Turning scientific discoveries into real-world therapies was the focus of the second international conference “Future Trends in Translational Medicine – From Molecules to Therapies,” held at the Complesso Universitario di San Giovanni a Teduccio in Naples on 30 -31 October 2025. Organised by Human Technopole together with its Centre for Innovation and Technology Transfer (CITT) in collaboration with Nature Italy and with patronage of Regione Campania, the event gathered international researchers, clinicians, and industry representatives to explore how discoveries in molecular biology, genetics, and pharmacology can be translated into concrete clinical applications.
The conference was centred around four pressing themes – metabolism, cancer, rare genetic disorders, and gene therapy and RNA therapeutics – each representing a frontier where unmet medical needs intersect with new therapeutic opportunities. With two keynote lectures and a wide range of cutting-edge presentations, the event offered a snapshot of how basic science can have an impact on people health and well-being. Crucially, a fil rouge was established across themes to highlight the entire translational journey, from basic molecular discoveries to the development of targeted therapies, thus vividly reflecting the overarching goal of the conference.
Keynote Highlights
The conference began with an engaging keynote lecture by Dr. Richard Finn, Professor at UCLA, whose seminal work on CDK4/6 inhibitors has transformed the treatment landscape for breast cancer patients. He traced the journey from generalised, “one-size-fits-all” cancer therapies to the era of precision oncology, where treatments are tailored based on tumour biology and genetic markers. The concept is that a complex disease like cancer can have different molecular origins and manifests differently depending on each individual’s genetic and environmental factors. Therefore, not all patients can benefit from the same therapy; patients must be selectively targeted based on their predicted ability to respond to the therapy. As Dr. Finn reminded the audience, precision medicine also brings new challenges: identifying reliable biomarkers, designing smarter clinical trials, and ensuring equitable access to advanced therapies. “The challenge with targeted therapy,” he noted, “is getting the right drug to the right patient.”
On the second day, Dr. Stefanie Dimmeler, Director of the Institute for Cardiovascular Regeneration at Goethe University Frankfurt, delivered a captivating keynote on ageing and cardiovascular disease. Her talk emphasised that vascular ageing, inflammation,and clonal haematopoiesis are active contributors to disease rather than merely age-related phenomena. Small molecules known as senolytics, which kill senescent cells, are showing early promise in restoring cardiac function and reducing inflammation. These insights offer a glimpse into the development of anti-aging strategies that might soon become part of cardiovascular care, especially in elderly populations.
Reframing Metabolism: From Fuel to Signalling
The first major thematic session delved into metabolism, beginning with Dr. Heidi McBride, Professor at McGill University, who calls for a complete rethink of how we view mitochondria. Traditionally seen as the cell’s energy factories, mitochondria are now emerging as key regulators of immunity, signalling,and cellular decision-making. Future therapies may indeed involve targeting inter-organelle communication and mitochondrial dynamics, especially in diseases like cancer and neurodegeneration.
Next, Dr. Shingo Kajimura, Professor at the Howard Hughes Medical Institute (HHMI), addressed the problem of weight rebound after GLP-1 receptor agonist therapy, drugs that have recently transformed obesity treatment. His lab has identified calcium cycling as a promising target for sustaining energy expenditure, independent of appetite suppression. By studying ATP-wasting processes and the underlying molecular events, he hopes that it will be possible to develop therapies that prevent fat regain while preserving muscle mass, a crucial step in maintaining long-term metabolic health. Dr. Sadaf Farooqi, Professor at the University of Cambridge (UK), tackled the complex genetics of severe obesity, a disease often thought to be primarily rooted in lifestyle. She showed how rare mutations in genes encoding regulators of energy homeostasis and appetite, can lead to early-onset obesity. These discoveries have led to targeted treatments that address the original cause, offering hope to patients who often face not only medical challenges but also social stigma.
Rethinking Cancer: Targeting the Moving Parts
The cancer session featured a diverse lineup of speakers addressing new advances in molecular processes and clinical strategies. Dr. Celeste Simon, Associate Director at the University of Pennsylvania, opened with a discussion on clear cell renal cell carcinoma (ccRCC), a type of kidney cancer that shows unique metabolic dependencies, particularly on cholesterol and bile acids. Targeting these pathways through inhibition of their molecular regulators offers a new route to weaken this class of tumours, enhance immune response, and bypass resistance mechanisms.
Challenging the focus on genetic mutations, Dr. Pier Giuseppe Pelicci, Director of Research at the European Institute of Oncology (IEO) in Milan, explored the plasticity of cancer cells and how non-genetic adaptation contributes to chemoresistance and metastasis. His research on interferon signalling in leukaemia cells suggests that restoring immune pathways could re-sensitise tumours to therapy. This dynamic view of the immune-surveillance of cancer offers a new perspective vs. the traditional mutation-based models.
On the cutting edge of immunotherapy, Dr. Alice Giustacchini, Research Group Leader at Human Technopole, presented a novel approach to treating paediatric acute myeloid leukaemia (pAML) based on single cell multi-omic profiling. Her team identified leukaemia stem cell (LSC)-specific markers, paving the way for safer and more effective dual CAR T-cell therapies. Her work exemplifies how research based on high-resolution cellular analysis can guide precision immunotherapy, particularly for vulnerable paediatric populations.
Rare Genetic Disorders: From Incurable to Treatable
The second day’s thematic sessions began with a focus on rare genetic diseases, long considered too complex or unprofitable for pharmaceutical development. But as Dr. Alberto Auricchio, Scientific Director of the Telethon Institute of Genetics and Medicine (TIGEM) in Naples, demonstrated, this narrative is now changing. His group pioneered dual Adeno-associated viruses (AAV)-based gene therapies for genetic disorders like Stargardt disease and Usher syndrome, successfully overcoming challenges in gene size and delivery. The pre-clinical and early clinical studies suggest that these once-untreatable conditions may soon have durable therapies.
Dr. Alessandro Aiuti, Deputy Director of San Raffaele Telethon Institute for Gene Therapy (TIGET), provided a retrospective on hematopoietic stem cell gene therapies (HSC-GT), which have revolutionised treatment for conditions like ADA-SCID, metachromatic leukodystrophy (MLD), and Wiskott-Aldrich syndrome (WAS). These “once-and-done” therapies use a patient’s own gene-corrected cells, offering a durable cure. But as Dr. Aiuti noted, the challenges are not just scientific: they are regulatory, logistical, and ethical, especially in ensuring equitable access to transformative treatments.
Complementing this effort, Dr. Nicole Soranzo, Head of Genomics Research Centre – Population & Medical Genomics at Human Technopole, presented findings from the Moli-sani study, a large-scale population genomics project involving individuals in Southern Italy. By integrating polygenic risk scores and functional genomics, the study reveals how even small, rare genetic variations can influence disease risk. These insights support early detection, personalized prevention, and the refinement of treatment strategies, especially when applied to specific regional populations with unique genetic backgrounds.
Gene Therapy and RNA Therapeutics: A Glimpse into the Future
The final session was a tour de force on the future of genetic and RNA-based medicines. Dr. Guangping Gao, Professor and Chair at the University of Massachusetts, continued along the line of Dr. Alberto Auricchio’s presentation, and showed how rational design of AAV vectors, from capsid engineering to tissue targeting, can dramatically improve the precision of in vivo gene therapy. His work bridges preclinical success in animals to human trials, including promising treatments for Canavan disease and maple syrup urine disease.
Dr. Katie Whitehead, Professor at Carnegie Mellon University, continued with an overview of lipid nanoparticles (LNPs), the delivery vehicles that enabled mRNA vaccines and are now being adapted for other organs beyond the liver. Her team has expanded the reach of RNA-based therapies to the lungs, pancreas, and brain, opening doors to new treatments in cancer, neurodegeneration, and gene editing.
Rounding out the session, Dr. Laura Sepp-Lorenzino, Special Advisor and Former CSO of Intellia Therapeutics, Inc., emphasised the broader potential of nucleic acid therapies, from oligonucleotides to CRISPR-based genome editing, as tools to address previously “undruggable” diseases. With platforms in place for rapid development and delivery, she described a future where one-time, curative interventions could become a clinical reality for a wide range of conditions.
Closing Reflections: A Model for Impact
The final remarks, delivered by Human Technopole Board Member and Former General Director of Telethon Foundation Dr. Francesca Pasinelli, who offered a powerful reminder that translating science into therapies also requires infrastructure, strategy,and vision. She outlined how the Telethon Foundation has developed a unique mission-driven, end-to-end translational model, from research funding to clinical trials and even manufacturing, to ensure patients still receive life-saving therapies. This was an outstanding example of how basic research in Italy can led to the development of therapeutics even when companies have no financial interest in producing them.
Conclusion
The Naples conference captured a vibrant moment in the evolution of translational medicine, propelled by conceptual advances and powerful technological developments. Across topics as diverse as mitochondrial signalling, cancer immunotherapy, rare disease genomics, and RNA-based therapeutics, a single theme emerged: the journey “from molecules to therapies” is no longer a distant ideal but a present-day reality. However, turning molecular discoveries into practical treatments is no easy feat. It requires creativity, collaboration, regulatory finesse, and a deep sense of responsibility to patients. This conference not only showcased scientific progress but also inspired the collective effort needed to make the future of medicine equitable, effective, and inclusive.
