"Billionaire Bill Ackman's life was upended this February when his 26-year-old daughter collapsed in her Brooklyn apartment, suffering a brain hemorrhage that would render her unable to move, speak or see," writes the Washingon Post. But then... The hedge fund manager did what any good father would do — at least, any father with vast financial resources and what friends describe as a "Mr. Fix-It" tendency. He sprung into action, getting her topline care at New York City's Mt. Sinai Hospital and consulting a network of well-connected friends all over the world. He helped her access a novel cell therapy that has been gaining traction among longevity enthusiasts, who are seeking ways to prolong human life. And then — motivated by a newfound desire to accelerate scientific research — he vowed to spend more than $400 million to launch an institute dedicated to longevity and brain health... He posted this week that the U.S. Food and Drug Administration, in May, gave his daughter Lucy's medical team a first-time emergency authorization to transplant mitochondria — the energy-producing part of human cells — from her leg muscle to her eyes, in the hopes that the tiny cell powerhouses would facilitate healing in her optic nerves and perhaps even restore her vision. Ackman's story highlights the increasingly prominent role of the ultra-wealthy in making expensive medical bets on longevity treatments and other novel therapies, altering the direction of research as federal funding for science declines in the second Trump administration. But his saga is a deeply personal one, spurred by his daughter's medical crisis. The focus on longevity by Ackman, who made billions on Wall Street as a hedge fund manager, is aligned with similarly ambitious efforts from some of Silicon Valley's biggest names. OpenAI CEO Sam Altman has backed Retro Biosciences, a start-up whose goal is to add 10 healthy years to the human lifespan through cellular reprogramming. Amazon founder Jeff Bezos is reportedly a funder of Altos Labs, which focuses on cellular rejuvenation and resilience... Billionaire investor Peter Thiel, Google co-founder Sergey Brin and Oracle co-founder Larry Ellison are also deeply invested in life-extension methods and attempts to rewrite the biology of aging... The therapy given to Ackman's daughter Lucy, known as mitochondrial transplantation, is of great interest not only to people who have suffered injuries, but to longevity-focused scientists who believe that mitochondria may hold promise for reversing some of the effects of aging... In recent years, scientists have discovered that mitochondria have many other functions beyond converting food into energy. They serve as clearinghouses for damaged cells, opening up the possibility that they can help with cell regeneration and healing, said Daria Mochly-Rosen, professor of chemical and systems biology at the Stanford University School of Medicine and co-author of " The Life Machines: How Taking Care of Your Mitochondria Can Transform Your Health." "I am one of those who believes that focusing on mitochondrial health is a way to address many human diseases, including aging itself," she said. "It's exciting."

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The FDA has cleared the first Alzheimer's blood test for symptomatic patients as young as 40. The test measures amyloid and tau biomarkers from a single blood draw and identified positive biomarkers with 97.6% accuracy in a validation study. New Atlas reports: PrecivityAD2 is a single blood test that quickly assesses the proteins amyloid and tau, using mass spectrometry to measure plasma AB42/40 and p-tau217/np-tau217. The results are then scored, qualifying as negative, likely positive, or positive for AD. "Just over a year ago, there were no FDA-cleared blood tests for Alzheimer's, and now we have three," Isobel Coleman, chief executive officer of the Alzheimer's Drug Discovery Foundation (ADDF), said in a statement. "This is a proof point that sustained, early investment in translational science plays a catalytic role in moving promising ideas from the lab to patients." In a recent clinical validation study, 1,142 symptomatic adults were tested using PrecivityAD2. The process accurately identified 97.6% of positive biomarkers. What's more, the results remained consistent even in patients with comorbidities like chronic kidney disease and diabetes, which can distort plasma amyloid samples. It's worth noting that the test isn't intended for general health screenings, but for patients who already show some cognitive decline. "As PrecivityAD2 and tools like it move further into clinical practice, they will enable earlier detection, sharpen clinical trials, and build the foundation for precision medicine and combination therapies," Coleman added.

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Reuters reports "a major success in a new field of cancer treatment this week, as Moderna and Merck announced a personalized mRNA cancer vaccine "reduced the risk of recurrence and spread of melanoma in a late-stage trial". The vaccine was tested with Merck's widely used immunotherapy drug Keytruda: Thousands of patients who have undergone surgery to remove high-risk melanoma tumors could benefit as soon as next year if regulators approve the vaccine, Moderna President Stephen Hoge said in an interview. This is the first positive late-stage trial result for an mRNA cancer vaccine, which trains a patient's immune system to fight tumors by targeting specific mutations in those cells, and the first such study to show that adding a treatment to Keytruda worked better than that therapy alone... Interim results of the ongoing study, released on Wednesday, found the treatment, Intismeran, met both its primary target of reducing cancer recurrence and its secondary goal of preventing tumors from spreading to other parts of the body, compared with Keytruda alone. When the vaccine is injected into a patient, the patient's cells produce copies of mutations for the immune system to recognize and destroy... Merck said the companies are already in talks with regulators about the treatment. "Merck, Moderna and other companies are testing similar approaches against lung, breast and pancreatic cancers," the article points out. It adds that Karen Knudsen, CEO of the Parker Institute for Cancer Immunotherapy, "said the Moderna results could signal a new era for treating solid-tumor cancers." More from CNN: The trial result is "a monumental leap forward," validating mRNA technology as a cancer treatment, said Dr. Julie Gralow, chief medical officer of the American Society of Clinical Oncology... Roche and BioNTech, whose mRNA technology was used in the Pfizer COVID vaccines, are testing a similar treatment called autogene cevumeran in mid-stage studies in colon and pancreatic cancer patients who have undergone surgery. Results of the colon cancer trial are expected in 2027, with pancreatic trial results to follow in 2031. "We should be hearing results from multiple studies over the next few years and there's every reason to hope now, with this news, that many of them will be positive," said Dr. Robert Vonderheide, director of Penn Medicine's Abramson Cancer Center and president-elect of the American Association for Cancer Research.

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Pour la première fois, une thérapie à ARNm conçue sur mesure franchit avec succès son grand test de phase 3, dans la perspective de la lutte contre le cancer de la peau. Développé par Moderna et Merck, ce traitement personnalisé réduit significativement le risque de récidive.

An anonymous reader quotes a report from The Guardian: Scientists have made the first viruses designed by artificial intelligence in a milestone that raises hopes for new medicines but also concerns over how to ensure the technology remains safe. The viruses are specific kinds known as bacteriophages, which only infect bacteria and are used around the world to treat patients with persistent infections. In lab tests, a cocktail of the AI-designed viruses killed E coli bugs that were resistant to natural bacteriophages. Dr Brian Hie, a chemical engineer at Stanford University in California, used genome language models, the genetic equivalent of the large language models behind AI chatbots, to design functioning genomes for bacteriophages. The viruses were then made in the laboratory and pitted against E coli in a dish. The ability to "rapidly design" genomes and tune them for specific bugs while overcoming resistance could "transform phage therapy" and "expand biotechnological toolkits," the researchers wrote in the journal Science. But beyond the potential benefits, the scientists said the work raised "important biosafety, biocontainment and biosecurity considerations" and urged others who were designing whole genomes to "consult both safety and security professionals throughout the project." In an accompanying article, Prof Tom Inglesby and Dr Moritz Hanke at the Center for Health Security at Johns Hopkins University in Baltimore, reinforced the warning, writing: "Although this is promising for life sciences applications, it also raises urgent biosafety and biosecurity questions. The ability to compose viral genomes using generative AI now exists; the governance to safely steer it does not." Tom Ellis, a professor of synthetic genome engineering at Imperial College London, said the work was impressive, but revealed how hard it would be to make more complex genomes. "This is literally the smallest and easiest genome to make," he said. An AI trained on the genetic code of dangerous bugs could be used to design more harmful viruses, Ellis said, but controlling access to genetic data and having restrictions on making genomes that look dangerous would help. "Governments are working hard to do this already," he added. "But honestly," he said, "the threat from full AI design and writing of a genome of a virus or bacteria is very overblown when we consider that just taking existing pathogens and making gain-of-function changes to their genomes is so much easier and much more likely to be a real pathogenic threat." Dr Filippa Lentzos, a reader in science and international security at King's College London, said the most important point to intervene at the moment was when DNA was being manufactured. "It's important to see the bigger governance picture and not focus regulation solely on the AI model," she said. "A layered approach makes more sense: safeguards around model development and access, responsible research review, synthesis screening, and established laboratory biosafety and biosecurity."

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The FDA has approved the first mRNA flu vaccine in the United States after a clinical trial found it was about 27% more effective than a standard flu shot. Manufactured by Moderna and marketed as mFlusiva, the vaccine is expected to be available this fall for adults ages 50 to 64 and those 65 and older, though approval for the older group is conditional on Moderna conducting an additional clinical trial, NBC News reports. From the report: Many scientists and public health experts have touted the idea of an mRNA-based flu vaccine, which uses the same messenger RNA platform as the Covid vaccines from Moderna and Pfizer. That's because mRNA vaccines can be manufactured much faster than traditional vaccines, allowing scientists to better match circulating influenza strains. Moderna said it takes two to three months from picking the strain to rolling out its flu shot, compared with about six months for traditional flu shots.

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La branche logicielle du groupe français de défense a annoncé jeudi 23 juillet le rachat au suédois Nordic Capital de la plateforme américaine d’intelligence artificielle consacrée à la biotech ArisGlobal. Une façon de renforcer sa présence sur le marché convoité des sciences de la vie.

© PHOTO Sarah Meyssonnier/REUTERS