Tuesday, December 12, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

Who was biopharma's worst CEO in 2017?

Bad data, bad deals, and losing streaks — this year's crop of worst CEO nominees has it all.

In part two of Adam Feuerstein's proud annual tradition, we ask you to vote for the biopharma executive who made the most massive mess of 2017, viewed through the eyes of the hapless shareholders who watched their money evaporate after putting trust in one of these men (they're all men).

Adam will announce the results next week. In the meantime, be sure to vote in the sunnier competition for 2017's best CEO.

Gene editing needs that personal touch

Looks as though CRISPR could work differently from person to person, according to a new PNAS paper. As this technology proceeds to the clinic, we're learning that a technique that might work in one patient could have dangerous off-target effects in another — thanks, of course, to the multivariate differences in each human’s (or rat’s, or sea anemone’s) genome. 

The CRISPR tool is designed to zero in on a specific stretch of DNA. But when that target sequence varies from the CRISPR template, even by a single base pair, efficacy can be compromised, the study says. Treatment could fail. 

So investigators suggest that in a clinical setting, each CRISPR-based therapy should be tailored to the individual — upping the ante on this whole precision medicine game a notch or two or 12.

Heading into ASH Tuesday

Some interesting developments at the American Society of Hematology meeting, as we enter the annual confab's final day. Among them: 

  • Spark Therapeutics offered a surprising disappointment yesterday, delivering less-than-impressive data on its hemophilia A gene therapy program. BioMarin seems to have pulled ahead in the hemophilia game. 
  • A Novartis study deemed Novartis's $475,000 CAR-T product cost-effective
  • This happened.
Of course, you know much of this already if you've been reading Adam Feuerstein's ASH newsletter, archived here

New York wants to be a biotech hub, Vol. XIV

Today in the seemingly endless narrative about New York's biotech inferiority complex, a life sciences-focused coworking space is opening at NYU Langone Health, offering office and lab space at startup-friendly rates.

The idea is to tackle a problem that has long kept New York on the fringes of the biotech conversation: Rent is too expensive. The new space, called BioLabs New York, is in the vein of LabCentral, Cambridge Innovation Center, and the many other startup accelerators that have made greater Boston and the Bay Area into biotech's most fertile loci.

If this sounds familiar, it's because New York's state and local governments — with the help of industry — have been banging away at the issue for years, standing up startup factories in hopes of capturing the Cambridge, Mass., magic. And if you were curious, biotech's geographical incumbents don't feel threatened.

More reads

  • Can GSK’s new R&D strategy resuscitate the worst performer in Big Pharma? (Endpoints)
  • No, Google's AI program can't build your genome sequence. (Forbes)
  • An oral history of Pfizer's little blue pill. (Bloomberg)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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