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The Readout Damian Garde

CRISPR is coming to a bookshelf near you


The relatively brief history of CRISPR genome editing features a host of eurekas, billions in venture investment, and a seemingly eternal patent dispute. Now it’s getting the long-form nonfiction treatment.

At least four books on CRISPR are in the works, ranging from an examination on the present-day ethics of genome editing to an expansive look at the decades of scientific discovery that got us here.

Of note, one of the authors is Walter Isaacson, the historian behind best-selling biographies of Albert Einstein and Steve Jobs. And the authors, while ostensibly in competition for the public’s finite CRISPR appetite, all said they’ve been trading words of encouragement rather than literary taunts. (And before you ask, the two sides of that immortal patent fight aren’t playing authorial favorites.)
 
Read more.

Who’s leading the DMD gene therapy race?


Friday’s big news in Duchenne muscular dystrophy led to a widespread conclusion: Pfizer’s gene therapy seems fine, but Sarepta Therapeutics has a better one. Now that we’re a few days removed from it all, are we sure it’s that simple?

STAT’s Matthew Herper and Adam Feuerstein had something of a salon on the topic, digging into the debate over Pfizer and Sarepta. On the one hand, Sarepta’s therapy looks optically better than Pfizer’s, but on the other, this whole debate is based on data from 10 cumulative patients.

The important answers lay ahead. Pfizer needs to figure out how to address some emerging side effects, while Sarepta needs to replicate the promising signal it saw in an earlier trial. And, perhaps most pressing, the world needs to learn more about how to measure the benefits of a one-time treatment for DMD.

Read more.

We’re going to have PCSK9 debate all over again


Monthly or bi-monthly treatments that drastically lower cholesterol haven’t exactly made a mint for their makers. But what if you could do the same job with a twice-a-year treatment?

That’s the proposition behind the Medicines Company and its inclisiran, an injected treatment that uses RNA interference, or RNAi, to shut down the production of PCSK9 and reduce cholesterol. Some time this quarter, the company will disclose data from three late-stage trials that will determine whether long-term exposure to inclisiran is safe and effective.

PCSK9 is the target of two antibody therapies from Amgen and the partnership of Sanofi and Regeneron Pharmaceuticals. Those two therapies proved to lower cholesterol and improve long-term patient outcomes, but they spent years as poster children for commercial disappointment, gaining traction only after their makers more than halved their prices to appease payers used to cheap, generic statins.

And thus the question becomes: Does the world need the Medicines Company’s every-six-months treatment? Inclisiran’s late-stage data, whenever they're available, will rekindle an old biotech debate.

Zebrafish in the clinic


Precision medicine has ushered in a new era when it comes to the treatment of certain cancers. But the potential for transformation extends into far more diseases.

In a case study published yesterday in Nature Medicine, researchers at the Children’s Hospital of Philadelphia detailed how they used zebrafish to save the life of a boy with a rare, complex lymphatic disorder. In short, they identified the mutation causing the boy’s illness; engineered the mutation into zebrafish; waited for the fish to develop a piscine version of the boy’s disease; and then tested multiple drugs on the fish — until they found one that worked.

“I think this approach definitely could be used clinically,” one medical geneticist told STAT’s Sharon Begley.

Read more.

More reads

  • You can now get your whole genome sequenced for less than an iPhone. (OneZero)
  • At Purdue Pharma, business slumps as opioid lawsuits mount. (Wall Street Journal)
  • Woodford’s flagship fund suspension is extended indefinitely. (Financial Times)
  • Senators widen probe into groups with ties to opioid makers. (STAT Plus)

Thanks for reading! Until tomorrow,

Megan

Tuesday, July 2, 2019

STAT

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