“As a researcher, I can’t contain my own enthusiasm. It is quite remarkable,” said De Vivo, a professor at Harvard Medical School and the Harvard T.H. Chan School of Public Health. “And it’s mostly because the entire world came together to work on this technology. … It is truly a multidisciplinary discovery and implementation in the clinical setting.”

Speakers discussed CRISPR science, which advances by some 6,000 new academic papers each year, a pace too fast for those interested in reading to keep up with, according to Sylvain Moineau, Canada Research Chair in Bacteriophages for the Université Laval in Quebec.

Though CRISPR’s potential to repair DNA-based conditions in human patients has grabbed headlines, Moineau said CRISPR has already had a major impact in the lab, one profound enough that he would judge the technology a success even if it is never used directly for human therapeutics.

“It’s a tremendous, tremendous research tool. To me, it’s already a success,” Moineau said.

While clinical applications are pending, the tool has the potential to impact agriculture even more rapidly, Sylvain said. He predicted a new generation of genetically modified plants, which may play important roles in feeding the globe’s rapidly rising human population at a time of increasing environmental stress.

The event also examined potential hurdles to the use of CRISPR-based therapies, such as the expense of bringing them to market, the temptation to take unethical shortcuts, and the difficulty of ensuring access to treatment to rich and poor alike.

Jonathan Kimmelman, James McGill Professor and director of the Biomedical Ethics Unit at McGill University, said despite CRISPR technology’s promise, its actual clinical impact when all is said and done may be low.

That’s because safety trials will inevitably slow progress and related science won’t stand still as trials drag on, Kimmelman said. The improved conventional treatments that result will erode the potential patient base for gene therapy. Safety trials will narrow the population further, ruling out use in children or other vulnerable groups, for example. Finally, practical concerns — cost and logistics — may rule out large populations in developing nations. In the end, he said, such “transformative” technology may indeed have an important impact, but it will likely be on a much narrower population than originally envisioned.

Panelists discussed the 2018 case of a Chinese scientist who used CRISPR to make heritable changes believed to protect against HIV to a set of twins. The controversial work highlights the importance of adopting ethical and regulatory guidelines around the world, panelists said.

As treatment options and ethical considerations are discussed, one critical population to involve is the disease community, according to Vence Bonham Jr., senior adviser to the director on genomics and health disparities at the National Human Genome Research Institute. Bonham ran focus groups of people with sickle cell disease in 2017, before the beginning of a gene therapy trial for that condition. Bonham found that the patient community was more willing than the general population to consider gene therapy, but nonetheless cautious about making changes that could be passed to future generations. Bonham quoted one participant: “Sometimes you are playing with things that should not be played with.”