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.

The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

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).

A team of U.S. and South Korean researchers have developed an AI model called MSI-SEER that can not only predict microsatellite instability-high (MSI-H) tumors based on tissue slides, but also flag “what it does not know.” “Have you ever asked ChatGPT anything, and the response was, ‘I don’t know?’” Cheong Jae-ho asked during an interview with BioWorld. “Probably not, and that is the problem with AI now.”

The Nobel Committee announced today that it has awarded the 2025 Nobel Prize in Physiology or Medicine to three scientists for their discovery of regulatory T cells, which are a critical part of the way the body prevents autoimmune attacks.

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.

The Human Cell Atlas project has delivered a fresh tranche of data mapping fibroblasts in healthy and diseased skin and pointing to drug targets with potential in multiple diseases across a range of tissues. Using single cell sequencing and spatial genomics, a technique for showing how gene expression varies at different locations within a tissue, nine different subpopulations of fibroblasts were identified, six in healthy skin and three in disease samples.

The Royal College of Psychiatrists in the U.K. has published its first-ever guidance to support research into psychedelic drugs as therapies for conditions including treatment-resistant depression, substance abuse disorders and posttraumatic stress disorder, saying that in a fast-moving field there is a risk of jumping ahead of the evidence.

A new generative AI model trained on UK Biobank data can simultaneously forecast the risks and timing of developing over 1,000 different diseases a decade into the future. The developers applied similar algorithmic concepts to those used to develop large language models like ChatGPT and Gemini to build the model, using medical records from 402,799 participants in UK Biobank.