user-friendly way with the potential to edit virtually any site in the human genome. The technique relies on material derived from a bacterial immune system called CRISPR to direct a molecule called Cas9 to specific sites in the genome.
Now the company is refining the tool even further. A new license agreement, announced in August, gives Editas exclusive access to novel MGH technology for use in therapeutics.
The new technology—a collection of advanced Cas9 molecules that can home in on very specific sites in the DNA without introducing detectable alterations at other regions—expands the CRISPR toolbox by allowing researchers to make delicate changes to disease-linked genes without inadvertently disturbing other, nearly identical, areas of the genome. The molecules were designed in the laboratory of Keith Joung, MD, PhD, MGH Associate Chief of Pathology for Research, and the Jim and Ann Orr Research Scholar.
“Collectively, we hope these variants will improve the safety of therapeutics based on Cas9,” says Joung. The variants also expand the potential uses of Cas9 technology by allowing researchers to target more sequences than ever before.
In 2013, Joung co-founded Editas with four other investigators: George Church, PhD, Harvard Medical School; Jennifer Doudna, PhD, University of California, Berkeley; David Liu, PhD, Harvard; and Feng Zhang, PhD, Massachusetts Institute of Technology. Editas is also a portfolio company of the Partners Innovation Fund. The Fund was an original Series A investor in the company.
Editas is currently focused on developing CRISPR-based therapies for blood cancers, Duchenne muscular dystrophy and diseases of the eye and liver, says CEO Katrine Bosley. Each of these conditions stems from a different type of genetic error, so CRISPR-based therapeutics must be nimble, flexible and carefully tailored to each condition.
“These beautiful molecules offer greater flexibility by directing Cas9 to precise locations in the genome,” Bosley says.
The advanced Cas9 molecules mark an important milestone on the route to CRISPR-based medicine. Armed with the ability to make targeted edits, researchers can pursue methods of delivering these alterations to the particular areas of the body affected in each disease. Editas plans to pursue clinical trials of a CRISPR-based therapy for eye disease within the next year.
“CRISPR is a tremendously exciting area of science,” Bosley says. “We’re at the beginning of a whole new era of medicine here.”