The next gene editing patent battle, a Bell Labs for biology, & FDA’S new head of neuroscience

Wait, new research on protein design powered by artificial intelligence was written by researchers at Salesforce?

March 1, 2023 Check out STAT+

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By Elizabeth Cooney Reporter, Morning Rounds Writer

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science There's a new battle brewing over next-generation gene-editing tools Maria Fabrizio for STAT Remember the patent battle over who found CRISPR first? In one corner were UC Berkeley's Jennifer Doudna and Max Planck's Emmanuelle Charpentier, and in the other, the Broad Institute's Feng Zhang. The Broad eventually prevailed in court, but the two women won the 2020 Nobel Prize for their genome-editing discovery. "Take that, U.S. legal system," Sharon Begley memorably wrote. Now a new CRISPR battle is brewing, STAT's Jason Mast, Allison DeAngelis, and Megan Molteni report, but it won't be waged between ivory towers. At issue is the next generation of gene editing, known as prime editing (or sometimes gene writing). This patent fight could become a free-for-all among multiple companies and labs, a STAT analysis of publicly available information and, in one case, a company's confidential slide deck, reveals. Biotech IP expert Jacob Sherkow of the University of Illinois summed it up: "Mov[e] fast, break things, worry about the patent licenses later." Read more. biotech What's ahead for the new head of FDA's neuroscience office After Billy Dunn announced his retirement on Monday from the FDA, where he was chief of neurology drugs, all eyes are turning to Teresa Buracchio, a physician and 10-year veteran of the FDA who was named acting director of the Office of Neuroscience the same day. While Dunn had built a reputation as a hard-nosed negotiator but later shepherded controversial medicines to approval under the guise of FDA "flexibility," Burrachio is largely unknown. She's been involved in some hot-button approval decisions, including the clearance last year of Amylyx Pharmaceuticals' treatment for ALS. Now her office faces several critical approval decisions in the coming months. That includes: Overseeing final approval for the Alzheimer's drug Leqembi, granted accelerated approval in January A decision on a new treatment for Rett syndrome developed by Acadia Pharmaceuticals Ongoing reviews of a new depression treatment from Sage Therapeutics  STAT's Adam Feuerstein and John Wilkerson have more. research Fewer women and Black scientists are 'Super PIs' STAT's Usha Lee McFarling has this report: Women and Black scientists are less likely than white men to be NIH "Super PIs" — elite NIH principal investigators who have three or more funded research grants in a given year, according to a new analysis published in JAMA Network Open. With billions in NIH funding increasingly concentrated in the hands of a small proportion of researchers, the findings underscore important gaps in funding and diversity that need to be addressed in the grant selection process, the authors, led by Yale's Mytien Nguyen, write.  Women were 34% less likely than men to be Super PIs, and Black scientists were 40% less likely than white scientists. Black women fared the worst: They were 71% less likely to be Super PIs than white men. To close these disparities, the authors call for diversifying grant review committees; scoring the diversity of research teams; and widening the often short window between new grant announcements and submission deadlines.

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Closer Look Could Meta and TikTok become today's Bell Labs for biology?  Alex Hogan/STAT Wait, new research on protein design powered by artificial intelligence was written by researchers at Salesforce? STAT's Brittany Trang tells us tech companies from Meta to TikTok's parent ByteDance have research programs on large language models — the ones behind text-generating AI tools like ChatGPT. "And what are proteins, if not run-on sentences, each word another amino acid?" she asks. Now I get it. Some people in biology compare the research arms of tech companies today to the basic research moonshots of the AT&T Bell Labs and Xerox PARCs of the last century. Here's where protein design comes in: At their core, large language models are algorithms trained to repeatedly play a game of "fill-in-the-blank" in a sentence. With a little tweaking, these language models can use datasets of protein sequences. And new protein sequences could theoretically be used for anything from growing plastic-eating bacteria to new biologic drugs. Read more on the challenges and implications. health  Covid-19 surveillance of essential workers added a burden but told them little It sounds like the worst of both possible worlds: Having to submit to health checks but learning nothing from them. At the pandemic's peak, that was the reality for essential workers who had to go into factories or warehouses or food stores where they faced rampant tech-based surveillance, from overhead infrared thermometers to wearables that tracked how close they were to one another. But they gained little from these measures, rarely told when co-workers were sick because of privacy concerns. Having so little information made it difficult to know how best to protect themselves and their families. Researchers from the organization Data & Society conducted an anonymous survey of 50 essential workers in four industries: meatpacking and food processing, warehousing, grocery retail, and manufacturing. They conclude Covid-19 surveillance on these mostly Black, Latino, and low-income workers compounded information gaps and added stress and extra labor for people already vulnerable to these factors before the pandemic. STAT's Ambar Castillo has more. pandemic Vaccination may mean milder and shorter long Covid, another study says As long as long Covid has hampered people's lives with fatigue, brain fog, and more, there have been hints that vaccination may weaken the severity and shorten the duration of persistent symptoms. New research in BMJ Medicine bolsters that connection, based on a study that matched 455 pairs of people for such factors as age, sex, co-existing conditions, and long Covid severity. Four months after infection, twice as many vaccinated as unvaccinated patients said their long Covid symptoms had disappeared.  A systematic review of 16 observational studies from five countries, also in the journal, concluded that Covid vaccines might both protect against and help treat long Covid, with the caveat that more evidence is needed. "While these studies are encouraging, trials comparing vaccination with placebo in patients with long Covid … are required to definitively recommend for or against vaccination to improve symptoms of long Covid," a companion editorial says. by the numbers

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Thanks for reading! More tomorrow,

elizabeth.cooney@statnews.com

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