Aurora Therapeutics' first target is the rare inherited disease phenylketonuria, also known as PKU. Here at we've been writing about the gene-editing technology CRISPR since 2013, calling it the biggest biotech breakthrough of the century. Yet so far, there's been only one gene-editing drug approved. It's been used commercially on only about 40 patients, all with sickle-cell disease. It's becoming clear that the impact of CRISPR isn't as big as we all hoped. In fact, there's a pall of discouragement over the entire field--with some journalists saying the gene-editing revolution has " lost its mojo ."

Sam Berns was my friend. With the wisdom of a sage, he inspired me and many others about how to make the most of life. Afflicted with the rare disease called progeria, his body aged at a rapid rate, and he died of heart failure at just 17, a brave life cut much too short. My lab discovered the genetic cause of Sam's illness two decades ago: Just one DNA letter gone awry, a T that should have been a C in a critical gene called lamin A. The same misspelling is found in almost all of the 200 individuals around the world with progeria. This story is from the WIRED World in 2025, our annual trends briefing.

AI is used to compose music, suggests recipes and make investment decisions, but a company has designed a system that can edit human genes. California-based Profluent Bio developed a system capable of creating a range of bespoke cures for disease by developing molecules that have never existed in nature. The AI was trained on a database of 5.1 million CRISPR-associated (Cas) proteins, allowing it to create potential molecules that could be used in gene editing. The system then narrowed down the results to four million sequences, allowing it to identify the gene editor the team named OpenCRISPR-1. Experiments showed OpenCRISPR-1 performed as well as Cas proteins, but it also reduced the impact on off-target sites by 95 percent.