| In May, baby KJ Muldoon made history as the first person to get a custom-made treatment using the gene-editing tool Crispr. Now a push is underway to do the same thing for eight more kids. Fyodor Urnov, a researcher at University of California, Berkeley, is leading a new effort, the Center for Pediatric Crispr Cures, which said this week it will be funded by a $20 million grant from the Chan Zuckerberg Initiative, the philanthropic arm of Priscilla Chan and her husband, Facebook founder Mark Zuckerberg. The goal is to build on the success of baby KJ and make personalized Crispr treatments available to more kids with severe and extremely rare genetic disorders. Crispr is a Nobel-prize-winning technology that lets you edit DNA with the same ease as spell-checking a Word document. It holds the promise of treating, or even curing, many debilitating and deadly conditions caused by genetic flaws, from sickle cell disease to high cholesterol. The center’s efforts will be a collaboration between UC Berkeley, which will design and test the treatments, Danaher Corp., which will make the components, and the University of California, San Francisco, which will treat the patients. It will focus on two types of diseases: metabolic disorders, like one in which the body can’t break down certain proteins and fats, and inborn errors of immunity, which are genetic flaws that impair the immune system and leave kids susceptible to infections. Urnov, who helped develop KJ’s treatment, often gets emails from desperate parents who send photos of their kids with rare genetic disorders, asking if there’s a gene-editing treatment for them. Many can’t be treated using the existing technology. But even if a therapy was possible, there is little interest from companies. It’s expensive to make and typically aren’t reused, making it hard to recoup the cost. Like KJ, the eight children who will be treated by the new center will need treatments customized specifically for their unique genetic mutation. Normally, that entire process would cost around $15 million per patient and take about three years. For companies, that math doesn’t add up. And for a child who may have only a few months to live, it’s too long. Urnov believes the center can shorten the process to just four or five months and dramatically reduce the costs by not spending time on redundant pre-clinical studies. Instead, the center can simply tweak a component of Crispr, called a guide RNA, to address each child’s unique genetic flaw. He likened it to a pizzeria “where changing the topping does not require recertifying the oven or the person throwing the pizza disk.” Ultimately, Urnov wants to create a Crispr “cookbook” — a standardized process that anyone can use to make a personalized gene-editing treatment. If the idea is going to work for more kids, companies — and not universities — will need to take the lead, he says. “My hope is that the for-profit sector takes our cookbook and runs with it,” Urnov says. — Gerry Smith | 
