Nature Medicine, Published online: 24 June 2026; doi:10.1038/s41591-026-04480-w
Why Africa needs regional R&D hubs for basic science and translation research to accelerate local manufacturing.
Automated reanalysis of genomic data for rare disease diagnostics at scale
Nature Medicine, Published online: 24 June 2026; doi:10.1038/s41591-026-04477-5
Talos, a new tool for the automated analysis of genomic data, demonstrates the feasibility and diagnostic utility of systematic reanalyses of data in rare diseases.
STAT+: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval
Regenxbio said Wednesday it will apply for accelerated approval for its gene therapy for Duchenne muscular dystrophy — just a month after the company said regulators wanted to see another trial and that it would hold off on an application.
The announcement comes as the Food and Drug Administration seemingly reverses course on a slew of drugs it recently rejected or spurned. On Monday, Regenxbio said the FDA agreed to reconsider a Hunter syndrome gene therapy that it rejected just four months ago, when Marty Makary and Vinay Prasad were still at the agency’s helm. Both officials have left the FDA in recent weeks.
Wednesday’s announcement is unusual, however. In a press release, the company gave no indication reviewers had changed their stance on the company’s Duchenne gene therapy.
FDA finally analyzed its own youth tobacco data
Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here.
Good morning. A lot of people had reactions, comments, and theories regarding Lizzy’s story yesterday on the mystery patient who received retatrutide. What’s undeniable, though, is the strength of Lizzy’s reporting.
Opinion: How I used public radio to recruit 20,000 participants for a peer-reviewed study on walking breaks
Journalists don’t usually appear in the byline of peer-reviewed scientific papers. But recently, I received an email I’d been waiting on for nearly three years: A prestigious journal had accepted the findings from a study I helped lead with more than 20,000 participants across all 50 states. It was published Tuesday evening in the British Journal of Sports Medicine.
My team at NPR had joined forces with physiologist Keith Diaz’s at Columbia University Medical Center to test his lab findings. Specifically, we invited people to try taking movement breaks every 30 minutes, every hour, or every two hours. Our goal was to test whether short walking breaks, which have been shown to offset some of the damage of our sedentary, screen-bound lives, were actually feasible out in the real world.
More Daylight Exposure Could Lower Dementia Risk
Time spent in daylight could lower the risk of dementia and offer extra benefits for people at particularly high risk, new research suggests. The findings, published in General Psychiatry, could provide a low-cost way to support brain health.
Having less than 0.7 hours of bright daytime light per day was a stronger predictor of dementia than six traditional risk factors. Moderately bright natural light exposure—equivalent to an overcast day outdoors—was linked with a 16% reduction in the risk of dementia.
“Daytime light exposure may serve as a novel indicator of dementia risk,” said researcher Hongliang Feng, PhD, from Guangzhou Medical University in China. Natural cycles of darkness at night followed by bright light during the day are fundamental to entrain circadian rhythms. These regulate physiology, behavior, and cognition, with circadian disruptions common among people with dementia.
Noting that modern lifestyles limit daytime exposure to natural light, the researchers investigated exposure to day- and nighttime light using actigraphy devices that track body movements with built-in light sensors.
The study included 87,577 UK Biobank participants who wore accelerometers on their wrists to measure physical activity and natural light exposure for seven days. Over a median follow-up of 8.1 years, 741 of these people (0.85%) developed dementia.
Higher daytime light, both in terms of average exposure and the duration in bright light, was significantly associated with a lower dementia risk. Daytime light exposure above 1000 lux—a moderately bright light level equal to an overcast day outdoors—was associated with a hazard ratio of 0.84 for dementia. Longer exposure to brighter light of at least 0.70 hours with at least 5000 lux was linked with a further risk reduction, and a hazard ratio of 0.83.
In exploratory analyses, circadian rest-activity rhythms (CRAC) and brain structures mediated up to a third of this association, supporting the idea that improvements in circadian rhythms may have contributed to these results. Dementia protection from light exposure was stronger in people with high levels of nighttime light exposure, those with a “night owl” evening chronotype, or who carried the APOE ε4 allele, with a risk reduction of up to 41%.
Having more than 0.7 hours per day of bright daytime light of at least 5000 lux outperformed the established dementia risk predictors, including alcohol consumption, obesity, air pollution, hearing loss, use of vitamin D supplements, and traumatic brain injury.
However, nighttime light showed no significant association with dementia risk.
The findings point to the importance of higher daytime light exposure in reducing the chances of dementia and offer a simple, cost-free way to reduce this risk.
“Practical implementation pathways could include optimizing indoor lighting at home, community‐based outdoor activity promotion programs, and workplace lighting modifications designed to increase daytime light exposure, such as ensuring adequate illumination and access to natural light,” the researchers suggested.
They added: “Our findings underscore a more pronounced protective association of daytime light exposure in individuals with higher average nighttime light exposure, an evening chronotype, or APOE ε4 carrier status.
“In other words, these findings suggest a targeted approach to mitigate dementia risk by increasing daytime light levels for these populations.”
The post More Daylight Exposure Could Lower Dementia Risk appeared first on Inside Precision Medicine.
STAT+: Want high-quality generic drugs? One expert has ideas on how consumers can trust their supply
For many years, generic drugs have accounted for roughly 90% of the prescriptions doled out to Americans thanks to their lower cost. Yet reliable supplies have been an issue due to inconsistent quality — more than 60% of the generic shortages have been attributed to quality concerns, according to the Food and Drug Administration. Numerous manufacturers, many based in India, have been cited for violating manufacturing protocols that led to product recalls and, sometimes, bans on sending drugs to the U.S.
But Kevin Schulman, a professor and deputy director of the Clinical Excellence Research Center at the Stanford University School of Medicine, believes a solution is within reach. Schulman — who has also worked with an independent lab called Valisure that found impurities in some widely used medicines — argues the FDA should encourage testing by independent, accredited laboratories.
We recently spoke with him about the subject. This is an edited version of our conversation.
Quantum Mechanics Principles Help Researchers Build Cancer Prediction Model
A team of researchers at the University of Utah has developed a quantum mechanics-based artificial intelligence and machine learning method, which they say can improve the prediction of cancer outcomes and identify treatment targets using the comprehensive molecular background of individual patients. The approach, described in APL Quantum, addresses a major roadblock to leveraging conventional AI for predicting patient outcomes in clinical trials, namely the vast amounts of data needed to train large language models and to account for the complexity of disease drivers.
“It’s much more than just one gene—everything that’s happening in the cells of the patient matters,” said Orly Alter, PhD, associate professor of biomedical engineering at the University of Utah’s Scientific Computing & Imaging Institute. To take this into account, the team developed a method that is capable of analyzing multiple layers of molecular information simultaneously, including tumor DNA, blood DNA, and tumor RNA.
Clinical trials can enroll as few as 20 to 100 patients, while existing genomic datasets often contain data detailing millions to billions of molecular features. According to the researchers, many existing AI and machine-learning methods need more patient samples than genetic features to properly train the model. For instance, they pointed to a recent large language model of the 30,000-nucleotide genome of the COVID-19 virus, which needed 110 million samples. Extrapolating from this, the Utah team said that a complete modeling of the three billion nucleotides in the human genome would require 33 trillion patient samples.
To overcome this constraint, the investigators used a collection of algorithms known as multitensor comparative spectral decompositions, which Alter developed based on the quantum mechanical concepts of entanglement and superposition. The result, the team said, is analogous to a prism splitting light into its individual color components, providing data on multiple layers of a patient’s molecular makeup, including tumor and blood genomes and RNA transcriptomics, able to demonstrate linked patterns in cancer that can predict individual patient outcomes.
“The model rewrites a set of multiple omic profiles from one patient as a superposition of phenotypes, each represented by a set of multiple entangled patterns,” the researchers wrote. Importantly, data from one molecular profile can approximate an analysis from other profiles, which allows predictions to remain consistent among different types of biological data.
The researchers tested their model using an open-source dataset of the childhood cancer neuroblastoma. Their analysis found two previously unrecognized predictors of survival and treatment response, with each predictive element found in three separate, but interconnected data types: tumor genomes, blood genomes, and tumor transcriptomes. The study found that these predictors outperformed the currently used biomarker, the MYCN gene, for predicting treatment response and outcomes.
The new method builds on the substantial body of work by Alter and colleagues. Earlier research in this area had used related comparative spectral decomposition methods to analyze genomic and transcriptomic data in other tumor types, including glioblastoma.
The team will continue its work as it looks to develop an approach that can be used in the clinic. “That’s the ultimate precision medicine,” Alter said. “You have a single person. Can you take the data from just that one person and come up with a treatment for them? I think we can get there.”
The post Quantum Mechanics Principles Help Researchers Build Cancer Prediction Model appeared first on Inside Precision Medicine.
STAT+: Exclusive: Mystery man gets experimental GLP-1
You’re reading the web edition of D.C. Diagnosis, STAT’s twice-weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox on Tuesdays and Thursdays.
Let’s jump right in with Lizzy’s super interesting scoop about the one-and-only person granted compassionate-use access to an experimental GLP-1. Send news tips and best guesses to John.Wilkerson@statnews.com or John_Wilkerson.07 on Signal.
Why did one man get special access to an experimental GLP-1?
Here’s what we know about the sole person granted special access to Eli Lilly’s experimental obesity drug, thanks to Lizzy Lawrence’s reporting.
Solid Tumor CAR-T Therapy Approved in China, a World First
In a landmark advance for cellular immunotherapy, CARsgen Therapeutics has received regulatory approval in China for satricabtagene autoleucel (satri-cel; CT041), the first CAR-T cell therapy globally approved for the treatment of a solid tumor.
The National Medical Products Administration (NMPA) of China approved satri-cel for Claudin18.2 (CLDN18.2)-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma (G/GEJA) patients who have progressed after two prior lines of therapy. The decision is a turning point for the CAR-T field, which has improved hematologic malignancies but has struggled to overcome solid tumor biological barriers.
The approval addresses a major unmet need in gastric cancer, the fifth most commonly diagnosed cancer and the fifth leading cause of cancer-related death worldwide, with more than one million new cases and over 750,000 deaths annually. East Asia, particularly China, accounts for 40% of global cases due to risk factors like Helicobacter pylori infection, dietary exposures, and an aging population.
Despite advances in chemotherapy, targeted therapy, and immune checkpoint inhibitors, advanced gastric cancer patients have poor outcomes, especially after multiple treatment lines fail. CAR-T therapy first entered clinical testing for solid tumors in the late 1990s and early 2000s, with pioneering studies targeting ovarian cancer and later neuroblastoma and colorectal cancer, laying the groundwork for today’s next-generation cell therapies.
Satri-cel is an autologous CAR-T therapy that targets CLDN18.2, a stomach-specific tight-junction protein that is highly expressed in gastric and pancreatic cancers but has limited expression in normal tissues. The therapy uses a humanized anti-CLDN18.2 CAR construct that is linked to CD28 and CD3ζ signaling domains, which allows for targeted elimination of tumor cells.
The program’s CARsgen preconditioning strategy boosts CAR-T activity in the immunosuppressive solid tumor microenvironment. Patients receive low-dose nab-paclitaxel to increase CAR-T cell infiltration and antitumor efficacy in addition to cyclophosphamide and fludarabine lymphodepletion.
Clinical evidence supporting approval comes from a randomized confirmatory study published in The Lancet in 2025. In heavily pretreated patients with advanced G/GEJA, satri-cel demonstrated clinically meaningful efficacy and a manageable safety profile compared with available treatment options. The results provide one of the strongest demonstrations to date that CAR-T therapy can generate meaningful clinical benefit in solid tumors.
Importantly, CARsgen is already aggressively pursuing a development strategy beyond late-line gastric cancer. Currently, there are Phase Ib studies in advanced gastric, gastroesophageal junction, and pancreatic cancers, a confirmatory Phase II study in advanced G/GEJA, a Phase Ib study evaluating satri-cel as adjuvant therapy in pancreatic cancer, and investigator-initiated studies evaluating adjuvant and first-line sequential therapy. Satri-cel is being tested in a Phase Ib/II trial for advanced gastric and pancreatic adenocarcinoma outside China, demonstrating its global development goals.
The program has also been the subject of considerable regulatory attention. The FDA has designated satri-cel for CLDN18.2-positive gastric and gastroesophageal junction cancers as an RMAT and Orphan Drug. In Europe, the therapy has been awarded Orphan Medicinal Product designation and PRIME status by the European Medicines Agency. In China, the NMPA designated this product a Breakthrough Therapy for advanced gastric or gastroesophageal junction cancer patients who had failed at least two lines of treatment.
Satri-cel may be the first CAR-T therapy to clear the regulatory finish line in solid tumors, but the competition is heating up. Several companies are developing CLDN18.2-targeted CAR-T, T-cell engager, and antibody programs. AstraZeneca’s zolbetuximab franchise validated CLDN18.2 as a gastric cancer therapeutic target, and Chinese and U.S. biotech companies are developing cell therapy programs to replicate or improve on satri-cel’s results.
For cancer specialists and cell therapy specialists, satri-cel’s approval is not just a new treatment option but a proof-of-concept that engineered cellular therapies can successfully address the challenges of solid tumors. Whether this breakthrough can be applied to other tumor types remains to be seen, but the field has crossed a milestone that has eluded oncology for decades.
The post Solid Tumor CAR-T Therapy Approved in China, a World First appeared first on Inside Precision Medicine.