It’s been a monumental year for Crispr, the molecular tool scientists use to edit genetic material. This November, the United Kingdom authorized the first medical treatment using Crispr gene editing, giving people with sickle cell disease new opportunities to receive a one-time therapy to prevent episodes of terrible pain. This week, the US Food and Drug Administration is poised to make a decision about the therapy. What was once seen as a moonshot is already changing lives.
Right now, though, it’s still a rarefied treatment. “It’s expensive,” Jennifer Doudna, the pioneering biochemist who won a Nobel Prize in 2020 for her work on Crispr, told WIRED’s Emily Mullin at the LiveWIRED conference this week in San Francisco. The therapy is expected to be priced at over a million dollars a patient, which could make it inaccessible to many of the people who need it most.
It’s also a complicated process. Patients have stem cells taken from their bodies, edited in laboratory settings, and then put back in. Doudna is optimistic for a future where Crispr-based treatments are far less invasive than they are now. “Maybe even a pill at some point,” she says. “Today that sounds a little bit fantastical, but I think it’s very achievable.”
In 2014, Doudna founded the Innovative Genomics Institute to apply Crispr technology to health care questions. Doudna hopes that the IGI’s research can also help make these technologies more affordable and accessible; she’s also very interested in how Crispr might be used to fine-tune the microbiome.
Although Crispr-related medical breakthroughs are currently attracting fervent attention, Doudna suspects that the technology will break through on a mass scale outside of the health care world. “I think many of us will experience Crispr in the agricultural world before we experience it clinically,” she says. “By the food we eat, and the environmental impact.”
The IGI has expanded its mission to include agricultural research, and Doudna is especially excited about an ongoing project her team is working on in collaboration with researchers at the University of California, Davis to cut down the amount of methane cattle produce. In other words: It’s a project to make cow burps and farts pollute the air less. Not necessarily the most glamorous research, but it could prove revolutionary. “Being able to reduce or eliminate methane production in cattle would have an enormous impact on greenhouse gas production,” Doudna says. Ideally, researchers might develop a simple delivery system, like a probiotic drink, that could alter the cows’ methane production.
As she continues her research, Doudna is appreciative of moments when she can see how the work is already making a difference. “The reality for me came home when I met Victoria Gray,” she says. Gray, the first patient in the US to receive Crispr therapy for sickle cell disease, used to suffer from debilitating, chronic pain and fatigue because of her illness. Since she got the treatment, Gray has been able to enroll in business school and start a clothing company, pursuits she wasn’t well enough to do in the past. Doudna is heartened by how Gray’s case demonstrates the real-world impact of her research: “It completely transformed her life.”
