The Readout Damian Garde & Meghana Keshavan

A lottery with lives hanging in the balance

Whose child might live, and whose might not? In the case of children with a rare disease, a lottery devised by Novartis may be a factor.   

The lottery will randomly select children with spinal muscular atrophy, or SMA, to receive the company's gene therapy, Zolgensma, for free. The company aims to give away 100 doses of the extraordinarily expensive in countries where it is not yet available.

The ethics of this situation are fraught. On one hand, more kids do get access to the medication, when otherwise they might not. But framing the recipients as “lucky winners” is insensitive to everyone else.

“We applied for it because we desperately want this drug for our daughter, but you’re putting your daughter’s well-being and longevity in the hands of a lottery,” one parent told STAT’s Andrew Joseph. “I guess it’s the fairest way to decide who gets the drug and who doesn’t, but yeah, it’s not a great feeling.”

Read more.

Is peer review underrated?

Can biotech stop an outbreak? And would you mind giving us a call?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, STAT’s Sharon Begley joins us to explain how early access to scientific papers has been crucial — and occasionally problematic — during the novel coronavirus outbreak. Then, we discuss the drug industry’s attempts to find a treatment for the virus. Later, our colleague Matthew Herper joins us for a thought experiment: If biotech had a Mount Rushmore, who would be on it?

Also: We’re coming up on the 100th episode of this podcast, and we’re using the occasion to actualize our long-held dream of running a call-in show. Dial us at 617-517-6130 and leave a message with any question you’d like us to answer on the podcast, and you may just hear it on air in the coming weeks.

Listen here.

Beam Therapeutics has a $180M IPO

Cambridge CRISPRing outfit Beam Therapeutics just went public, hauling in a rather hefty sum. Starting Monday, it’ll trade under the ticker symbol BEAM.

The company, which was founded in 2017, uses the relatively new technique (isn’t this all quite new?) of base editing. Unlike the standard CRISPR analogy of scissors, base editing works more like a pencil, the company says — erasing and rewriting one letter in the gene.

Beam said that if things go smoothly, it could work with the FDA as early as next year to kick off clinical trials.

An embassy's Dutch drug war 

The U.S. is chastising the Dutch government, arguing that its policies undermine the patent rights of American drug makers. Specifically, the U.S. embassy in the Netherlands issued a scathing missive, saying Dutch officials were wrong to plan on expanding compulsory licensing and pharmaceutical compounding, STAT’s Ed Silverman writes.

This would “send a clear message” to companies that patents in the Netherlands “can be undermined or circumvented for short-term financial benefits,” the ambassador claims. 

Consumer advocates aren’t backing the embassy, however — saying, among other things, that it “gives the impression of cheap copies of patented medicines flooding the market.” 

Read more.

More reads

  • After cancer took his mother, James Allison taught our immune systems how to fight it. (Time)
  • GenapSys raises $75M to bring its tabletop DNA sequencer to Asia amid coronavirus outbreak. (FierceBiotech)
  • Scientists reveal the most extensive genetic map of cancers ever made. (The Economist)

Thanks for reading! More next week,


Friday, February 7, 2020


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