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JPM Day 3 had a cautiously optimistic outlook

In honor of JPM Week, you’re reading a special edition of our biotech newsletter, The Readout. To stay on top of the latest scoops and live reporting from the San Francisco conference, join our newsletter list.

We’ve reached the Wednesday of JPM Week, when gaits get sluggish, voices go hoarse, and early-morning meetings run an increasing risk of cancelation. Persevere. And while you’re at it, don’t miss STAT’s multimedia coverage of the conference, featuring decidedly lively interviews, commentary, and on-the-ground reportage.

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The consensus is that the dark skies over biopharma are clearing

Maybe it’s the abundance of sunlight that filled San Francisco this week. More likely, it’s the surge of deals that came in December. But the sentiment around Union Square has been markedly more positive this year.

“I’m feeling a lot of good energy, honestly,” said Mira Chaurushiya, senior partner at VC Westlake Village BioPartners. “It’s not only what happened the last few weeks of the year, but what people are projecting moving forward.”

Now, it’s not all rosy skies ahead. Biotechs are continuing to lay off staff and make painful strategic shifts to conserve cash. But the worst may be over. During the course of this week, we bumped into people like Celsius Therapeutics CEO Tariq Kassum, who made the difficult decision last August to lay off 75% of his staff in order to keep the company afloat long enough to run a clinical trial for its first drug candidate. The early data is in, and it looks like Kassum made the right move, he said.

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DNA sequencing doesn’t line up

Nvidia grabbed headlines by talking about how artificial intelligence would create an inflection point for drug development. But another technology that was supposed to do that, DNA sequencing, didn’t do so well at this year’s J.P. Morgan.

The new CEO of Illumina, Jacob Thaysen, basically gave investors what they want after the long, losing battle the company made to own cancer diagnostics firm Grail: a return to focusing on the core DNA sequencing business and a promise to move quickly on spinning off or selling Grail, which the company bought, disastrously, over regulators’ objections. Unfortunately, that was about as boring as it sounds: a company that had always had a healthy dose of wanting to change the world sounded a lot like a maker of, well, laboratory tools.

Element Biosciences, an upstart trying to move into the market, announced that it had booked $25 million in sales in 2023 and launched new machines that can also sequence RNA and proteins. Oxford Nanopore — probably currently the most exciting company in the group for its ability to sequence DNA on thumb-drive-like machines and to get much longer readouts of DNA code than the tiny bits of sequence other technologies must stitch together — announced earnings of £169 million, about £8 million less than investors expected. The company’s shares fell 14% on the London stock exchange.

Roivant’s drug hunt

Roivant Sciences CEO Matt Gline has been spending his JPM Week meeting with investors in an upper-floor hotel suite. The more interesting action is taking place in another hotel room floors below, where Roivant’s business development team, flush with $7 billion in cash largely from the recent Roche deal, is hunting for its next drug asset.

In an interview with STAT, Gline said Roivant’s licensing team has been meeting with pharma companies about existing drugs in their pipelines that may no longer fit with spending or research priorities, but which in Roivant’s hands could be developed further to create mutual value.

Roivant is not narrowing its search to any specific type of drug or disease area, said Gline, although it’s likely to be something in the middle- to late-stage of development. Cancer and gene therapy are not in Roivant’s wheelhouse, and while Gline would love to find the next great oral GLP-1 for weight loss, finding a hidden gem like that isn’t likely, he said.

“But that still leaves us with a whole bunch of potential drugs to consider,” he said. “The right thing for us to do is be patient.”

Think of genome editing as ‘a molecular surgical procedure’

That’s the advice of Verve Therapeutics CEO Sekar Kathiresan, whose company is at work on an audacious plot to move genome editing from the world of rare disease and tackle the world’s most common cause of death.

The plan is to use base editing, a successor to CRISPR, to silence a gene called PCSK9, which encodes for a protein involved in the body’s clearance of bad cholesterol. Shutting off PCSK9 should dramatically reduce cholesterol, which in turn would reduce the risk of heart attack. Verve’s multiyear plan to bring genome editing to the mainstream starts with treating a genetic form of high cholesterol, followed by studies in more common forms of heart disease, and, finally, proving that its one-time treatment should be a widely used preventive medicine.

But how do you turn genome editing, a technology associated with small patient populations and multimillion-dollar price tags, into something globally accessible?

“I’m not committing to anything right now, but you could imagine a world where this is priced much more like other one-time procedures in cardiovascular medicine, like a bypass surgery or a stent, other procedures that are permanent and one time and intended for lifelong benefit,” Kathiresan said. “That’s kind of how I see our medicine ultimately: Less a drug and more like a molecular surgical procedure.”

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