A dangerous strain of bird flu is spreading in US livestockMediaMedium/Alamy Since Donald Trump assumed office in January, the leading US public health agency has pulled back preparations for a potential bird flu pandemic. But as it steps back, another government agency is stepping up. While the US Department of Health and Human Servicespreviously held regular briefings on its efforts to prevent a wider outbreak of a deadly bird flu virus called H5N1 in people, it largely stopped once Trump took office. It has also cancelled funding for a vaccine that would have targeted the virus. In contrast, the US Department of Agriculturehas escalated its fight against H5N1’s spread in poultry flocks and dairy herds, including by funding the development of livestock vaccines. This particular virus – a strain of avian influenza called H5N1 – poses a significant threat to humans, having killed about half of the roughly 1000 people worldwide who tested positive for it since 2003. While the pathogen spreads rapidly in birds, it is poorly adapted to infecting humans and isn’t known to transmit between people. But that could change if it acquires mutations that allow it to spread more easily among mammals – a risk that increases with each mammalian infection. The possibility of H5N1 evolving to become more dangerous to people has grown significantly since March 2024, when the virus jumped from migratory birds to dairy cows in Texas. More than 1,070 herds across 17 states have been affected since then. H5N1 also infects poultry, placing the virus in closer proximity to people. Since 2022, nearly 175 million domestic birds have been culled in the US due to H5N1, and almost all of the 71 people who have tested positive for it had direct contact with livestock. Get the most essential health and fitness news in your inbox every Saturday. Sign up to newsletter “We need to take this seriously because whenconstantly is spreading, it’s constantly spilling over into humans,” says Seema Lakdawala at Emory University in Georgia. The virus has already killed a person in the US and a child in Mexico this year. Still, cases have declined under Trump. The last recorded human case was in February, and the number of affected poultry flocks fell 95 per cent between then and June. Outbreaks in dairy herds have also stabilised. It isn’t clear what is behind the decline. Lakdawala believes it is partly due to a lull in bird migration, which reduces opportunities for the virus to spread from wild birds to livestock. It may also reflect efforts by the USDA to contain outbreaks on farms. In February, the USDA unveiled a billion plan for tackling H5N1, including strengthening farmers’ defences against the virus, such as through free biosecurity assessments. Of the 150 facilities that have undergone assessment, only one has experienced an H5N1 outbreak. Under Trump, the USDA also continued its National Milk Testing Strategy, which mandates farms provide raw milk samples for influenza testing. If a farm is positive for H5N1, it must allow the USDA to monitor livestock and implement measures to contain the virus. The USDA launched the programme in December and has since ramped up participation to 45 states. “The National Milk Testing Strategy is a fantastic system,” says Erin Sorrell at Johns Hopkins University in Maryland. Along with the USDA’s efforts to improve biosecurity measures on farms, milk testing is crucial for containing the outbreak, says Sorrell. But while the USDA has bolstered its efforts against H5N1, the HHS doesn’t appear to have followed suit. In fact, the recent drop in human cases may reflect decreased surveillance due to workforce cuts, says Sorrell. In April, the HHS laid off about 10,000 employees, including 90 per cent of staff at the National Institute for Occupational Safety and Health, an office that helps investigate H5N1 outbreaks in farm workers. “There is an old saying that if you don’t test for something, you can’t find it,” says Sorrell. Yet a spokesperson for the US Centers for Disease Control and Preventionsays its guidance and surveillance efforts have not changed. “State and local health departments continue to monitor for illness in persons exposed to sick animals,” they told New Scientist. “CDC remains committed to rapidly communicating information as needed about H5N1.” The USDA and HHS also diverge on vaccination. While the USDA has allocated million toward developing vaccines and other solutions for preventing H5N1’s spread in livestock, the HHS cancelled million in contracts for influenza vaccine development. The contracts – terminated on 28 May – were with the pharmaceutical company Moderna to develop vaccines targeting flu subtypes, including H5N1, that could cause future pandemics. The news came the same day Moderna reported nearly 98 per cent of the roughly 300 participants who received two doses of the H5 vaccine in a clinical trial had antibody levels believed to be protective against the virus. The US has about five million H5N1 vaccine doses stockpiled, but these are made using eggs and cultured cells, which take longer to produce than mRNA-based vaccines like Moderna’s. The Moderna vaccine would have modernised the stockpile and enabled the government to rapidly produce vaccines in the event of a pandemic, says Sorrell. “It seems like a very effective platform and would have positioned the US and others to be on good footing if and when we needed a vaccine for our general public,” she says. The HHS cancelled the contracts due to concerns about mRNA vaccines, which Robert F Kennedy Jr – the country’s highest-ranking public health official – has previously cast doubt on. “The reality is that mRNA technology remains under-tested, and we are not going to spend taxpayer dollars repeating the mistakes of the last administration,” said HHS communications director Andrew Nixon in a statement to New Scientist. However, mRNA technology isn’t new. It has been in development for more than half a century and numerous clinical trials have shown mRNA vaccines are safe. While they do carry the risk of side effects – the majority of which are mild – this is true of almost every medical treatment. In a press release, Moderna said it would explore alternative funding paths for the programme. “My stance is that we should not be looking to take anything off the table, and that includes any type of vaccine regimen,” says Lakdawala. “Vaccines are the most effective way to counter an infectious disease,” says Sorrell. “And so having that in your arsenal and ready to go just give you more options.” Topics: #how #agriculture #agency #became #key

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology. The Trump administration has shut down more than 100 climate studies The Trump administration has terminated National Science Foundation grants for more than 100 research projects related to climate change, according to an MIT Technology Review analysis of a database that tracks such cuts. The move will cut off what’s likely to amount to tens of millions of dollars for studies that were previously approved and, in most cases, already in the works. Many believe the administration’s broader motivation is to undermine the power of the university system and prevent research findings that cut against its politics. Read the full story. —James Temple This architect wants to build cities out of lava Arnhildur Pálmadóttir is an architect with an extraordinary mission: to harness molten lava and build cities out of it.Pálmadóttir believes the lava that flows from a single eruption could yield enough building material to lay the foundations of an entire city. She has been researching this possibility for more than five years as part of a project she calls Lavaforming. Together with her son and colleague Arnar Skarphéðinsson, she has identified three potential techniques that could change how future homes are designed and built from repurposed lava. Read the full story.—Elissaveta M. Brandon This story is from the most recent edition of our print magazine, which is all about how technology is changing creativity. Subscribe now to read it and to receive future print copies once they land. The must-reads I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology. 1 America is failing to win the tech race against ChinaIn fields as diverse as drones and energy.+ Humanoid robots is an area of particular interest.+ China has accused the US of violating the pair’s trade truce.2 Who is really in charge of DOGE?According to a fired staffer, it wasn’t Elon Musk.+ DOGE’s tech takeover threatens the safety and stability of our critical data.3 Brazilians will soon be able to sell their digital dataIt’s the first time citizens will be able to monetize their digital footprint.4 The Trump administration’s anti-vaccine stance is stoking fear among scientistsIt’s slashing funding for mRNA trials, and experts are afraid to speak out.+ This annual shot might protect against HIV infections.5 Tech companies want us to spend longer talking to chatbotsThose conversations can easily veer into dangerous territory.+ How we use AI in the future is up to us.+ This benchmark used Reddit’s AITA to test how much AI models suck up to us.6 Tiktok’s mental health videos are rife with misinformationA lot of the advice is useless at best, and harmful at worst.7 Lawyers are hooked on ChatGPTEven though it’s inherently unreliable.+ Yet another lawyer has been found referencing nonexistent citations.+ How AI is introducing errors into courtrooms.8 How chefs are using generative AI They’re starting to experiment with using it to create innovative new dishes.+ Watch this robot cook shrimp and clean autonomously.9 The influencer suing her rival has dropped her lawsuitThe legal fight over ownership of a basic aesthetic has come to an end.10 Roblox’s new game has sparked a digital fruit underground marketAnd players are already spending millions of dollars every week.Quote of the day “We can’t substitute complex thinking with machines. AI can’t replace our curiosity, creativity or emotional intelligence.” —Mateusz Demski, a journalist in Poland, tells the Guardian about how his radio station employer laid him off, only to later launch shows fronted by AI-generated presenters. One more thing ​​Adventures in the genetic time machineAn ancient-DNA revolution is turning the high-speed equipment used to study the DNA of living things on to specimens from the past.The technology is being used to create genetic maps of saber-toothed cats, cave bears, and thousands of ancient humans, including Vikings, Polynesian navigators, and numerous Neanderthals. The total number of ancient humans studied is more than 10,000 and rising fast.The old genes have already revealed remarkable stories of human migrations around the globe.But researchers are hoping ancient DNA will be more than a telescope on the past—they hope it will have concrete practical use in the present. Read the full story.  —Antonio Regalado We can still have nice things A place for comfort, fun and distraction to brighten up your day.+ The ancient Persians managed to keep cool using an innovative breeze-catching technique that could still be useful today.+ Knowledge is power—here’s a helpful list of hoaxes to be aware of.+ How said it: Homer Simpson or Pete Hegseth?+ I had no idea London has so many cat statues. #download #climate #studies #are #being

Robert F. Kennedy Jr's HHS has cancelled Moderna's mRNA bird flu vaccine. That and new restrictions on Covid vaccines puts us at increased risk of deaths and a pandemic. #loss #mrna #bird #flu #vaccines

Nature, Published online: 21 May 2025; doi:10.1038/d41586-025-01635-6This is what happened when a Nature reporter designed his own protein using AI. Plus, the ‘Gollum effect’ that makes researchers hoard data and hopeful results for the first mRNA bird-flu vaccine for cattle. #daily #briefing #used #design #awful

May 23, 20257 min readWhat FDA’s Planned Limits on COVID Vaccinations Mean for HealthDespite the fact that vaccines against COVID have already undergone strict safety reviews and that people continue to die from the disease, Trump’s FDA is moving to reduce access to annual COVID boosters for healthy AmericansBy Stephanie Armour & KFF Health News aire images/Getty ImagesLarry Saltzman has blood cancer. He’s also a retired doctor, so he knows getting covid-19 could be dangerous for him — his underlying illness puts him at high risk of serious complications and death. To avoid getting sick, he stays away from large gatherings, and he’s comforted knowing healthy people who get boosters protect him by reducing his exposure to the virus.Until now, that is.Vaccine opponents and skeptics in charge of federal health agencies — starting at the top with Health and Human Services Secretary Robert F. Kennedy Jr. — are restricting access to covid shots that were a signature accomplishment of President Donald Trump’s first term and cost taxpayers about billion to develop, produce, and distribute. The agencies are narrowing vaccination recommendations, pushing drugmakers to perform costly clinical studies, and taking other steps that will result in fewer people getting protection from a virus that still kills hundreds each week in the U.S.On supporting science journalismIf you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today.“There are hundreds of thousands of people who rely on these vaccines,” said Saltzman, 71, of Sacramento, California. “For people who are immunocompromised, if there aren’t enough people vaccinated, we lose the ring that’s protecting us. We’re totally vulnerable.”The Trump administration on May 20 rolled out tougher approval requirements for covid shots, described as a covid-19 “vaccination regulatory framework,” that could leave millions of Americans who want boosters unable to get them.The FDA will encourage new clinical trials on the widely used vaccines before approving them for children and healthy adults. The requirements could cost drugmakers tens of millions of dollars and are likely to leave boosters largely out of reach for hundreds of millions of Americans this fall.Under the new guidance, vaccines will be available for high-risk individuals and seniors. But the FDA will encourage drugmakers to commit to conducting post-marketing clinical trials in healthy adults when the agency approves covid vaccines for those populations.For the past five years, the shots have been recommended by the Centers for Disease Control and Prevention for everyone 6 months and older. They have been available each fall after being updated to reflect circulating strains of the virus, and the vaccines have been shown to be safe and effective in clinical trials.Vinay Prasad, who leads the FDA’s division overseeing vaccines, cited “distrust of the American public” as he announced the new guidelines at a May 20 briefing.“We have launched down this multiyear campaign of booster after booster after booster,” he said, adding that “we do not have gold-standard science to support this for average-risk, low-risk Americans.”The details were outlined in a May 20 article in The New England Journal of Medicine, written by FDA Commissioner Marty Makary. He and Prasad later followed up with the briefing, which appeared the same day on YouTube.The added limits on access aren’t the result of any recent data showing there are new health risks from the covid vaccines. Instead, they reflect a different regulatory stance from Kennedy, who has a history of anti-vaccine activism, and Makary, who has questioned the safety data on covid mRNA shots.Announcing a major regulatory change in a medical journal and YouTube video is a highly unusual approach that still leaves many questions about implementation unanswered. It remains unclear when the changes will go into effect or whether there will be any public comment period. The changes were announced by the administration before an FDA advisory committee meeting on May 22 to consider the 2026 covid vaccine formula.It’s a sharp reversal from the first Trump administration, which launched Operation Warp Speed — the effort that led to the development of the covid shots. Trump called the vaccines the “gold standard” and a “monumental national achievement.”Concerns About Higher TransmissionThe announcement is rattling some patient advocacy groups, doctors, nursing home leaders, and researchers who worry about the ramifications. They say higher-risk individuals will be more likely to get covid if people who aren’t at risk don’t get boosters that can help reduce transmission. And they say the FDA’s restrictions go too far, because they don’t provide exceptions for healthy individuals who work in high-risk settings, such as hospitals, who may want a covid booster for protection.The limits will also make it harder to get insurance coverage for the vaccines. And the FDA’s new stance could also increase vaccine hesitancy by undermining confidence in covid vaccines that have already been subject to rigorous safety review, said Kate Broderick, chief innovation officer at Maravai Life Sciences, which makes mRNA products for use in vaccine development.“For the public, it raises questions,” she said. “If someone has concerns, I’d like them to know that of all the vaccines, the ones with the most understood safety profile are probably covid-19 vaccines. There is an incredible body of data and over 10 billion doses given.”Some doctors and epidemiologists say it could leave healthy people especially vulnerable if more virulent strains of covid emerge and they can’t access covid shots.“It’s not based on science,” said Rob Davidson, an emergency room doctor in Michigan and executive director of the Committee to Protect Health Care, which works to expand health care access. “It’s what we were all worried would happen. It risks peoples’ lives.”Current federal regulators say there is no high-quality evidence showing that vaccinating healthy people, including health workers who are near or around immunocompromised people, provides an additional benefit.“It is possible, actually, that such approvals and strategies provide false reassurance and lead to increased harms,” Prasad said.The covid vaccines underwent clinical trials to assess safety, and they have been subject to ongoing surveillance and monitoring since they obtained emergency use authorization from the FDA amid the pandemic. Heart issues and allergic reactions can occur but are rare, according to the CDC.On a separate track, the FDA on May 21 posted letters sent in April to makers of the mRNA covid vaccines to add information about possible heart injury on warning labels, a move that one former agency official described as overkill. The action came after the Permanent Subcommittee on Investigations, a panel of the Senate Homeland Security and Governmental Affairs Committee, held a hearing on alleged adverse events associated with covid vaccines.Limiting boosters to healthy people goes against guidance from some medical groups.“The COVID-19 vaccine is safe, effective, and the best way to protect children,” Sean O’Leary, chair of the Committee on Infectious Diseases at the American Academy of Pediatrics, said in an email. “Young children under 5 continue to be at the highest risk, with that risk decreasing as they get older.”Unsupported Claims About mRNA VaccinesThe covid booster clampdown is supported by many adherents of the “Make America Healthy Again” movement, which casts suspicion on traditional medicine. Some opponents of covid mRNA vaccines say without evidence that the shots cause “turbo” cancer, are genetic bioweapons, and cause more heart damage than the covid virus.There is no evidence the shots lead to rapid and aggressive cancers. Cancer rates decreased an average of 1.7% per year for men and 1.3% for women from 2018 to 2022, according to the National Institutes of Health. The covid vaccines debuted in 2021.Federal regulators say narrowing who can get the boosters will align the U.S. with policies of European nations. But other countries have vastly different economic structures for health care and approaches to preventive care. Many European countries, for example, don’t recommend flu shots for the entire population. The U.S. does in part because of the financial drain attributed to lost productivity when people are sick.They also want more information. “I think there’s a void of data,” Makary told CBS News on April 29. “And I think rather than allow that void to be filled with opinions, I’d like to see some good data.”A massive five-year study on covid vaccine safety by the Global Vaccine Data Network, involving millions of people, was underway, with about a year left before completion. The Trump administration terminated funding for the project as part of cuts directed by the president’s Department of Government Efficiency, and work on the study has stopped for now.There are a multitude of studies, however, on the vaccines’ effectiveness in preventing severe illness, hospitalization, and death.Limiting boosters for healthy people can be risky, some doctors say, because people don’t always know when they fall into higher-risk categories, such as individuals who are prediabetic or have high blood pressure. The covid vaccine restrictions could deter them from getting boosted, and they might experience worse complications from the virus as a result. For example, about 40% of people with hepatitis C are unaware of their condition, according to a study published in 2023.The number of people getting covid vaccines has already dropped significantly since the height of the crisis. More than half of the more than 258 million adults in the U.S. had gotten a covid vaccination as of May 2021, according to the CDC. In each of the past two seasons, less than 25% of Americans received boosters, CDC data shows.While deaths from the virus have dropped, covid remains a risk, especially when cases peak in December and January. Weekly covid deaths topped 2,580 as recently as January 2024, according to CDC data.Some high-risk individuals are worried that the new restrictions are just the first salvo in halting all access to mRNA shots. “The HHS motivation really is hidden, and it’s to dismiss all mRNA technology,” said Michael Osterholm, an epidemiologist at the University of Minnesota.Officials at the NIH have told scientists to remove references to mRNA in grant applications. HHS also announced plans in May to develop new vaccines without mRNA technology, which uses messenger RNA to instruct cells to make proteins that trigger an immune response.Rose Keller, 23, is concerned about future access to covid shots. She would be eligible under the current announcement — she has cystic fibrosis, a progressive genetic condition that makes the mucus in her lungs thick and sticky, so covid could land her in the hospital. But she is concerned the Trump administration may go further and restrict access to the vaccines as part of a broader opposition to mRNA technology.“I’ve had every booster that’s available to me,” said Keller, a government employee in Augusta, Maine. “It’s a real worry if I don’t have the protection of a covid booster.”KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — the independent source for health policy research, polling, and journalism. #covid #vaccines #face #potential #new

May 22, 20253 min readBird Flu Vaccine for Cows Passes Early TestResearchers have tested an mRNA vaccine against avian influenza in calves with promising resultsBy Humberto Basilio & Nature magazine Westend61/Getty ImagesAs bird flu sweeps across US poultry and cattle farms, researchers are racing to find ways to contain the outbreaks before they ignite a human pandemic. Now, a team of scientists has developed a fresh approach: the first mRNA bird-flu vaccine for cattle.Early findings, posted this month on the preprint server bioRxiv, reveal that the experimental vaccine triggers a strong immune response to the virus, and protects against infection in calves. The results have not yet been peer-reviewed.This development could mark a crucial step towards creating flu vaccines for livestock and reducing the risk of animal-to-human transmission of a virus that poses a “real pandemic threat”, says Scott Hensley, a virologist at the University of Pennsylvania in Philadelphia, and a co-author of the work.On supporting science journalismIf you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today.Fears of a bird-flu pandemic have been rising since the first confirmed outbreak of the H5N1 avian influenza virus in dairy cattle was reported in March 2024. Since then, the virus has affected more than 1,000 dairy herds across 17 US states. Health officials have linked 64 human infections and one death to the outbreak.A fresh approachTo create a cattle vaccine, Hensley and his team built on more than a decade of work on seasonal bird-flu mRNA vaccines. The researchers took one such vaccine candidate and swapped out its viral haemagglutinin gene — which encodes a protein known to elicit an immune response — with the corresponding gene from the new H5N1 virus found on dairy farms. “It’s so easy to switch,” says Hensley. “That’s really the value of using mRNA-based vaccines.”Last year, Hensley’s team showed that their vaccine protects against avian flu in ferrets, a commonly used laboratory model for testing flu vaccines. For the latest work, they inoculated 10 calves and, 49 days later, fed them milk from H5N1-infected cows — a suspected route of transmission among cattle.After that exposure, the vaccinated calves had significantly lower levels of viral RNA than the unvaccinated calves did, indicating that the vaccine helped to curb infection.The study tested only vaccine responses in calves; much of the avian-flu transmission on dairy farms occurs among lactating adult cattle, says virologist Richard Webby, director of the World Health Organization Collaborating Centre for Studies on the Ecology of Influenza in Animals and Birds in Memphis, Tennessee. Hensley’s team is already working on extra trials in lactating cows.Even without that data, the current results are a strong first step towards developing a vaccine: “It’s good news,“ Webby says.Uncertain futureOther livestock vaccines could be on the way. The US Department of Agriculture has approved at least seven candidates for field trials this year. In mid-February, the agency also conditionally approved a bird flu vaccine for chickens.But political headwinds against mRNA vaccines could threaten Hensley’s effort. Republican lawmakers in South Carolina, Texas and Montana have introduced bills to ban mRNA vaccines in livestock, arguing that they pose risks to human health. And some scientists worry that vaccine scepticism in US President Donald Trump’s administration will lead to cuts in funding for mRNA-vaccine development.“I’m optimistic that they will continue to support the development of these vaccines,” Hensley says. “It would be a crime right now to stop it.”Other scientists question whether vaccines for cattle will be economically viable for farmers. That will depend on how many doses are needed and its price, says microbiologist Shollie Falkenberg at Auburn University in Alabama. “The livestock industry is in the business of making money,” she says. “At the end of the day, people want to see the economics behind it.”Still, vaccinating cattle might soon become necessary to prevent further infections, potential deaths and mounting economic losses, says Webby.“I don’t think that cattle vaccines on their own are sort of a silver bullet,” he says. “But we have to do something different because what we're doing now is clearly not working.”This article is reproduced with permission and was first published on May 21, 2025. #bird #flu #vaccine #cows #passes

N=1 From birth to gene-edited in 6 months: Custom therapy breaks speed limits The N-of-1 accomplishment provides a template for swift, personalized genetic therapies. Beth Mole – May 16, 2025 2:00 pm | 58 Rebecca Ahrens-Nicklas, MD, PhD, and Penn Medicine’s Kiran Musunuru, MD, PhD, visiting KJ Credit: CHOP Rebecca Ahrens-Nicklas, MD, PhD, and Penn Medicine’s Kiran Musunuru, MD, PhD, visiting KJ Credit: CHOP Story text Size Small Standard Large Width * Standard Wide Links Standard Orange * Subscribers only   Learn more News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ultra-rare mutation in KJ that breaks a liver enzyme. That enzyme is required to convert ammonia, a byproduct of metabolism, to urea, a waste product released in urine. Without treatment, ammonia would build up to dangerous levels in KJ—and he would have a 50 percent chance of dying in infancy. While the gene-editing treatment isn't a complete cure, and long-term success is still uncertain, KJ's condition has improved and stabilized. And the treatment's positive results appear to be a first for personalizing gene editing. Now, who doesn't love a good story about a seemingly miraculous medical treatment saving a cute, chubby-cheeked baby? But, this story delivers more than an adorable bundle of joy; the big triumph is the striking timeline of the treatment's development—and the fact that it provides a template for how to treat other babies with ultra-rare mutations. Breakneck breakthrough The gene editing technology used for KJ is not exactly new, nor is the delivery system. CRISPR has already proven to be a successful gene therapy. What's stunning is the speed. KJ's mutation was identified within days of his birth. Within weeks, researchers were growing cells in petri dishes that carried genetic sequences copied from KJ. In month two, they used those cells to train molecular gene-editing machinery to target and correct KJ's mutation—a spot in the DNA coding for the liver enzyme where there's a Tinstead of a C. At the beginning of the third month, researchers had created genetically engineered mice that carry KJ's specific mutation, too. In the boy's fourth month, researchers were meeting with the Food and Drug Administration to discuss regulatory approval for a clinical trial—a trial where KJ would be the only participant. They were also working with the institutional review boardat Children’s Hospital of Philadelphia to go over the clinical protocol, safety, and ethical aspects of the treatment. The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month five, they started toxicology testing in mice. In the mice, the experimental therapy corrected KJ's mutation, replacing the errant A-T base pair with the correct G-C pair in the animals' cells. The first dose provided a 42 percent whole-liver corrective rate in the animals. At the start of KJ's sixth month, the researchers had results from safety testing in monkeys: Their customized base-editing therapy, delivered as mRNA via a lipid nanoparticle, did not produce any toxic effects in the monkeys. A clinical-grade batch of the treatment was readied. In month seven, further testing of the treatment found acceptably low-levels of off-target genetic changes. The researchers submitted the FDA paperwork for approval of an "investigational new drug," or IND, for KJ. The FDA approved it in a week. The researchers then started KJ on an immune-suppressing treatment to make sure his immune system wouldn't react to the gene-editing therapy. Then, when KJ was still just 6 months old, he got a first low dose of his custom gene-editing therapy. “Transformational” After the treatment, he was able to start eating more protein, which would have otherwise caused his ammonia levels to skyrocket. But he couldn’t be weaned off of the drug treatment used to keep his ammonia levels down. With no safety concerns seen after the first dose, KJ has since gotten two more doses of the gene therapy and is now on reduced nitrogen scavenging medication. With more protein in his diet, he has moved from the 9th percentile in weight to 35th or 40th percentile. He's now about 9 and a half months old, and his doctors are preparing to allow him to go home from the hospital for the first time. Though he will have to be closely monitored and may still at some point need a liver transplant, his family and doctors are celebrating the improvements so far. In the past, the development of such a treatment would have taken years. But the swift and diligent response to KJ's condition led to timely treatment. The timeline and steps provide a guide for development of other customized gene therapies. KJ's treatment was presented this week at the American Society of Gene & Cell Therapy Annual Meeting in New Orleans. It was also simultaneously published in the New England Journal of Medicine. In an accompanying editorial, Peter Marks—a former top regulator at the FDA—called KJ's  treatment a "platform technology" that could be used as a template for treating millions of others with rare genetic conditions. "The development of gene-editing products to address N-of-1 disorders with the use of mRNA encapsulated in lipid nanoparticles represents one of the most obvious opportunities for the application of a platform-technology approach that could be transformational," he wrote. In all, KJ's treatment "shows the potential strength of the application of cutting-edge science and technology with a forward-leaning regulatory approach to safely expedite the development and availability of life-saving medicines," Marks wrote. KJ's doctors agree. “Years and years of progress in gene editing and collaboration between researchers and clinicians made this moment possible, and while KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient’s needs," said Rebecca Ahrens-Nicklas, a pediatrician and gene therapy expert at CHOP and the University of Pennsylvania, who treated KJ. Beth Mole Senior Health Reporter Beth Mole Senior Health Reporter Beth is Ars Technica’s Senior Health Reporter. Beth has a Ph.D. in microbiology from the University of North Carolina at Chapel Hill and attended the Science Communication program at the University of California, Santa Cruz. She specializes in covering infectious diseases, public health, and microbes. 58 Comments #birth #geneedited #months #custom #therapy