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.
Researchers at the Fred Hutchinson Cancer Research Center found that autoantibodies targeting the exoproteome reshaped checkpoint inhibitor responses and opened new avenues to enhance immunotherapy. In the study published in the July 23, 2025, issue of Nature, the authors set out to address a long-standing question in cancer immunotherapy: why patients with the same type of cancer, treated with the same immunotherapy, can experience such drastically different outcomes.
Current treatments for Alzheimer’s disease have limited effects. While they can slow cognitive decline or alleviate symptoms, they do not reverse this complex neurodegenerative condition caused by multiple factors. Researchers from the Gladstone Institutes and the University of California, San Francisco (UCSF) have screened FDA-approved drugs in search of agents that could potentially modify the disease.
The human genome has yielded another round of secrets with the publication of two back-to-back papers in Nature on July 23, 2025. Both studies re-sequenced probands from the open-access 1000 Genomes Project, which was one of the first projects to sequence individuals from diverse populations. While one paper “goes very deep and tries to reconstruct a few genomes to basically near completion,” the other specifically looked at structural variants in a larger number of genomes. Together, they give new insights into genome variation.
We all look different to HIV, a virus that destroys the immune system. The defensive cells record every interaction with foreign agents, infections from viruses and bacteria, but also with mechanisms occurring within the body, such as microbiome metabolism, the effects of aging, or the development of diseases. At a preconference session at the 13th IAS Conference on HIV Science (IAS 2025), scientists explained the interactions of different microorganisms with HIV.
There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done?
After two decades of research elucidating the basic science, Tafalgie Therapeutics SA has delivered the first clinical data for its non-opioid analgesic.
Scientists at Newcastle University U.K., have reported the births of eight healthy babies following mitochondrial transfer, in which the fertilized egg of a woman carrying mutations in their mitochondrial DNA was placed in the enucleated egg of a non-carrier.
After a 10-year project and a £60 million (US$80 million) investment, the UK Biobank has completed the whole body scans of 100,000 volunteers and is making the 1 billion images available for researchers worldwide.