This Week: Gene Editing in a Baby, 23andMe Acquisition Plans, and More

This week’s post will be a little different. There’s a lot going on, and I don’t want to do a slow drip-drip of the news over the next few weeks, so instead I’ll serve it all up in summary form today.

Gene Editing Addresses Rare Disease in a Baby

You probably saw these headlines last week: doctors at the Children’s Hospital of Philadelphia worked with scientists across the U.S. and Canada to deliver a groundbreaking treatment for an infant with a rare genetic disease. The urea-cycle disorder is fatal in many cases, with a lot of nasty symptoms and complications for those who do survive. At only six months of age, baby KJ Muldoon was treated with a novel gene-editing therapy designed to change the single mutation in his DNA causing this devastating disorder. While it’s not yet clear if the treatment cured his disorder, doctors are quite hopeful because KJ has achieved key developmental milestones that would not be expected for a baby with this disorder.

Gene editing has been a really exciting innovation in the field of genomic medicine. It began with scientists harnessing naturally occurring editing processes that take place in bacteria and applying them to other types of cells. In labs, gene editing has become almost commonplace. But its potential for use in medicine — while theoretically very promising — is a completely different challenge. For in-person use, the edit must be delivered to all relevant cells, or at least enough to tip the scales. It has to be specific, editing only the target mutation without inadvertently making changes anywhere else. And, at least for now, each treatment has to be custom-developed for each patient. That’s not just one mountain to climb, it’s a whole mountain range.

That’s why people are downright giddy about KJ Muldoon’s case. It could herald a new era in clinical gene editing, paving the way for this approach to be more feasible for babies with a range of genetic disorders.

If you’re interested in the technical details behind the gene editing approach used, Eric Topol has a great explanation. For the real nitty-gritty, check out the peer-reviewed medical publication describing the months-long process.

Incidentally, another gene editing story has been reported, this time in a teenager with a dangerous condition affecting his immune system. This case has not yet gone through peer review, so it’s too early to understand exactly how this worked or how well it performed.

Genetic Data Company 23andMe Will Be Acquired

In other recent news, biopharma company Regeneron has made a deal to acquire 23andMe, which was in bankruptcy proceedings, for $256 million. This is good news for the biomedical community: 23andMe had amassed quite a collection of genetic data that should be valuable for drug discovery programs. But for 23andMe customers, the news is more mixed. Privacy agreements for data like this rarely transfer from an acquired company to its new owner. If you have a 23andMe account and you’re concerned about how your data might be used, MIT Tech Review has a handy guide for how to delete your data.

Looking to Join the Resistance?

Worried about federal cuts to biomedical research, but not sure what you can do? This week I learned about a new volunteer-operated group called Unbreaking that aims to document what’s going on.

Here’s how they describe it: “We believe that mapping the damage done and its human costs—and the pushback and resilience work already underway—is necessary groundwork for building and retaining political agency.” They add: “In our work at Unbreaking, we’ll help orient and ground our communities in clear and rigorously cited explanations of what’s happening to our government and why it matters.”

Unbreaking is looking for volunteers — no expertise or scientific knowledge required — to help collect data and write explainers about the impact of these changes. The link above will take you to a page where you can raise your hand to get involved if you’re interested.

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