NEW LEADERSHIP AT TIGEM: ALBERTO AURICCHIO APPOINTED AS DIRECTOR AT THE TELETHON INSTITUTE OF GENETICS AND MEDICINE IN POZZUOLI (NA)

Pozzuoli (NA), 15 April 2024 – After 30 years, the leadership of TIGEM, the Telethon Institute of Genetics and Medicine in Pozzuoli, Italy, is changing: Andrea Ballabio is passing the torch to Alberto Auricchio, the former coordinator of the institute's Molecular Therapy program. TIGEM was founded by Telethon in 1994, with the mission of studying the mechanisms underlying rare genetic diseases and developing innovative therapies. The decision was made following a selection process that was open to scientists worldwide and conducted by a committee of six scientists of international stature.

Alberto Auricchio, full professor of Medical Genetics at the Federico II University of Naples, is set to inherit an important legacy of excellence in the field of rare genetic disease research. Notably, TIGEM, which also took part in the Human Genome Project, has identified numerous genes responsible for genetic diseases and, since 2016, has been at the forefront of programs in Italy focused on undiagnosed diseases. TIGEM’s scientific contributions extend to precision medicine, particularly in the realm of gene therapy for conditions affecting vision and metabolism. The institute’s quality is further affirmed by its industrial agreements and successful spin-offs.  Among these, AAVAntgarde Bio, founded by Auricchio himself, is dedicated to the developing new gene therapy platforms for hereditary forms of blindness. These platforms aim to overcome a key limitation: the “loading capacity” of viral vector transport.

Alberto Auricchio’s appointment represents a continuation of TIGEM’s impactful work, and his expertise will undoubtedly shape the future of genetic research and therapeutic advancements.

"I am honored to receive this significant appointment and I thank Fondazione Telethon for the trust placed in me. I thank, also on behalf of the Institute, Andrea Ballabio for leading TIGEM for the last 30 years, elevating it to the level of excellence we all recognize”, said the newly appointed director, Alberto Auricchio. “My background is strongly oriented towards translational research, and I aim to foster this focus at the institute in the years to come. Balancing TIGEM’s high scientific standards across both its fundamental and translational aspects promise to be an exciting and challenging endeavor. I know I can rely on the support of the Institute and an ecosystem that includes Fondazione Telethon, its spin-offs, universities with their affiliated hospitals, the CNR and local authorities, collaborative connections that have been fruitful over the years. Therefore, I am convinced that by working together, we will succeed in achieving the ambitious goal of translating our discoveries into treatments for patients with rare genetic diseases”.

Andrea Ballabio, full professor of medical genetics at the Federico II University of Naples and visiting professor at Baylor College of Medicine in Houston, USA, has directed TIGEM since its foundation. His group is credited for the discovery of an important mechanism that allows cells to dispose toxic substances, which is very relevant to the development of therapeutic approaches for genetic storage diseases and for some forms of cancer. For this discovery he received the 2016 Louis-Jeantet Prize for Medicine, a prestigious award given annually to outstanding researchers in Europe.

" One day, back in 1994, I received a phone call that changed my life. I was in Houston, Texas, where I was running a research laboratory in the world's largest Medical Center. Calling me was Mrs. Susanna Agnelli to propose, on behalf of the Fondazione Telethon, that I return to my country to start a new research institute. Accepting the opportunity, I returned to Italy along with a small group of young researchers in love with their work to bring TIGEM to life – recalled Andrea Ballabio “Thirty years later, I can say that accepting that offer was one of the best decisions I have ever made. I sincerely thank Fondazione Telethon and everyone who helped us build a top-notch research center in our country. Today I am truly happy to pass on the torch to Alberto Auricchio, a scientist of the highest professionalism, who will certainly make TIGEM achieve even greater goals in the fight against genetic diseases". 

Excellence, which is widely recognized internationally characterizes both Andrea Ballabio and Alberto Auricchio. In 2023, they achieved a remarkable feat by securing funding from the European Research Council (ERC) for the third time. Out of all researchers who participate in major ERC calls (including starting, consolidator, and advanced grants), only 10 percent receive funding each year. Within this select group, less than 1 percent have ever managed to secure three grants. This achievement not only signifies significant professional recognition for the winners, but also serves as a testament to the Fondazione Telethon’s ability to attract valuable researchers to its institutes.

“TIGEM in Pozzuoli stands as an extraordinary organization, spearheading cutting-edge research on rare genetic diseases. The arrival of a figure like Professor Auricchio, who has devoted much of his life to scientific research, to its leadership position represents the optimal path toward ensuring a sustainable future for our country and the Southern Regions – said the President of Fondazione Telethon, Luca Cordero di Montezemolo. I would like to personally extend my gratitude to Professor Ballabio for his exceptional efforts since the institute’s inception. His monumental achievements have significantly shaped the institute’s trajectory. With the new director at the helm, I am confident that the institute will continue to attract the finest Italian and international scientists. This achievement fills us with immense pride.

There are currently 26 research groups working at TIGEM, 269 people including researchers, research support staff, scientific and administrative offices. In total, Telethon's investment to date amounts to 119 million euros, in addition to the important external funding obtained thanks to the excellence of its researchers, such as those from the ERC (16 of those obtained from 2007 to 2022, 8 of which are currently in progress: for a total funding of more than 16 million euros).

“I express my deepest gratitude to Andrea Ballabio on his remarkable 30-year tenure at TIGEM. Under his visionary leadership, the research center has evolved into a preeminent institute that fills us all with pride. It serves as a vibrant hub, attracting researchers from across the globe.” Commented Francesca Pasinelli, Managing director of Fondazione Telethon. The unwavering dedication of our researchers, coupled with the generous support of donors, has yielded significant breakthroughs for individuals affected by rare diseases. Moreover, these collective efforts have contributed to scientific progress on a broader scale. As we turn the page on the past three decades, I am filled with immense pride. The institute’s history, richly written during this time, will now embrace a new chapter under the capable leadership of Alberto Auricchio.

TIGEM, which has been based in the former Olivetti complex in Pozzuoli since 2013, also deserves recognition for its role in attracting Italian researchers who dedicate their knowledge and expertise to studying rare genetic diseases within the institute. This contribution not only impacts scientific advancement but also plays a crucial role in terms of employment and regional redevelopment.

About Fondazione Telethon ETS                                                                                                                        

Fondazione Telethon is one of the main Italian biomedical charities, founded in 1990 on the initiative of a group of patients suffering from muscular dystrophy. Its mission is to achieve the cure of rare genetic diseases through scientific research of excellence, selected according to the best practices shared internationally. Through a unique method in the Italian panorama, it follows the entire "research chain" dealing with fundraising, selection and funding of projects and the research activity itself carried out in the centers and laboratories of the Foundation. Telethon also develops collaborations with public health institutions and pharmaceutical industries to translate the results of research into therapies accessible to patients. Since its foundation, it has invested over 660 million euros in research, funded 2,960 projects with 1,720 researchers involved and studied 630 diseases. To date, thanks to Fondazione Telethon, the world's first stem cell gene therapy has been made available, born thanks to the collaboration with the pharmaceutical industry and intended for the treatment of ADA-SCID, a severe immunodeficiency that impairs the body's defenses from birth. In 2023, Fondazione Telethon became responsible for the production and distribution of the drug in the European Union. Another gene therapy from Telethon research that has become available is for a serious neurodegenerative disease, metachromatic leukodystrophy. This therapeutic approach is at an advanced stage of clinical trials for another immunodeficiency, Wiskott-Aldrich syndrome, for. beta-thalassaemia and some metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, targeted therapeutic strategies for other genetic diseases, such as certain hereditary eyesight defects, are also at an advanced stage of study or development within the Telethon institutes. In parallel, the study of basic mechanisms and potential therapeutic approaches for as yet unanswered diseases continues in all Telethon-funded laboratories.