News & Views

A new study expands the utility of the gold-standard ferret model to assess the risks that are associated with consuming raw milk and cheese contaminated with influenza A virus — marking an important step toward improving risk assessments in public health.

The first-in-human trial of AAV2i8-I1c (AB-1002) — an adeno-associated virus vector that targets a protein phosphatase inhibitor to cardiac muscle — suggests a cautious return of cardiac gene therapy after earlier setbacks, with a new target, dose optimization and improved vector design shaping future directions.

A new study provides physiological insights into the dying process, which could have substantial implications for resuscitation science, death determination and organ donation.

Three studies that leverage blood-based gene expression data identify molecular and cellular host response signatures in sepsis and critical illnesses, opening a pathway to mechanism-anchored precision therapy.

Two phase 1 studies report promising outcomes with chimeric antigen receptor (CAR) T cell therapies in patients with systemic lupus erythematosus, but the next stage of development must address key pitfalls around comparator arms and ancillary immune-modulating treatments.

A clinical trial of pregnant women provides critical safety and immunogenicity data in support of a two-dose Ebola vaccine regimen — and reinforces the importance of maternal immunization research, urgently needed for other preventable diseases.

Despite mixed efficacy outcomes, a phase 1/2 umbrella trial brings the glioblastoma field closer to a new era of diagnostic precision and targeted therapy.

Imaging data support the clinical effects of RNA interference agent vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy — and point to a potential new role for echocardiographic parameters in monitoring individual treatment responses.

A proteomic study begins to identify the direct and indirect effects of GLP-1 treatment on metabolic dysfunction-associated steatohepatitis (MASH), and reveals insights into treatment-induced changes in liver histology.

A clinical trial designed to overcome limitations of previous trials now confirms that individuals consume more calories from ultra-processed diets than from minimally processed diets, even when both diets meet UK dietary guidelines and participants are losing weight.

A study demonstrates the feasibility of a multi-site infusion approach to ensure broad distribution of an adeno-associated virus-based gene therapy for GM2 gangliosidosis, providing valuable lessons for the treatment of this and other rare lysosomal storage disorders.

A pair of phase 1 trials evaluating antibody–drug conjugates identify potential strategies to treat advanced gastroesophageal cancers and highlight the rapid expansion of claudin18.2-targeting drug development in solid tumors.

A new study leverages powerful multi-omics techniques to analyze immune dysregulation in smoke-exposed individuals, offering crucial insights for the development of novel diagnostics and interventions.

The phase 2 TUXEDO-3 trial identifies the HER3-targeting antibody–drug conjugate patritumab deruxtecan as a promising new therapeutic option for patients with leptomeningeal metastasis from solid tumors.

Positive results from a first-in-human clinical trial validate the ANGPTL3–ANGPTL8 complex as a new therapeutic target for reducing triglycerides and, potentially, cardiovascular disease risk.

A new Global Neurodegeneration Proteomics Consortium provides a wealth of data to boost mechanistic understanding, biomarker research and drug development during aging and in neurodegenerative diseases — and dementia in particular.

An explosion of digital tools and AI is catalyzing innovations in the mission to scale up mental health support worldwide. These paradigms currently rely on a ‘human in the loop’ — but who is the human in question, and does it matter?

A randomized phase 2a clinical trial of an AI-discovered drug and target combination for idiopathic pulmonary fibrosis shows safety and signs of efficacy, marking a concrete step forward in bringing AI-enabled drug discovery into the clinic.

A new analysis supports dietary guidelines to reduce the consumption of processed meat, sugar-sweetened beverages and trans-fatty acids — highlighting the need for a collaborative, meticulous health assessment framework for ultra-processed foods.

A new study identifies a novel subset of mast cells that cross-present tumor antigens in patients triple-negative breast cancer — and a pilot clinical trial shows how these can be exploited in immunotherapy strategies.