A better, faster way to deliver CRISPR, if it works

CRISPR is very good at editing DNA; it’s not terribly precise when it comes to hitting one organ versus another.

And so one group of researchers has an idea: Just as a magnetic wand can put metallic hair on the face of Wooly Willy, could magnetic nanoparticles do the same for genome editing?

As STAT’s Sharon Begley reports, the answer appears to be yes, in mice. Scientists at Rice University took a standard CRISPR cocktail and added in some iron oxide nanoparticles. They injected it into mice and flopped them onto some magnets, and the result was a better, faster CRISPR’ing that focused on the liver and spared the heart, lung, kidney, and other vital organs.

Researchers around the country are now kicking the tires on nanoparticle-based delivery technologies that could one day ferry CRISPR molecules with precision.

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The most controversial biotech stock you’ve never heard of

Jena, Germany, has been home to Johann Wolfgang Goethe, Friedrich Nietzsche, and Karl Marx. Now it’s the site of a little-known but hotly debated biotech company whose fate could swing hundreds of millions of dollars on Wall Street.

InflaRx, headquartered in Jena, is at work on drug called IFX-1, meant to treat the skin disease hidradenitis suppurativa. And that, STAT’s Adam Feuerstein reports, is the only thing on which bulls and bears agree.

InflaRx believes IFX-1 can work, pointing to data from a 12-patient trial in which the response exceeded 80%. But investors betting against the stock say those numbers are inflated or even impossible.

Here’s everything you need to know.

What it’s like to work for Elizabeth Holmes

Erika Cheung was a 22-year-old biochemist when she met Elizabeth Holmes, inspired by the Theranos CEO’s vision of a world in which health care was affordable and blood testing was universally available. Within nine months, she was blowing the whistle on her employer.

In an interview with STAT, Cheung explained how she went from a “starry-eyed” admirer of Holmes’s entrepreneurial spirit to an agent of Theranos’s eventual demise. She also weighs in on how long her former boss should spend in jail, why the Theranos story has captivated the public, and the biggest outstanding question: Was Elizabeth Holmes a fraud from the very start?

“At lot of people disagree with me but I don’t think that was the case,” Cheung said. “I think she went in, at least initially, with good intentions. But she let her ego get in the way. She was more focused on being the next Steve Jobs of health care.”

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Treating SMA is about to get more interesting

The 2016 approval of Biogen’s Spinraza was a watershed event for patients with spinal muscular atrophy, a muscle-wasting disease for which there were previously no treatments. Three years later, those patients see a future in which they might be able to choose between Spinraza, a gene therapy, and a daily pill — or combine all three.

And we want to talk about it. Later this month, we’re hosting a call with an SMA expert who will answer all your questions about how the future of treatment might shake out.

By then, Novartis will have presented new data on its gene therapy, and Roche will have disclosed some details on its oral treatment. If both work, and Spinraza’s demonstrated benefits hold up, how will doctors decide which patients get which treatment?

If you’d like to find out, sign up here for STAT's premium service, STAT Expert Advantage.

A better, faster way to deliver CRISPR, if it works

The most controversial biotech stock you’ve never heard of

In-depth biopharma, business, and policy analysis at your fingertips

What it’s like to work for Elizabeth Holmes

Treating SMA is about to get more interesting

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