The COVID-19 pandemic presented many new dilemmas to medical science, some of which will take years to be fully understood — and learned from. But one truth was laid bare by the end of 2020, as vaccinations against the virus were being rolled out: there is so much more that we could do to speed up the translation of research findings into clinical applications. Less than a year after the World Health Organisation had declared a pandemic, the first shots were available, initially with an emergency authorization and then, a few months later, with full regulatory approval. Those vaccines were based on a new vaccine technology, mRNA, that had been in development in laboratories for decades, but had never reached the clinic because of technical challenges that seemed too hard to overcome, and because the market was not deemed to be big enough. It took unprecedented public and private investments, a new model of collaboration between academia, big industry and biotech, and some regulatory changes to deliver a new therapy that not only saved millions of lives during the pandemic, but that is now being applied to other therapeutic areas.

Medical research is producing new knowledge and technologies at an amazing pace, also thanks to the rise of big data and computational methods that help researchers extract knowledge from genes, chemical libraries, human population. There may be dozen discoveries in labs worldwide that could have a huge impact, but need an extra push to bridge the gap between bench and bedside. And many of them may well be in Italy, a country that already has an amazing track record in developing medical innovation: four out of seventeen advanced therapies (an umbrella term covering gene therapy, stem cells, tissue engineering) authorised to date in the European Union came from Italian academic research.

Where will the next breakthrough come from? How can we make sure that its potential is not lost? Can we replicate for other therapies the model that gave us mRNA vaccines? These questions were at the core of Future trends in translational medicine, the conference that Nature Italy co-organized with Human Technopole in Milan in October 2023.

The conference explored four hot areas of current medical research that have the potential to translate into new therapies. The first one was RNA therapies – including mRNA, antisense and interfering RNA – and gene therapy: both are the result of decades of research in the human genome, and could be applied to many diseases, including rare ones for which there is currently no cure. A second area was about data science and genomics, the application of computational methods to make sense of the trove of data made available by genome sequencing technologies that keep becoming more efficient and more accessible. Then came computational drug discovery, that aims to leverage the recent spectacular advances in machine learning to shorten the time needed to develop new drugs, by searching for associations between candidate molecules and their molecular targets. Finally, a session on organoids, tri-dimensional structure created by stem cells, that reproduce key features of human tissues and are emerging as a crucial platform for personalized medicine, and for studying organ development, cancer, and reaction to drugs.

The conference report covers the presentations from a diverse group of academic and industry researchers from institutions in the United Kingdom, the United States, and Europe.

In this accompanying collection we have gathered articles from Nature Italy and from various journals in the Nature family that report on recent scientific advancements in the research fields covered by the conference, as well as on funding and regulatory obstacles that are preventing some of those innovation from reaching all the patients that could benefit from them.

Overall, the report and the collection highlight the great potential of new methods and technologies in medicine, but they also sound a wake-up call for funders, regulators and the industry, on the barriers that need to be overcome to realise that potential, in particular in Italy, a country that keeps producing great results but where a more systematic effort on technology transfer is very much needed.