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San Raffaele wins three ERC Consolidator Grants 2024

European Research Council (ERC) has announced the winners of the Consolidator Grant 2024: among them, winner for the IRCCS San Raffaele Hospital is Dr. Daniela Cesana, leader of the Tissue dynamics and biomarker signature discovery program of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget); for Vita-Salute San Raffaele University (UniSR), the winners are Dr. Alessio Cantore, UniSR researcher and Group Leader in the Liver Gene Therapy Unit of SR-Tiget, and Dr. Daniela Latorre, currently leading the Human Neuroimmunology group at ETH Zurich.

This year, the ERC evaluated 2313 proposals, selecting 328 of them for a total amount of € 678 million. Of these, 94 are dedicated to research in the Life Sciences fields. Italy wins 20 ERC Consolidator Grants, in the three domains Life Sciences, Physical Sciences and Engineering, and Social Sciences and Humanities. In the field of Life Sciences, 4 projects have been funded in Italy, 3 of which were awarded to San Raffaele.

The prestigious ERC grants support, with up to 2 million euros for five years, innovative research in different disciplines, with scientific excellence as the only selection criterion. In particular, ERC Consolidator Grants are designed to support excellent researchers at the stage of their career when they are still consolidating their team or independent research program.

San Raffaele continues to to achieve remarkable successes: these three Consolidator Grants join the three Starting Grants won in September, totalling 34 ERC grants awarded since 2007, the year the programme was born. These successes not only highlight the quality and originality of the research conducted, but also demonstrate our ability to compete internationally, promoting innovative ideas and addressing frontier scientific challenges.

The TalesfromDeath project

Gene therapy is revolutionizing the treatment of inherited and acquired diseases by leveraging engineered cells to restore normal bodily functions. Liquid biopsies, performed with a simple blood draw, are increasingly recognized as a powerful, non-invasive approach for detecting dynamic changes in patients caused by disease progression or responses to specific treatments. With the "TalesfromDeath" project, won for the IRCCS Ospedale San Raffaele, Daniela Cesana will exploit liquid biopsies, to analyse extracellular DNA to monitor biological events throughout the body, both before and after treatment with gene therapy. We will thus obtain detailed information on the effectiveness of gene therapy in restoring physiological functions in patients with genetic diseases and tumors, offering us the possibility to predict clinical outcomes. These findings could lead to more effective treatments and personalized therapies, improving patient care.

Dr. Cesana states:

I am honored to receive this prestigious ERC grant award, a recognition that fills me with immense pride and joy. This achievement reflects years of dedication, perseverance, and passion for my work. The journey has not been without challenges, but every obstacle has been a valuable lesson, shaping me both personally and professionally. I share this moment with all those who have supported and inspired me along the way. Above all, my family, whose unwavering care and love have been my greatest strength, as well as my colleagues and mentors. Their encouragement has been a driving force behind my efforts. This award motivates me to continue striving for excellence and contributing meaningfully to my field. I am humbled and will cherish this milestone as I move forward with renewed determination.”

The “HEPAGENE” project

The genetic modification of hepatocytes, the main liver cells, can potentially cure many severe and rare genetic diseases of liver metabolism. Until now, this goal has been obtained in adult patients affected by certain genetic diseases by using techniques that do not allow the genetic modifications to be maintained following cell proliferation, such as during the growth of the liver, and thus cannot be applied to pediatric patients. On the other hand, other genetic engineering technologies are available to provide stable genetic correction even following cell proliferation, as they modify the cellular genome, and the modification is passed over to the daughter cells. Alessio Cantore’s group has contributed to developing some of them. However, they are at an early stage of development and have not yet been tested in humans. With the HEPAGENE project, Dr. Cantore’s group will focus on the process of post-natal liver growth and maturation at the cellular and molecular level, assessing the efficacy, safety and stability of different genetic engineering strategies when applied during the early stage of life. The ultimate goal of the project is to develop new genetic correction treatments for pediatric patients with the potential to become definitive cures for severe genetic diseases of liver metabolism.

Dr. Cantore states:

“Receiving this prestigious grant not only validates my passion and dedication to my field but also provides invaluable resources to expand my impact and accelerate my professional growth and ability to contribute to the broader community. I will do my best to deserve this thrust and carry out the proposed project to expand our understanding of liver biology and potentially deliver novel gene therapies to children affected by rare genetic diseases of liver metabolism.”

The AUTO-T-NERVES project

Auto-immune diseases affect people of all ages and can damage various organs including the nervous system. In the latter case, myelin, those sheaths that insulate nerves and allow them to function, is one of the main targets. Among the autoimmune myelin diseases, Daniela Latorre's AUTO-T-NERVES project, won with the Vita-Salute San Raffaele University, deals with diseases affecting peripheral nerves, so-called peripheral neuropathies. These are precisely autoimmune diseases, possibly triggered by an infection and sustained by a florid inflammation, which, although relatively infrequent, can become severe and disabling, as they are characterised by progressive muscle weakness that can even lead to complete paralysis in the most severe cases. However, the mechanisms underlying these diseases remain unclear to this day and this severely limits the possibility we have of predicting both the course of the disease and the effectiveness of the treatments used and usable. The AUTO-T-NERVES projects stems from recent findings by Dr. Latorre’s lab, which revealed the involvement of a type of immune cells, T lymphocytes, in a subset of patients. Dr. Latorre, who will run her own laboratory thanks to funding from the prestigious Career Development Award granted by the Armenise-Harvard Foundation, will use advanced experimental tools to systematically investigate the involvement of these cells in different subtypes of inflammatory peripheral neuropathies. This research aims to deepen our understanding of the mechanisms underlying these distinct disease variants and pave the way for more targeted, subtype-specific treatments.

Dr Latorre states:

“I am deeply honoured and grateful for this prestigious award. Since childhood, my passion for science has been a constant in my life, leading me to research experiences both in Italy and abroad, which allowed me to enrich my skills and broaden my perspectives. These experiences have not only taught me to develop critical thinking but also to trust my intuitions. This funding, which joins that obtained by the Armenise-Harvard Foundation, will enable me to consolidate my scientific career, while continuing and deepening my work on diseases of the nervous system, a line of research that I initiated a few years ago and has recently led to significant discoveries. I am convinced that UniSR represents the ideal environment to continue this research, providing close integration between basic and translational research. My special thanks go to my parents, who have always believed in me, and to all the colleague scientists and collaborators who continue to support my work.”

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Daniela Cesana

Daniela Cesana, Ph.D. in Cellular and Molecular Biology (2010), specializes in gene therapy safety, developing preclinical models to assess oncogenic risks of integrative vectors. Her 2016 research identified how HIV-1 disrupts host gene transcription to persist. As Project Leader (2019) and now Program Leader at SR-Tiget (since 2023), she advances safety and efficacy technologies for gene therapy. With 30+ peer-reviewed publications, 2 patents, and numerous grants, she received the 2019 Excellence in Research Award from the American Society for Cell and Gene Therapy, focusing on vector genotoxicity and HIV persistence.

Alessio Cantore

Alessio Cantore, Ph.D., specializes in liver-directed gene therapy, focusing on improving the safety and efficacy of lentiviral vectors. He completed his Ph.D. in Cellular and Molecular Biology in 2013 at the Open University, London, and Vita-Salute San Raffaele University, under the mentorship of prominent researchers. He has authored 19 research articles and 6 reviews, holds 12 patents, and received multiple awards, including the 2018 Young Investigator Award and Excellence in Research Awards (2019, 2023, 2024). Since 2017, he has been an Assistant Professor of Histology and a Group Leader at SR-Tiget. He also co-founded GeneSpire, a start-up focused on gene therapies for inherited liver diseases.

Daniela Latorre

Daniela Latorre is specialized in human immunology. She obtained her PhD in Immunology at "Sapienza" University of Rome and then moved to Switzerland for postdoctoral training in Prof. Federica Sallusto’s laboratory. Since 2020, supported by various national and international career research grants and awards, including the Swiss National Foundation, she leads the Human Neuroimmunology group at ETH Zurich, focusing on T cell immunity in immune-mediated neurological diseases.