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The Readout Damian Garde & Meghana Keshavan

Beam is putting CRISPR inside CRISPR

More or less anyway. The company’s approach to potentially curing sickle cell disease involves embedding a DNA-editing enzyme inside a classic CRISPR-Cas9 molecule, creating a Russian nesting doll of genome editing that could clear a key hurdle.

As STAT’s Megan Molteni reports, it all stems from what’s called an “activity window.” Beam Therapeutics’ base editors can make precise, letter-by-letter changes to DNA, but they can only bind to certain spots on the genome. The sickle cell hemoglobin gene proved to be outside the activity window of traditional base editing. But by inserting the base-editing enzyme into a regular CRISPR molecule, Beam’s scientists were able to efficiently edit cells taken from a sickle cell patient.

The editor-within-an-editor approach is somewhere between “gutsy” and “crazy,” said Fyodor Urnov, scientific director at the University of California, Berkeley’s Innovative Genomics Institute. But that doesn’t mean it won’t work. Urnov said Beam’s idea looks to be on its way toward first-in-human trials.

Read more.

Vertex thinks editing sickle cell is worth $9 billion

Vertex Pharmaceuticals, which is working with CRISPR Therapeutics on a slightly less cutting-edge approach to curing sickle cell disease, is paying $900 million for a larger stake in the in-development therapy, implying the treatment’s value is worth more than $9 billion.

Under the deal, disclosed yesterday, Vertex is trading the cash in exchange for another 10% share of the treatment, called CTX001. The two companies were operating under a 50-50 split, meaning the latest agreement gives Vertex a 60% stake. CTX001, which involves using CRISPR to edit stem cells outside the body, is the most advanced genome-editing sickle cell disease, with a Phase 1/2 trial expected to complete enrollment this year.

To Baird analyst Brian Skorney, the deal — and its implied valuation for CTX001 — looks a little rich considering the amount of potential competition in the space, from both genome-editing technologies and traditional gene therapies. But there is an upside for investors, Skorney wrote in a note to clients: After paying so much for a bigger cut of CTX001, Vertex seems that much less likely to throw billions of dollars toward buying CRISPR Therapeutics outright.

As goes J&J

Because Johnson & Johnson is the largest health care company in the history of the world, its financial fortunes tend to be a bellwether for the industry at large. And parsing J&J’s positive results from the last quarter, released yesterday, analysts see hope that the sector is gradually crawling out of a pandemic-related slump.

J&J beat Wall Street’s revenue expectations for both its pharmaceutical and medical devices businesses, which Cowen analyst Joshua Jennings expects will improve investor sentiment across the board. The broad readthrough is that patients are again seeing their doctors, filling their prescriptions, and scheduling elective procedures, all of which is good news for the industry’s bottom line.

All this comes as J&J’s Covid-19 vaccine remains in limbo due to a small number of severe clotting incidents now under investigation by global regulators. The situation has massive implications for the global vaccination effort, but, because J&J is selling its product on a nonprofit basis during the pandemic, it wasn’t terribly material to the discussion of corporate earnings.

Shareholders want Pfizer to reconsider its political spending

After January’s riot at the Capitol, Pfizer joined much of corporate America in rethinking its donations to political campaigns. One group of shareholders wants the company to go further, demanding Pfizer take a harder look at where its money goes in Washington.

As STAT’s Ed Silverman reports, a philanthropic organization called the Tara Health Foundation has filed a shareholder proposal that would require Pfizer to publish an annual report analyzing its donations, with a particular focus on those that might jeopardize the company’s reputation. The organization’s examples include Pfizer’s past donations to attorneys general who sued to repeal the Affordable Care Act and to state legislators who have voted to restrict abortion rights.

Pfizer’s board argued that the company already explains its political giving in an annual report, making the proposal “an unnecessary expenditure of corporate resources [that] would not be useful to shareholders.” 

The proposal is the latest in a series of efforts by activists to use the staid structure of shareholder votes to pressure major drug companies toward corporate transparency, following a move to get Eli Lilly to disclose more about how it punishes executives for misconduct.

Read more.

More reads

  • The billionaire scientist behind the Pfizer-BioNTech vaccine has not sold a single share of his booming stock. (Forbes)
  • Biotech funding tops the charts again. (LifeSciVC)
  • Top Senate Republican holds up Biden’s CMS nominee over Texas Medicaid waiver. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, April 21, 2021

STAT

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