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The CRISPR breakthrough that can change the future of gene editing

July 2, 2021, 12:03 AM UTC

Happy Thursday, readers.

While we continue to keep a close eye on the emergence of COVID variants, and what we may have to do about it, going into the Independence Day weekend, I have DNA on my mind.

This week started off with a remarkable bit of news from the world of CRISPR gene-editing, a biotech platform about a decade in the making and glorious potential. We’re talking slicing and dicing genetic code in order to permanently cure genetic diseases or inhibit the creation of villainous proteins stemming from inherited disorders, cancer, and dozens of other maladies. And Intellia Therapeutics, co-founded by Nobel Prize winner Jennifer Doudna (one of the inventors of CRISPR technology), has apparently reached a watershed scientific moment in the space.

You see, CRISPR technology to date has largely relied on an outside-of-the-body approach. That is, you have to take biological material out of a patient, and then apply the gene editing therapy which replaces aberrant genomic code with more acceptable biological permutations, and then put it back into the patient.

That’s called an ex vivo approach. What Intellia has done with its experimental CRISPR therapy NTLA-2001 is transform the process into an in vivo approach. That means “inside the body,” and it requires directing a molecular guide to home in on the genetic offender and then partner it with a messenger RNA (mRNA, which is at the heart of the multiple COVID vaccines) to initiate the process of correcting the genetic flaw and letting the body do the heavy lifting.

The initial results in patients with transthyretin amyloidosis (ATTR), a rare degenerative disease that can lead to nerve damage and the stiffening of the heart, are impressive. Intellia says that its therapy was able to reduce the level of transthyretin, which is associated with the disease, in the liver by 87% with a high dose of the treatment. That’s a significant improvement over currently available therapeutics.

I’d also like to ask a favor of readers: We are looking for nominations for our annual Change the World list. This is one of my favorite Fortune lists as it recognizes the companies that are doing well by doing good. Not philanthropies or non-profits, but corporations that recognize the role they have to play in the world and have a grasp on how to balance social responsibility with stakeholder interests and the profit motive. Please submit any nominations you may have to this Google form, and feel free to contact us via changetheworld@fortune.com if you have any questions.

Read on for the day’s news, and see you again next Thursday.

Sy Mukherjee
sy.mukherjee@fortune.com
@the_sy_guy

DIGITAL HEALTH

Pinterest loses interest in the weight ad game. It isn't exactly a secret that social media platforms and their affiliated marketplaces play a part in normalizing what can be harmful behavior, whether that be propagating vaccine disinformation or being a vessel for marketing campaigns meant to actively shame users. Pinterest is saying, "no thanks" to the latter part, banning any advertisements that fat- or body-shame people who may be among its customer base. “This stance makes Pinterest the only major platform to prohibit all weight loss ads,” the company said in a statement. “It’s an expansion of our ad policies that have long prohibited body shaming and dangerous weight loss products or claims. We encourage others in the industry to do the same and acknowledge, once and for all, that there's no such thing as one-size-fits-all.” (Fortune)

Can Regeneron crack the genetic code to obesity? My colleague Erika Fry reports on an exciting genomic development (though not of the CRISPR variety) in the biotech world. NY-based Regeneron says it has been able to identify rare genetic mutations that may be linked to obesity and could potentially spur treatments that curb it. The science of obesity is fraught, which is what makes this discovery so interesting. There isn't quite a clear map of how, or how much, genetic factors contribute to someone's risk for obesity. Regeneron's findings could help shine a new light on this area of drug development. (Fortune)

INDICATIONS

Activist pressure continues to build for GlaxoSmithKline. British drug giant GlaxoSmithKline has had some rollercoaster years, and it appears the ride isn't over quite yet. Following aggressive restructuring of the corporate suite, deals with outside companies, spinoffs, and a reset of the drug maker's therapeutic pipeline, the pressure is still on from activist investors led by Elliott Management who are openly calling for a managerial and strategic shakeup given the company's influence and potential. One key criticism levied by Elliott? "GSK has underperformed its peers on shareholder returns, R&D investments and other key indicators," the firm wrote in an open letter following an investor update last week. "The causes of such severe underperformance stem from issues that stretch back more than a decade. GSK's biopharma business is viewed as overly bureaucratic." (FiercePharma)

THE BIG PICTURE

The Delta variant is here, but testing is tricky. The COVID Delta variant is now the greatest threat to getting back to normalcy. The highly infectious strain is making up a larger and larger share of new coronavirus cases in the U.S., and has been identified in more than 80 countries. But can conventional COVID tests detect this specific variant? In short: Sort of. Some tests do, and some tests don't. Many require more complex analysis of patient's biological sample which may have to be conducted in a lab and aren't quite as fast as rapid antigen tests which can return results within a half hour. But companies are already working on tailoring diagnostics to sniff out the Delta variant. What's most important, however, is to get vaccinated, no matter which COVID strain may be going around, since initial studies have shown that currently available shots help prevent serious illness, hospitalization, and deaths across the coronavirus spectrum. (Fortune)

REQUIRED READING

Facebook is being investigated over vaccine disinformationby Bloomberg

The Trump Organization is facing a 10-count indictmentby Nicole Goodkind

Employers have a tricky legal landscape on vaccinesby S. Mitra Kalita

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