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The Readout Damian Garde

Attn biotech: This man wants to be CRISPR’d

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Malakkar Vohryzek believes genome editing might cure his untreatable — and undiagnosable — condition. (DANIA MAXWELL/STAT)

Malakkar Vohryzek has a problem: His skin is ultra-sensitive to the sun, such that a daytime stroll can leave him with irritating moles he believes could grow into cancer. And he’s found what could be a solution: A genetic tweak that, in nature, seems to protect cells from harmful radiation. All he needs is someone to CRISPR him and find out.

As STAT’s Sharon Begley reports, Vohryzek has spent the past few months emailing scientists, biotech companies, and biohackers with pleas for help. He wants someone to edit his DNA, and he’s willing to sign away any right to redress if things go awry.

It’s unclear whether what Vohryzek has in mind is even possible, let alone advisable, but bioethicists say his story is unlikely to be the last of its kind. There are scores of untreatable (and, in Vohryzek’s case, undiagnosable) diseases. Coupled with the promise and hype around CRISPR, it seems inevitable that patients will look to unproven science for hope.

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The AveXis situation got that much spicier


Last we heard from AveXis, the Novartis division behind the summer’s data-falsification scandal, the company said it dismissed two top scientists in connection with the issue. Now, in a lengthy letter to the FDA, AveXis said those two scientists, who are brothers, “either manipulated or pressured laboratory personnel to manipulate data” and then refused to cooperate with an internal investigation.

As STAT’s Ed Silverman reports, the latest disclosure sheds light on an increasingly messy situation. The FDA has made quite clear its displeasure with Novartis’s decision to wait more than three months before reporting the data manipulation, and the company’s resulting explanation seems to have escalated its dispute with the departed AveXis scientists, Brian and Allan Kaspar.

Brian Kaspar has denied any allegations of wrongdoing, and his attorney said yesterday that he cooperated with Novartis’s investigation. “AveXis shamelessly attempts to blame others for its own disclosure decisions to the FDA,” the attorney said.

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Bayer’s plot to evolve beyond ‘the aspirin company’ in the eyes of Americans


The German drug giant Bayer spends more than $3 billion a year trying to invent new medicines, giving it an enviable reputation abroad. But when research chief Dr. Joerg Moeller goes through customs in the U.S., mentioning his employer brings a predictable response: “Ah, the aspirin company!”

Moeller stopped by STAT to explain how the storied company is working to boost its profile, an effort that includes investing in immuno-oncology, researching new cardiovascular therapies, and diving into novel approaches to cancer, all in hopes of finding treatments that might move the needle.

“What is meaningful is not decided by us,” Moeller said. “It’s decided by payers, patients, and prescribers. You may actually get a drug approved, only to find that you don’t actually get any prescriptions.”

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It turns out IPO dollar amounts don't predict much


The difference between $60 million and $80 million is about enough to buy the Magna Carta. But when it comes to predicting whether a biotech IPO will flourish, it turns out to be pretty negligible.

EP Vantage looked at nearly 250 biotech IPOs over five years and found that the amount of money raised had almost no correlation with the long-term success of the company. Over time, the dollar figures have risen, and so too the valuations, but the spread of winners and losers has been pretty consistent.

What’s particularly interesting is that EP Vantage’s findings also don’t support the emerging idea that companies with high valuations make for mediocre IPOs. Ex-unicorns Moderna Therapeutics and Rubius Therapeutics have seen their share prices slump after going public, but the likes of Allogene, Denali Therapeutics, and Orchard Therapeutics are doing just fine.

More reads

  • Trump’s trade war is hurting companies like mine — and the biotechnology industry. (STAT)
  • Facebook to spend $1 billion on brain-control start-up CTRL-labs. (Financial Times)
  • A big biotech bet hiding in plain sight. (Wall Street Journal)
  • FTC issues warning on ads used to file lawsuits over drug side effects. (STAT Plus)

Thanks for reading! Until tomorrow,

Wednesday, September 25, 2019

STAT

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