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More than four years after the last of four boys died in a troubled Phase I/II trial of a gene therapy candidate for X-linked Myotubular Myopathy (XLMTM), Astellas Gene Therapies says it remains intent on treating the rare muscle weakening disease through a genetic medicine. But instead of developing the adeno-associated virus (AAV)-based resamirigene bilparvovec (AT132) on its own, Astellas is now partnering with Kate Therapeutics—acquired in 2024 by Novartis for up to $1.1 billion—to develop a MyoAAV capsid-based gene therapy for XLMTM that the Japanese biopharma believes will be safer than AT132. The therapy, which Kate calls KT430, is…

Scribe’s CRISPR-based cholesterol reduction therapy is part of a three-pronged approach to combating cardiovascular disease. Genetic medicine biotech Scribe Therapeutics is readying for first-in-human trials of a CRISPR-based therapy aimed at one of the most common and stubborn drivers of cardiovascular disease: hypercholesterolemia. The Alameda, CA–based company has revealed plans to commence its first clinical study later this year – a liver-targeted therapy designed to lower low-density lipoprotein cholesterol by epigenetically silencing the PCSK9 gene. Scribe’s lead program, called STX-1150, is the first in a pipeline that also includes programs targeting lipoprotein(a) and severe triglyceride disorders, which, when combined, account…

© Le French Gut Among all the things we owe our mums is a lifelong gift of health: the foundation of our gut microbiome. This vast community of billions of microorganisms—mostly bacteria—plays a central role in digestion, immunity, and metabolism throughout life. During the first two years, this microbial core develops and stabilizes depending on factors such as breastfeeding, family environment, and exposure to pets or antibiotics. Scientists have a good understanding of how the microbiome evolves in early life and again in adulthood. Thousands of studies have described how these microbial communities change over time and how such shifts…

GlaxoSmithKline (GSK) researchers at work in an R&D laboratory at the company’s Stevenage, U.K., site. GSK has agreed to acquire Rapt Therapeutics for $2.2 billion, in a deal intended to bolster the buyer’s pipeline with Rapt’s ozureprubart (RPT904), a mid-phase anti-immunoglobulin E (IgE) monoclonal antibody which is being developed to protect against food allergens. [GlaxoSmithKline] GlaxoSmithKline (GSK) has agreed to acquire Rapt Therapeutics for $2.2 billion, the companies said today, in a deal intended to bolster the buyer’s pipeline with Rapt’s ozureprubart (RPT904), a mid-phase anti-immunoglobulin E (IgE) monoclonal antibody which is being developed to protect against food allergens. Ozureprubart…

New research from scientists at Trinity College Dublin could help explain why babies from multiple species, from mice to humans, are affected by infantile amnesia. The study, published in PLOS Biology, found that microglia, the immune cells of the central nervous system, might modulate this form of forgetfulness. The data also suggests that dysfunctional microglia could contribute to “altered memory trajectories” in neurodevelopment disorders, according to the team. Their new paper is titled “Microglial activity during postnatal development is required for infantile amnesia in mice.”  As infants grow, they absorb large quantities of information that help them make sense of…

This postdoc studies how warming winters reshape freshwater phytoplankton communities.Image credit:Anila Ajayan, ©iStock.com, WirestockAnila Ajayan is a postdoctoral researcher at the University of Vermont. In this postdoc portrait article, she discusses her work in freshwater phytoplankton ecology.Q | What’s your research background? I am currently working on trait-based approaches to understanding community interactions under environmental change. My research mostly spans mesocosm experiments, long-term datasets, and functional trait analyses. I am passionate about linking ecological theory with real-world freshwater conservation and global biodiversity initiatives.Q | How did you first get interested in your field of research? My interest in research began…

Rhinovirus, computer illustration. The rhinovirus infects the upper respiratory tract and is the cause of the common cold. It is spread by coughs and sneezes. [Kateryna Kon / Science Photo Library / Getty Images] Researchers at Yale School of Medicine have demonstrated how the cells lining our nasal passages work together to defend us from infection by rhinoviruses (RVs)—the most frequent cause of the common cold. Studying an organotypic model of the human nasal mucosa, the researchers found that it’s our body’s defense to rhinovirus—not the virus itself—that typically predicts whether or not we catch a cold, as well as…

Credit: Mumemories/Getty Images Vaccination against shingles could offer extra benefits beyond simply preventing the disease itself, with research suggesting it lowers several key measures biological decline. The study, in The Journals of Gerontology: Series A, provides further evidence that adult vaccines could play a role in promoting healthy aging beyond the prevention of infection. Vaccination against shingles, which is also known as herpes zoster, slowed two genetic signs of aging. It also reduced levels of inflammation, which at chronic, low levels has been linked with age-related conditions such as heart disease, frailty, and cognitive decline through a phenomenon dubbed “inflammaging,”…

Sponsored content brought to you by Nucleic acids are emerging as a third generation of pharmaceuticals following the clinical accomplishments of small-molecule drugs and biologics like proteins and monoclonal antibody (mAb) therapies. In particular, the initial widespread success of mRNA therapeutics with COVID-19 vaccines is propelling mRNA therapeutics into new spaces such as autoimmune diseases, rare diseases, and immuno-oncology. As mRNA based therapeutics move beyond the early vaccine successes to encompass diverse modalities, manufacturers face rising expectations to deliver products with consistent purity and quality. This raised bar is in turn forcing the evolution of traditional purification and quantitation processes…

Credit: Mohammed Haneefa Nizamudeen/Getty Images University of Virginia and Mount Sinai researchers report they have identified the mechanism that helps explain why cardiovascular disease accounts for more than half of deaths in patients with advanced chronic kidney disease (CKD). The study, published in the journal Circulation, shows that diseased kidneys release circulating extracellular vesicles (EVs) that contain cardiotoxic microRNAs that impair cardiac function, induce cardiomyocyte death, and contribute directly to heart failure and solve a long-standing riddle of why so many patients with CKD ultimately die from cardiovascular events. “Kidney and heart disease can develop silently, so they are often…