Normal Science

BioByte 167: Prediction of Ligand-Residue Interactions with Ptarmigan-1, Improvements in the Physiological Relevance of DepMap, and Resetting the Growth Trajectory of Developing Organs After Injury

Welcome to Decoding Bio’s BioByte: each week our writing collective highlight notable news—from the latest scientific papers to the latest funding rounds—and everything in between. All in one place.Subscribe nowRobert Havell after John James Audubon, American Ptarmigan and White-tailed Grous, 1838, hand-colored engraving and aquatint on Whatman wove paperWhat we readPapersStructure-free, site-resolved contrastive learning extends small-molecule discovery beyond the reach of structure-based model...

STAT+: Study reveals new role for tau, a top target for Alzheimer’s drug development

In the race for new ways to treat Alzheimer’s and other devastating neurologic diseases, the protein tau has become a tantalizing target for drug developers. A new study lends support to those efforts by describing a previously unknown role that tau plays in neurodegeneration — and suggesting a novel way researchers might block the protein’s activity. A team led by Stanford scientists found that tau can gum up the works of a cell’s mitochondria, its energy-producing powerhouse. The protein sends...

FDA approves Moderna’s mRNA flu vaccine, the first to use the technology

In a move that seemed out of reach just six months ago, the Food and Drug Administration approved Moderna’s influenza vaccine for older adults on Wednesday, the first licensed flu shot made using messenger RNA technology. In February, Vinay Prasad, the FDA’s head of biologics at the time, overturned the recommendation of career staff and issued a rare “refusal-to-file” notice to the company, saying the agency wouldn’t review the vaccine based on the available data package. Two weeks later — afte...

STAT+: Federal regulators invite industry, researchers, and lobbyists to closed-door meetings on clinical AI

Federal health officials held closed-door conversations with prominent technology companies last month as the health department and its subagencies aim to safely boost adoption of clinical artificial intelligence. During a “clinical AI demo day” on July 8 at the Food and Drug Administration’s White Oak headquarters, officials from the FDA and the Centers for Medicare and Medicaid Services hosted leaders from 10 companies, according to an agenda for the event reviewed by STAT: Anthropic, Counsel ...

STAT+: Once again, child dies in gene-editing trial in China, rekindling debate on transparency and safety

Last year, a little-known Chinese startup took center stage at the American Society for Gene and Cell Therapy annual convention in New Orleans.  Speaking before a sprawling conference hall for the presidential symposium, HuidaGene CEO Alvin Luk presented data from one of the world’s first trials to test if CRISPR gene editing could treat children with Duchenne muscular dystrophy, an intractable, fatal disease. Similar efforts in the U.S. ran aground amid technical challenges, but HuidaGene had s...

STAT+: Startup promising to scale custom CRISPR therapies abruptly scraps plans

Aurora Therapeutics, a startup focused on building personalized gene-editing drugs, has scrapped its lead program and slashed staff, just seven months after it launched, STAT has learned.  A company spokesperson declined to say how many employees were laid off, or whether any of the startup’s three-person leadership team would stay on. Aurora’s chief scientific officer left the company this year.  The company appears to have been undercut by Beam Therapeutics, a larger and more established compe...

Sarah Urbut: Predicting Your Health Arc

Sarah Urbut is a physician-scientist at Harvard Medical School and the Massachusetts General Research Institute. She and her colleagues published a landmark paper in Nature on patient health journeys for 3 different cohorts—the Mass General Brigham patients, the UK Biobank, and the All of Us research program—with a cumulative 683,000 individuals, 348 diseases, and up to 52 years of follow-up. Integrating electronic medical records and polygenic risk scores (N=36). An AI model was developed and v...

Gene drives meet germline biology

I’ve written previously about gene drive technology, which allows gene editing of entire species. This works by engineering alleles that are inherited at greater than the expected Mendelian frequency of 50%. One example is a CRISPR gene introduced into mosquitoes that copies itself to all offspring, while making only the female offspring sterile. This results in an increase in the proportion of males, shortly followed by a population crash.I wrote “Gene drives: why the wait?” nearly four years a...

STAT+: Sandoz to pay $478.5 million to settle price-fixing allegations in long-running battle with states

Sandoz agreed to pay $478.5 million to settle allegations by dozens of states and resellers of medicines that the company engaged in a widespread conspiracy to artificially inflate and manipulate prices of generic drugs and harmed consumers by reducing competition. Under one settlement, the company agreed to pay $400 million over seven years starting in 2027 to resolve all remaining claims by U.S. states in three pending cases and make an additional payment of about $50 million to states that se...

Heart transplant candidates fail if they get too weak. New program tries inpatient prehab 

Cardiologists see this happen all too often: A patient is waiting in the hospital for a heart transplant, only to be disqualified because their fitness has fallen with each day in bed.  Dismayed by such cases, Radha Gopalan, a heart transplant cardiologist at Banner–University Medical Center Phoenix, decided to try a twist on other rehabilitation programs.Read the rest…

Does Employment Slow Cognitive Decline? Evidence from Labor Market Shocks

Founded in 1920, the NBER is a private, non-profit, non-partisan organization dedicated to conducting economic research and to disseminating research findings among academics, public policy makers, and business professionals.

Group averages obscure how an individual's brain controls behavior: study

Studying brain scan data from individuals — not group averages — reveals key brain-function differences in children who struggle with goal-oriented tasks, a Stanford Medicine study found.

Underwater robot tracks sperm whale conversations in real time

Researchers print structural colour with an inkjet printer

A nanoparticle ink that can print structural colour onto flat or 3D surfaces could be used to create anti-counterfeit images, smart windows and displays, and vibrant artworks

Urban Birds Are Rising Earlier Because of Traffic Noise (2013)

A male house sparrow. Photo by Edoddridge via Wikimedia Commons Most city dwellers have been woken up at some point by the chirping of birds outside their apartment window, but a new...

Forget LASIK: Safer, cheaper vision correction without lasers or surgery

Researchers are developing a futuristic alternative to LASIK that reshapes the eye without lasers or incisions. Using mild electrical pulses and platinum contact lenses, they temporarily soften the cornea so it can be molded into a new shape. Early tests on rabbit eyes successfully corrected nearsightedness in about a minute while preserving the eye’s structure.

Men's average testosterone levels have halved in last 50 years

Exclusive: Researchers warn of ‘major crisis in male reproductive health’ partly driven by obesity and diabetes

Perturb-MARS: Reading mouse experiments through a human lens

Multiplexed in-vivo perturbations, interpreted by a foundation model that has only ever seen human tumors.

Decline in IQ linked to frequent or dependent use of cannabis use in youth

Intelligence quotient decline following frequent or dependent cannabis use in youth: a systematic review and meta-analysis of longitudinal studies - Volume 51 Issue 2

Laser Phase Plate Cryo-Electron Microscopy

Learn how Biohub and UC Berkeley solved cryo-EM's contrast problem with a laser phase plate, opening a new window into the molecular machinery of living cells.

Nocturnal migratory birds follow rhythm of the moon

STAT+: Trump administration revises rebate pilot for 340B drug discount program, angering hospitals

The Trump administration has revised the terms of a pilot effort that will allow some drugmakers to offer rebates to certain hospitals and clinics for purchased medicines, a controversial move that may transform a key tenet of a federal drug discount program. The anticipated pilot for the 340B Drug Pricing Program, which is slated to go into effect on Jan. 1, 2027, will allow pharmaceutical companies to provide “timely” rebates, rather than offering upfront discounts. The program targets specifi...

STAT+: Novo Nordisk inflammation-targeting drug misses mark in heart disease study

Novo Nordisk said Friday that its investigational anti-inflammatory drug did not reduce the risk of major heart events in a pivotal trial, raising questions about the increasingly popular approach of targeting inflammation to prevent cardiovascular complications. In a Phase 3 study that enrolled over 6,300 people with heart disease, kidney disease, and inflammation, the drug, ziltivekimab, did lower markers of inflammation, Novo said. However, that did not translate into a reduction in risk of m...

STAT+: FDA expert committee votes in favor of Replimune’s melanoma treatment

A group of outside experts convened by the Food and Drug Administration on Thursday voted in favor of Replimune’s experimental treatment for advanced melanoma. In a 10-3 vote, the advisers determined that the biotech company has collected sufficient data to allow the FDA to review RP1, an engineered viral immunotherapy for melanoma. The agency tends to follow the recommendations of its expert panels but is not bound by them. What comes next could be seen by some as a signal of how the FDA, under...

NSF testing new Ph.D. model giving trainees academic and industry experience

Young life scientists have been increasingly abandoning academia, a seismic shift that has fueled calls for rethinking graduate education. On Wednesday, the Trump administration announced that it’s funding a pilot program to test a new strategy: Giving Ph.D. students industry experience — and dollars — as part of their training. The program is creating four-year Ph.D. programs that are jointly bankrolled by universities and industry partners, with additional support from the National Science Fou...

STAT+: From ‘lost cause’ to gold rush: Biotechs swarm to cure AATD 

Biotechs are spending billions to cure a rare liver disorder most Americans have never heard of. The contentious race features dueling technologies, patent wars, a broken alliance, and a boiling competition between the U.S. and Chinese drug industries. The disease, known as alpha-1 antitrypsin deficiency (AATD), is a slow-moving disaster for patients. Thanks to a single misspelled letter of DNA, their livers produce a mutant version of a protein that normally travels through the bloodstream and ...

STAT+: Preliminary trial results suggest a path to a safer Alzheimer’s treatment

An interim look at an Alzheimer’s disease clinical trial evaluating an amyloid-targeting treatment from ProMIS Neurosciences showed low rates of brain bleeding and no incidences of brain swelling — results reported Tuesday that suggest the drug could be safer than approved treatments.  In a blinded analysis that pooled safety data from patients receiving the ProMIS drug and a placebo, the total rate of ARIA was 4.4%, with all cases mild and asymptomatic, the company said.  None of the patients e...

FDA advisory panel narrowly rejects compounding of one peptide, backs two others

WASHINGTON — A Food and Drug Administration advisory panel recommended on Friday that compounding pharmacies be allowed to manufacture the peptides epitalon and semax, but narrowly voted to recommend against manufacturing emideltide.  The votes, which followed the panel’s decision on Thursday to recommend allowing pharmacies to make four other peptides, bring health secretary Robert F. Kennedy Jr. one step closer to his mission of making these unapproved compounds more available for Americans. P...

STAT+: Fatal gene-editing trial in China raises ethics and oversight questions

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Today, a win for RFK Jr., as an FDA panel narrowly voted in favor of allowing compounding pharmacies to produce certain unregulated peptides. Also, in heart-wrenching detail, Science writes of the six-year-old girl who died in China after receiving an experimental, brain-directed CRISPR therapy. The Readout’s taking a summer break next week, so we’ll see you in August!Conti...

STAT+: New data on Eli Lilly’s ‘triple-G’ drug leave open questions on heart benefit

Eli Lilly reported more results Thursday showing that its next-generation obesity drug is highly efficacious at weight loss, but the data from the study, focused on people with heart disease, aren’t enough to answer a critical question about the therapy — whether it reduces cardiovascular risk. The Phase 3 study enrolled nearly 2,000 people with severe obesity and heart disease, with or without diabetes. Those on the highest dose of the drug, called retatrutide, lost 22.6% of their weight after ...

STAT+: FDA staff and peptide enthusiasts set to clash at advisory panel

As more Americans experiment with synthetic peptides despite warnings about their unproven benefits and potential risks, an advisory panel to the Food and Drug Administration will meet on Thursday and Friday to discuss changes that could make peptides more accessible.  At issue is whether to allow compounding pharmacies to manufacture seven peptides that are currently designated as unsafe for them to make. The panel discussion is expected to be unusual in more ways than one.  First, many of the ...

New study supports testing of Merck’s Ebola vaccine in DRC outbreak

New data from a study in humans provides additional support to the idea that Merck’s Ebola vaccine Ervebo, licensed to target the Zaire species of ebolaviruses, could also offer some protection against another species currently circulating in a rapidly expanding outbreak in the Democratic Republic of the Congo. The new paper, published Wednesday in the New England Journal of Medicine after previously having been posted online before peer review, is one of a growing number of studies pointing to ...

STAT+: ‘Cochlear implant for blindness’ debuts in Europe, could launch in U.S. in 2027

Europeans can now receive a device that can partially restore their vision loss. Americans may not have to wait long to get it, either. Science Corporation announced Wednesday that European regulators had approved the commercial sale of the startup’s retinal implant that improves eyesight for patients with age-related macular degeneration in their central vision, enough to read books and road signs more clearly.  “We have a cochlear implant for vision now,” said Science founder and CEO Max Hodak...

STAT+: Broad Institute, partners unveil new initiative to develop gene therapies for rare diseases

The Broad Institute, Boston Children’s Hospital, and Maine’s Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for people with rare diseases and to make the life-saving therapies cheaper and more accessible to patients. With relatively few gene therapies on the market, families of children with rare genetic disorders have few options but to partner individually with researchers and raise millions of dollars to develop treatments for their conditions. The newly formed...

STAT+: Kalshi comes for biopharma, with prediction markets for clinical trials and FDA approvals

Prediction markets have permeated the worlds of sports, politics, and reality television shows. Now, the biopharma industry could be next. On Thursday, Kalshi, one of the world’s largest prediction market exchanges, said it will start taking bets on clinical trials and regulatory approvals. It’s starting with a small number of markets chosen in collaboration with its partner, AppliedXL, a tech company that monitors and predicts the outcomes of clinical trials.Continue to STAT+ to read the full s...

STAT+: Brain implant restores the sensation of touch in a person with quadriplegia

Keith Thomas just wanted to pet his dog.  A diving accident in 2020 damaged Thomas’ spinal cord and left him with virtually no ability to move or feel his limbs below his neck, so when Bow, his Malshipoo, grazed his legs or nuzzled in his lap, Thomas couldn’t feel it or return the affection.  His family got Bow right as Thomas received a brain implant in 2023 as part of an experimental study. The device changed his life. When it zapped his brain and spinal cord with tiny jolts of electricity at ...

STAT+: New study untangles how Epstein-Barr viral infection triggers immune response in multiple sclerosis

In a new study published in Science Translational Medicine on Wednesday, researchers say they have uncovered how Epstein-Barr virus launches immune responses that lead to the inflammation and nervous system damage seen in people with multiple sclerosis.  “It’s very nice now to be able to understand more about the underlying mechanisms of how EBV likely causes MS,” said lead author Kjetil Bjornevik, an assistant professor of epidemiology and nutrition at the Harvard T.H. Chan School of Public Hea...

STAT+: Drug metabolism AI competition results show that bigger may not always be better

In 2020, the CASP competition vaulted AlphaFold to prominence and a Nobel Prize. But the era of people being impressed by an artificial intelligence model correctly predicting the structure of a protein — once a challenge many experts didn’t think would be solved in their lifetime — is over. Now drug developers want AI that can solve their big problems, like discerning whether the body is going to attack a drug candidate and render it useless.  One such example is the pregnane X receptor, or PXR...

STAT+: A breakthrough in pancreatic cancer has experts excited — and braced for what’s to come

LONDON — When researchers started planning a pancreatic cancer conference here, the aim was to simply offer a counterpart to a regular U.S. meeting, where international scientists and doctors could discuss the latest developments in the specialty.  Then came the biggest shake-up in pancreatic cancer care in decades.  “It’s one of those moments,” Talia Golan, an oncologist at Israel’s Sheba Medical Center, said on a panel that conference organizers hastily added to the schedule centered on a new ...

Medicine is Moving From Calendars to Clocks

This week Tony Wyss-Coray and I published an invited review paper in Nature Medicine entitled “Biological Aging Clocks in Health and Disease.” The concepts and content of the paper may be difficult to understand for people not working in life science, but they are groundbreaking and have exciting implications for the future of medicine. So this edition of Ground Truths is intended to make all of the progress comprehensible for you and to hopefully see the coming inflection point in medicine. I’l...

STAT+: AstraZeneca, Ionis report major trial failure with heart disease drug

LONDON — In a blow to its cardiovascular aspirations, AstraZeneca said Thursday that its drug for a heart disease — one that has become an increasingly competitive target for biopharma companies — failed in a pivotal trial. The drug, called Wainua, on which AstraZeneca is partnered with Ionis Pharmaceuticals, did not outperform placebo in reducing cardiovascular death and clinical events for patients with a condition known as ATTR-CM, or transthyretin-mediated amyloid cardiomyopathy.  AstraZenec...

STAT+: 931 days. The drug approval scandal hiding in plain sight

This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. Northwest Biotherapeutics, a public biotech company developing a treatment for brain cancer, submitted a marketing application to U.K. regulators in late December 2023. The review was supposed to take 150 days, under an expedited regulatory pathway for drugs that address serious unmet medical needs. As you’re reading this newsletter, two year...

STAT+: Prime wins Beam arbitration, clearing path to clinic

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. A father fights to create and then save his daughter’s gene therapy, the White House nears a decision on an FDA chief, and the Trump administration pushes drugmakers to reshore generics manufacturing. A quest to save Grace — and help rare disease patients everywhere With $70 million and seemingly every fiber of his being, Matt Wilsey built a gene therapy company from scratc...

Outbreak of diarrhea-causing parasite grows to more than 1,000 cases

NEW YORK — Nearly 1,000 people in Michigan have been diagnosed with a parasitic infection that can cause weeks of watery diarrhea, making it the largest such outbreak in state history and one of the nation’s largest in years. No deaths have been reported and the source of the cyclospora infections hasn’t been identified. Meanwhile, investigations into similar illnesses have been going on in 28 other states, including in Ohio, where people just across the Michigan border are also becoming sick.Re...

STAT+: ARPA-H launches $160 million effort to develop custom gene editing drugs

ARPA-H, the U.S.’ “moonshot” agency for health research, announced Thursday that it will spend up to $160 million to push forward custom gene editing treatments for a spate of rare diseases.  The program, called THRIVE, will back seven different teams pursuing various groups of conditions affecting different organ systems.  Each team has a deadline of starting clinical trials by year three of the program, although some may start much sooner.  Continue to STAT+ to read the full story…

Transplants extended survival for patients whose stage 4 lung cancer hadn’t spread, study says

Could a lifesaving lung transplant strategy that contained the Covid-19 virus also save patients whose advanced lung cancer hasn’t spread?  That’s the question that inspired surgeons and oncologists at Northwestern Medicine to offer double lung transplants to patients who had run out of treatment options but whose late-stage cancer hadn’t left their lungs. People with late-stage lung cancer have not been transplant candidates before for two reasons: rates were high for cancer recurrence and low ...

A Conversation with Jack Scannell: The Intractable ‘Sociotechnical’ Problems of Drug Discovery and Development, Genetic Triumphalism and Predictive Validity

Jack is one of the sharpest thinkers on the pharmaceutical industry, with a talent for coining terms that then propagate among industry insiders and into wider tech circles. He coined Eroom’s Law in 2012, and has had a large impact by establishing predictive validity as the dominant variable in drug R&D productivity.In this interview we discuss:Whether the four original causes of Eroom’s Law still hold, and if we’ve seen a change in the trend.Why feeding AI with data from poor biological models ...

BioByte 166: A Deep Dive into Progressable Binders and Collaboration Between Bacteria to Survive Antibiotics

Reminder to apply to attend our fourth annual AI x Bio Summit at the NYSE on July 23rd! Speakers have been announced, view the full list + apply for your spot here.Subscribe nowWassily Kandinsky, Binding, 1932, oil on canvasWhat we readBlogsProgressable Binders: The Binders that Matter [Law and Kamichetty, Xaira Therapeutics, June 2026]Why it matters: This note from the CSO and CTO at Xaira Therapeutics broadly outlines how the company approaches de novo protein design efforts to ensure that mol...

Statins and blood pressure drugs changing health risks of obesity, study suggests

People with obesity have worse cardiovascular health than people with normal weight, especially as they get older, right? Not necessarily. People over 40 with obesity appear to have both their blood pressure and cholesterol under control at levels rivaling their peers with normal body mass index, research published Wednesday in the Lancet has found. The new study tracked these cardiovascular risk factors in adults of varying ages and BMIs for 25 years, an era preceding new obesity drugs but coin...

STAT+: A ‘historic’ FDA clearance raises the question: Is the LLM an interface or the decision-maker?

Digital health company UpDoc prompted a flurry of discussion when it announced in late June that its app for people with diabetes had received the first Food and Drug Administration clearance for medical software using “patient-facing large language models.”  Founded in 2023, UpDoc received clearance for the device in December and is just now publicly revealing its plans to use the technology as part of a care model that helps health systems manage patients outside the clinic with the help of AI...