German researchers have cracked the decades-long mystery of why males are more susceptible to acute kidney injury than females, demonstrating that estrogen has a protective effect in females.

The U.K. Medicines and Healthcare products Agency (MHRA) is calling for more research into the vaginal microbiome as a way to redress the historic under-representation of women in clinical studies, which it said has contributed to “critical shortcomings” in understanding of female-specific conditions.

Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.

The largest genome-wide association study to date of myalgic encephalomyelitis/chronic fatigue syndrome has identified eight genetic loci that are significantly associated with the chronic debilitating condition.

Subtyping is what made precision medicine in cancer a reality. And for successful drug discovery in all its stages, finding subtypes in Alzheimer’s disease is all but imperative. Prior to the approval of the modestly effective Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd.), Kisunla (donanemab, Eli Lilly and Co.), and the since-withdrawn Aduhelm (aducanumab, Biogen Inc./Eisai Co. Ltd.), more than a dozen failed phase III clinical trials were all that amyloid-targeting drugs had to show for themselves for decades of effort.

Deep learning tools for protein design can also be used to create molecules that bind to them. Certain peptides, such as intrinsically disordered proteins (IDPs), are challenging to target due to their variable nature. However, scientists from the lab of Nobel laureate David Baker have developed a method to generate binders for IDPs by searching the world’s largest protein database with their AI-powered tool RFdiffusion.

Chinese scientists have discovered a common mechanism by which structurally distinct proteins elicit an allergic reaction, showing they cause the formation of pores in epithelial airway cells. That allows calcium ions to enter and trigger a type II immune cascade, which results in the release of histamine from mast cells.

Around 10 million people globally live with the life-threatening human T-cell lymphotropic virus type-1 (HTLV-1), yet it remains a poorly understood disease that currently has no preventative treatments and no cure. That could soon change after Australian researchers discovered that existing HIV drugs can suppress transmission of the HTLV-1 virus in mice.

At the 2025 Alzheimer’s Association International Conference (AAIC), one of the bigger splashes was made by a cardiovascular drug. In a presentation on July 30, Newamsterdam Pharma Co. NV presented data showing that its cholesterol drug obicetrapib lowered levels of the Alzheimer’s Disease biomarker p-tau217.

An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.