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Driving CRISPR to the Clinic

Keith Joung, MD, PhD

In the short time since the emergence of CRISPR gene editing technology, Keith Joung, MD, PhD has been impressed with all the research applications that have been developed to harness its potential.

“It is remarkable how it’s been used to build cell lines and model organisms, gain new insights into more of the mechanisms of disease and understand why certain mutations or gene alterations have the impacts they do,” Joung says.

Dr. Joung, an investigator in the Department of Pathology at Massachusetts General Hospital and Professor of Pathology at Harvard Medical School, has played a key role in many of these breakthroughs through the work in his lab—and through open-source sharing with other scientists.

As the recent co-founder of the biotech companies Editas Medicine and Beam Therapeutics, he is poised to play a key role in bringing this technology to the clinic.

“I’m a physician-scientist by training,” he says. “Although my lab does a lot of basic science, the dream is to see those technologies implemented in patient care. I always knew that if we wanted to see that happen, industry would have to be involved at some point.”

Expanding Into Industry

Dr. Joung’s first foray into industry came in 2013 when he joined four other scientific leaders in the gene editing field—Feng Zhang, PhD, of MIT and the Broad Institute; George Church, PhD, of Harvard; Jennifer Doudna, PhD, of UC Berkeley; and David Liu, PhD, of Harvard—to launch the Cambridge, MA-based Editas Medicine.

Joung has founded two more companies with Editas co-founders Zhang and Liu. In March 2018, the three launched Pairwise Plants, which will look at ways to apply gene editing technology in agriculture.

In May 2018, they launched Beam Therapeutics to explore applications for the DNA-based editing technology that was developed at Liu’s lab at Harvard and additional RNA-based editing technology developed in Dr. Zhang’s lab at MIT/Broad.

Balancing Academia and Industry

Joung’s forays into the worlds of entrepreneurship and therapeutic development have provided an opportunity to grow and refine a new set of skills, although he remains firmly rooted in his academic lab.

“In terms of my day-to-day life, it’s a relatively small percentage of my daily activity,” he says. “My actual day job is running my lab, so the vast majority of my time is focused on that. But the outside consulting activities make my days very interesting. There is no one day exactly like another.”

Venture capitalists have provided more than $155 million to grow the multiple companies he co-founded.

“We have been very committed to open-source well before CRISPR arrived,” he says.

“Because we were doing that already with zinc fingers and TALENs when CRISPR emerged, the precedent had been set. So, everyone who worked on CRISPR technology followed that example and tried to share everything. That has been nice to see.”

“I will say that the MGH and Partners Innovation have been great in supporting us… there has never been any doubt in my mind that they were 100 percent behind us as we’ve put those reagents out there for people to use.”

What’s Next

Joung is excited to see what the next few years will bring with the hope that eventually patients with serious diseases will have access to CRISPR and other gene-editing therapeutics. Seeing the results of the first treatments may provide some extra motivation for him and his team.

“You see what you are doing in the lab might actually matter and could impact the development of therapeutics. But I enjoy the work and think it is fun and interesting, so it’s not like I need a lot more motivation.”