This year has been a monumental year for Crispr, the molecular tool scientists use to edit genetic material. In November this year, the UK approved the first treatment using Crispr gene editing, giving patients with sickle cell disease a new chance to receive a one-off treatment to prevent the onset of severe pain. The U.S. Food and Drug Administration is expected to make a decision on the treatment this week. What once seemed like a moonlight is already changing people’s lives.
However, it is still a rare treatment at this time. “It’s expensive,” Jennifer Doudna, a pioneering biochemist who won a Nobel Prize in 2020 for her work on Crispr, told WIRED’s Emily Marin at the LiveWIRED conference in San Francisco this week. The treatment is expected to cost more than $1 million per patient, potentially leaving many people who need it most without treatment.
It’s also a complex process. Patients harvest stem cells from their bodies, edit them in a laboratory, and then turn them back into their original cells. Doudna is optimistic about a future where Crispr-based treatments are much less invasive than they are today. “Maybe someday I’ll take a pill,” she says. “It sounds a little fanciful now, but I think it’s very achievable.”
In 2014, Doudna Institute for Innovative Genomics Applying Chrispr technology to healthcare problems. Doudna hopes IGI’s research will help make these technologies more affordable and accessible. She is also very interested in how Crispr can be used to fine-tune the microbiome.