The Readout Damian Garde

Are we underestimating biology?

What's the deal with Cory Booker and pharma? And who owns the scientific means of production?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, we dissect a pair of disappointing clinical results from newfangled ideas in biotech. Then, blogger Derek Lowe joins us to talk about his disagreement with Rep. Alexandria Ocasio-Cortez on the relationship between publicly funded research and industry-marketed drugs, a conversation that touches on science, policy, and sexism. Finally, STAT Washington correspondent Lev Facher calls in to discuss the slate of candidates lined up to challenge President Trump in 2020 and the ideas they bring to the world of drug pricing.

You can listen to the episode here. To listen to future episodes, be sure to sign up on iTunes, Stitcher, Google Play, or wherever you get your podcasts.

Genome editing makes a typo

CRISPR may get all the headlines, but the first human trial involving genome editing actually used a less-heralded technology called zinc fingers. That less-heralded technology finally made headlines yesterday, but, unfortunately, they were negative.

The genome-editing trial from Sangamo Therapeutics turned up disappointing results in a rare, inherited disease called MPS II, also known as Hunter syndrome. The therapy showed no activity in five treated patients and offered only a glimmer of promise in a sixth, results that sent Sangamo’s stock price down more than 30 percent.

So what does this mean for that _other_ genome editing technology? The three big CRISPR companies — Intellia Therapeutics, Editas Medicine, and CRISPR Therapeutics — are expected to generate some human data of their own this year. Here’s a question for you: Do Sangamo’s results change your expectations for the whole genome-editing field?

No. There’s a reason CRISPR, not zinc fingers, gets so much attention.
Yes. The hype has outpaced the risks of early science.

A father-son biotech saga

Remember Neurochem? STAT's Adam Feuerstein does. A dozen years ago, Neurochem was running a Phase 3 trial on a treatment for Alzheimer’s disease that CEO Francesco Bellini was convinced would succeed. But, like most drugs with similar aims, it did not, and after that failure, Neurochem faded from view.

But it didn't die. The company has since become Bellus Health, and while its CEO has since retired, there's another Bellini in his place, a son named Roberto. Bellus has ditched Alzheimer's in favor of chronic cough and is now plotting to start a mid-stage trial later this year.

Read more.

There will be biotech IPOs in 2019 after all

A painful downturn gave way to widespread worries and a government shutdown, but it finally happened: A biotech company went public in 2019.

Alector, developing immunological therapies for Alzheimer’s disease, priced in the middle of its range and raised $176 million in the process. The company’s stock traded about 5 percent on its first day out. That might have been disappointing, say, last summer, but when you consider that the biotech IPO immediately preceding Alector’s collapsed out the gate, maybe it’s not such a bad return.

Hours later, Gossamer Bio priced its offering according to plan and will start trading today. In the wings are Harpoon Therapeutics, TCR2 Therapeutics, and Stealth Biotherapeutics, each hoping that Alector’s feat means biotech can return to normalcy in 2019 after an icy winter.

More reads

  • CRISPR babies: a view from the center of the storm. (Development)
  • Novum, best known for astronomical price hikes, files for bankruptcy. (FiercePharma)
  • Scientists take a page from tortoises to design a new type of drug delivery. (STAT Plus)

Thanks for reading! Until next week,


Friday, February 8, 2019


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