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How Altos is spending $3 billion, Vertex braves a 'graveyard' for new drugs, & scientists near a holy grail in contraception

 

 

The Readout

Good morning, all. Damian here, live from the second day of the 2022 STAT Summit. Read on for an update on an upstart whose ambitions are rivaled only by its funding, the future of Vertex Pharmaceuticals, and a holy grail in contraceptive medicine.

Altos has $3 billion and a particular aversion to ‘longevity’

The minds behind Altos Labs, an ambitious company that raised $3 billion to fund its research, would like people to stop calling it an anti-aging biotech.

The big idea at Altos is cellular rejuvenation, CEO Hal Barron said at the 2022 STAT Summit, based on a Nobel Prize-winning technique in which researchers can introduce four different molecules into a mature cell and reverse it back into a stem cell. The idea is to make cells more resilient to stresses of injury, disease, or — but not solely — aging.

“We know at least in animal models, this is possible,” Barron said. “It’s an unbelievably big idea, it’s an unbelievably complicated idea, with lots of ways to fail. But we just thought, you know, if this works, it changes everything.”  

Read more.

Despite its Covid success, mRNA has a delivery problem

The superlative success of mRNA vaccines for Covid-19 has left no doubt that the technology can safely put the body’s machines of protein production to work. But getting it to specific spots in the body remains an unsurmounted challenge.

As STAT’s Jonathan Wosen reports, turning mRNA into a vaccine required just a shot into the muscle of the arm, but for other uses — such as targeting a discrete set of tumor cells in a part of the body that’s hard to access — delivery won’t be so straightforward.

To many companies, the solution lies in making molecular tweaks to lipid nanoparticles, the fatty vehicles used to ferry molecules to their targets. Others are setting their sites on mRNA, engineering it to only produce protein in certain cells, regardless of where it lands.

Read more.

Vertex braves a ‘graveyard of drug discovery’

Vertex Pharmaceuticals went from the verge of corporate oblivion to the zenith of biotech thanks to a series of powerful medicines for cystic fibrosis. The plan now is to replicate that success by taking a similar approach to seemingly disparate diseases — including one that has been “a graveyard of drug discovery,” as CEO Reshma Kewalramani put it.

“Our goal for ourselves was to do what we have already done with cystic fibrosis again, and again, and again,” CEO Reshma Kewalramani said at the 2022 STAT Summit. 

That includes developing a novel treatment for pain — the aforementioned scientific graveyard — and potentially curative medicines for sickle-cell disease and Type 1 diabetes.

Read more.

After decades of false starts, scientists are closer than ever to male birth control

It’s been described as five years away since the 1970s, but after a few false starts and one high-profile setback, scientists say male birth control is closer than ever to becoming a medicinal reality.

As Eleanor Cummins reports for STAT, researchers are optimistic about early but promising data from an investigational contraceptive gel, and men around the world seem to be getting more receptive to the idea of such a treatment. 

“I am not always the cup half-full person,” said Diana Blithe, chief of the NIH Contraceptive Development Program. But in light of emerging data, “I am way half-full — I am three-quarters full. This is really working so much better than we expected.”

Read more.

More reads

  • ‘I pushed back’: Fauci on how his response to Trump on Covid turned him into ‘public enemy No. 1,’ STAT
  • A rare biotech IPO lands on Nasdaq, Endpoints
  • Perfection is too high a bar for CRISPR treatments, says STAT Biomedical Innovation Award winner David Liu, STAT

Thanks for reading! Until tomorrow,

@damiangarde
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Wednesday, November 16, 2022

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