George Church, who is in the thick of both the science and the business of editing genomes with the now Nobel-Prize-winning technology called CRISPR, warmly greeted Wednesday’s award in chemistry to fellow pioneers Emmanuelle Charpentier and Jennifer Doudna.
The Harvard biologist endorsed the Swedish Academy’s choice of Charpentier of the Max Planck Institute in Germany and Doudna of the University of California, Berkeley, for this year’s prize. Their award made history as the only science Nobel ever won by two women. Church praised the pair’s seminal work and dismissed concerns about ongoing patent litigation between the University of California, where Doudna works, and the Broad Institute, home to CRISPR scientist Feng Zhang.
“I think it’s a terrific choice. I think it’s maybe a little iconic and it represents a whole bunch of revolutions that include editing in general,” Church told STAT. “But definitely they made the key discovery, which was that you … can program an enzyme, Cas9, to cleave at a particular place in DNA.”
As for the patent dispute between UC and the Broad that has swirled over who discovered what, when, and in which organism, he said: “I think the patents in general aren’t that critical. I think in this specific case, it’s not that important. I could be wrong, but typically by the time you get through something that’s very useful to society, there’s a lot of winners and they each have a bunch of patents and they cross-license patents and they have different products.”
Church, Doudna, and Zhang started a company called Editas Medicine, and Doudna and Church have been involved in another company, Intellia. Doudna and Church are also working with Inari Agriculture, a seed company whose goal is to reduce the land, water, and other requirements for producing food and feed.
CRISPR Therapeutics and Vertex have launched clinical trials using CRISPR to cure sickle cell disease and beta thalassemia, while Editas is working on a clinical trial using CRISPR to cure a form of congenital blindness. Many other human studies are in the planning stages, with the goal of curing diseases as different as Duchenne muscular dystrophy, cystic fibrosis, type 1 diabetes, hemophilia, and more.
“There’s plenty of different ideas and companies to go around,” Church said. “In fact, if anything, there’s not enough patents. Not enough companies.”
In the research arena, what Doudna and Charpentier did was biochemistry, Church said, which a chemistry Nobel recognizes, as opposed to an invention that might get the nod in medicine.
“They discovered a way to cut DNA, in vitro, not in human cells. I think they knew earlier where this was all going,” he said. “But here, I think the key thing they did was discover this guide RNA can target cutting, that RNA can target cutting.”
Other scientists had worked on gene editing before, Church said, “but this was a key turning point. And then there were people after them that turned it into a technology.”
Looking back, Church mentioned Francisco Mojica, Rodolphe Barrangou, Luciano Marraffini, and even earlier work on informatics that showed “funny repeats” in the genome that looked like junk DNA. And then there were people working a step away from cutting DNA in vitro, he said, which was a step away from actually using it as a gene editor.
“Now that work is making its way into clinical use,” Church said. “This was definitely a turning point from phenomenology in vivo to understanding in vitro and then setting a path towards everything.”
Church counts Doudna as a close friend, but has not yet heard from her about the prize she shares with Charpentier.
“I was with her last night, virtually. I mean, you know, it’s Covid,” he said. “And I sent her an email this morning.”