How to ride the new wave of anticancer drug R&D

Hello Nature readers,
This week we learn that prime editing has been used to alter DNA inside a person’s body for the first time, explore the progress of T-cell-based therapies and hear how new technologies and human-derived models have the potential to change anticancer drug discovery.

First use of prime editing inside the body

A powerful gene-editing technique called prime editing has been used to alter DNA directly in a person’s body for the first time. Biotech firm Prime Medicine used the method, which can insert, delete and rewrite segments of DNA, to treat a condition called Wilson disease. The edit targeted a mutation that prevented liver cells from eliminating excess copper. Prime Medicine is one of a slew of companies aiming to engineer cells in the body, which could be cheaper and faster than the conventional method of extracting cells, editing them and reinjecting them. Results from the clinical trial are expected next year.

Is US R&D heading to Australia?

Biotech and pharma companies are increasingly looking to Australia to run clinical studies due to pricing pressures and the volatile research environment in the United States, according to analysis by BioSpace. One Australian contract research organization — which manages research for drug companies — said inquiries for its services increased by about 25% this year compared to 2025. State-based incentives are building on national tax incentives for drug development, but the smaller population and limited manufacturing capacity are still holding the country back from becoming a biotech innovation hub.

Vaccine cuts risk of skin cancer coming back

A personalized mRNA vaccine has reduced the risk of skin cancer returning or the recipient dying by 42% when used alongside an immunotherapy drug. The vaccine, called intismeran, is designed to prevent tumours regrowing in people who’ve had surgery to remove them. A more than 40% risk reduction “would send many researchers over the Moon”, says geneticist Seth Cheetham. Researchers from US vaccine developers Merck and Moderna presented the results at the European Society for Medical Oncology over the weekend, but announced them early after the data leaked online.

A zoom in on T-cell-based therapies

• A reality check for autoimmune CAR-T-cell therapy: Recent trial pauses have revived fears around safety, patient selection and acceptable risk, despite progress in the use of CAR-T cells for autoimmune disease. (Nature Medicine | 11 min read)
• Translation of in vivo chimeric antigen receptor (CAR)-T therapies in China: The coordination of research institutions, industry and hospitals has allowed for rapid progress in the field. (Nature Reviews Bioengineering | 4 min read)
• T cells give cancer the blues: The first ‘ImmunoMemphis in May’ conference brought together 400 participants to discuss recent advances in T-cell immunotherapy. (Nature Immunology | 13 min read)

Features and opinion

Can the system manage gene therapies?

“In this changing landscape, a key question is whether current infrastructure can sustain innovation while ensuring patient access,” write a group of medical researchers about a boom in gene and cell therapies. The market for these medicines is predicted to grow to around US$29 billion by 2035. As these therapies hurtle towards the clinic, academic research centres will need better infrastructure and seamless integration with the rest of the R&D pipeline to take on the riskier, early clinical studies, which drug companies are increasingly outsourcing, argue the authors.

How to ride the new wave of anticancer R&D

New technology holds the power to change our understanding of what anticancer drug discovery avenues are worth exploring and can work hand-in-hand with new human-derived models — such as organoids grown from stem cells — to turbo charge research in the area, argue drug researchers Min Huang, Mei-yu Geng and Jian Ding. The Acta Pharmacologica Sinica editorial brings together 15 reviews that map the evolving landscape of cancer research and drug discovery, including cracking of ‘undruggable’ targets such as KRAS and new drug modalities such as antibody-drug conjugates.

The microbiome and chronic disease

“The microbiome is among the most tractable levers available to address the chronic disease epidemic,” write a group of microbiome scientists. But, while “the science is ready”, not enough is being done to translate this into “coordinated action, infrastructure and implementation”, they write. The group, brought together by the US Department of Health and Human Services, outlines a ten-point to-do list to realize the potential of the human microbiome in biomedical science, including reshaping approval pathways for safe interventions that fall outside current frameworks, such as live biotherapeutics, and prioritizing personalized approaches.

Quote of the day

Neuroscientist Kenneth Kosik discusses the value of testing natural products and existing drugs in people who have blood markers of Alzheimer’s disease but no symptoms, to find treatments that could delay the disease's progression. (Science | 8 min read)

As winter rolls around, I find myself heading to the cinema more and more: I am interested to see how film and science come together in the new documentary about the days leading up to the imprisonment of Elizabeth Holmes, who led the failed blood-testing start-up Theranos.

Please send documentary recommendations, and any other feedback on this newsletter, to [email protected].

Thanks for reading,
Laura Woodrow, editor, Nature Briefing: Translational Research
With contributions by Jacob Smith

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