A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them.

Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.

By transplanting a pig kidney into a brain-dead person, researchers have been able to conduct the first long-term study of the physiological processes occurring in both the transplant recipient and the pig organ for 61 days. The findings were published in the Nov. 14, 2025, issue of Nature in two papers – one focusing on physiological and immunological measurements, the other on multiomics.

The U.K. government has published a road map for phasing out animal testing in life sciences research and announced £75 million (US$98.6 million) for work to develop nonanimal models, leaving scientists concerned because they say, in many cases, there can never be meaningful alternatives to using live animals.

Researchers at Monash University in Melbourne, Australia, have uncovered the hidden code governing how genetic mutations affect RNA splicing and result in disease. The researchers were able to identify the specific mutations that cause changes in RNA splicing, Sureshkumar Balasubramanian, the lead researcher at Monash University’s School of Biological Sciences, told BioWorld.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

The 2025 Nobel Prize in Chemistry “is a story full of holes, but with enormous capacity to absorb all your attention,” Heiner Linke told reporters. “And other things.” Linke is Chair of the Nobel Committee for Chemistry. On Oct. 8, 2025, the committee announced that it has awarded the 2025 Nobel Prize in Chemistry to Susumu Kitagawa, Richard Robson and Omar Yaghi “for the development of metal-organic frameworks” (MOFs).

Pharma companies are collaborating to boost the power of artificial intelligence (AI) in drug discovery by allowing access to proprietary structural data to train a large language model. Each of the partners is contributing data from several thousand experimentally determined protein:ligand interactions, creating one of the most diverse datasets and the richest chemistry assembled to date for model training.