Crispr’s Next Frontier Is In-Human Treatment, Co-Inventor Says

(Bloomberg) -- As investors await results from the first U.S. clinical trials of the gene-editing system known as Crispr, scientists are focused on finding ways to administer it directly into humans, according to the technology’s co-inventor, Jennifer Doudna.

Right now, in studies using Crispr that have treated patients, researchers have had to extract their cells to be able to make edits to faulty DNA before infusing them back into the body for treatment. Being able to do precise edits directly inside humans, animals or plants could open the door to new applications, Doudna said.

“With advances and delivery techniques, it may be possible to do that kind of very highly efficient targeted genome editing in the patient, without having to remove cells, but actually to just do a treatment in the patient where the delivery vehicle takes the editing molecule to the right cells,” she said in a telephone interview before the Welch Foundation Conference on chemical research next week. “Sounds fantastical today, but I think that’s coming.”

In essence, Crispr is a gene-editing system that can splice away parts of human DNA that make people susceptible to disease or defects. While it can be used in plants and animals, scientists are working on therapeutic applications that can offer a one-time cure for certain diseases. Crispr Therapeutics AG was the first company to start a human trial back in February, and is due to report initial results by year-end.

Editas Medicine Inc. is leading efforts in “in-vivo,” or inside the body, testing and initiated a clinical study in July. Intellia Therapeutics Inc. is expected to follow with its own study next year. A safe delivery of Crispr directly into humans would shorten manufacturing times and offer new opportunities for the companies.

The biggest challenge is to find a way to deliver gene-editing molecules into specific cell types safely and efficiently, Doudna said. “That’s kind of the next frontier,” she said. “If we figure that out, it really does open the way to many, many more kinds of applications in genome editing than are possible today.”

Crispr and Intellia Therapeutics have licensed their technology from the University of California at Berkeley, Doudna’s academic home, while Editas is using inventions from the Broad Institute in Massachusetts. The two institutions are fighting over who was first to invent breakthrough gene-editing technology. Doudna is a co-founder of Editas and other Crispr startups and is a scientific board member at Intellia.

The gene-editing field, which only recently entered human testing and has been plagued by research raising safety concerns, recently got some encouraging news. Chinese researchers safely treated a man with leukemia and HIV using gene-edited stem cells, according to a report in the New England Journal of Medicine. While the attempt to cure his HIV failed, his cancer is in remission 19 months after the treatment, and the modified cells integrated into his body.

The case, which is the first detailed report in a major academic journal of how doctors are using Crispr in living patients, is an “important milestone” and suggests that gene editing will be “a safe technology and that the challenge now is to have it be really effective in different disease settings,” Doudna said.

The field is facing a crucial few months as researchers from the University of Pennsylvania are also preparing to release data from their own trial. “We should start to see some really interesting information about how Crispr is working in those cancer patients in the near future,” Doudna said.

Last year, a Chinese researcher stunned scientists after claiming to have created the world’s first genetically edited babies, which sparked an international backlash and calls to put a ban on using Crispr to create permanent changes in a subject’s DNA.

To contact the reporter on this story: Tatiana Darie in New York at tdarie1@bloomberg.net

To contact the editors responsible for this story: Catherine Larkin at clarkin4@bloomberg.net, Richard Richtmyer, Chris Nagi

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