Tigem

We are delighted to share the upcoming International Mammalian Synthetic Biology Workshop (mSBW2025)—the only conference fully dedicated to the fast-growing field of mammalian synthetic biology organized by AIChE - American Institute of Chemical Engineers Mammalian synthetic biology is reshaping how we think about gene regulation, cell therapies, and disease modeling. By designing synthetic circuits—engineered gene networks that precisely control cellular behavior—researchers are unlocking powerful tools with real-world impact in biomedicine. The mSBW is the only scientific event dedicated exclusively to this exciting area, bringing together experts to discuss breakthroughs that are driving innovation from bench to bedside. This year’s edition promises cutting-edge science, visionary applications, and a vibrant community pushing the limits of biology, engineering, and medicine. 📍 August 11–12, 2025 📌 Beckman Center, Irvine, California   Don’t miss the opportunity to be part of this essential conversation and to meet Diego di Bernardo, Coordinator of the Genomic Medicine Program at TIGEM and within this year Workshop Organizing Committee. 🗓️ Call for Abstracts open until April 28, 2025 👉 Submit & Register here: https://lnkd.in/ea2udmd6 #SyntheticBiology #mSBW2025 #MammalianCells #GeneCircuits #AIChE #Biotech #SystemsBiology #GenomicMedicine

🧠💪 Today is the International Pompe Disease Day — a time to raise awareness, honor patient stories, and reflect on the progress made in the fight against this complex genetic disorder. At TIGEM, with the support of Fondazione Telethon, we draw on a multidisciplinary approach to tackle Pompe disease from every angle. By uniting expertise in genetics, molecular biology, clinical research, and patient advocacy, we: • Identify novel biomarkers via OMICS and clinical data integration • Gain groundbreaking insights into lysosomal biology, building on discoveries around TFEB and the CLEAR network • Conduct pioneering studies on chaperone-based therapies to enhance enzyme replacement therapy (ERT) • Design innovative systemic gene therapy strategies that target not only muscles but also the central nervous system and other affected organs It all began with science — and a powerful act of compassion. In 2002, long before access to treatment was widespread, a small amount of the first experimental enzyme replacement therapy (ERT) reached a little girl in Naples, Rossella, thanks to a heartfelt connection between her physician, Dr. giancarlo parenti, Senior Principal Investigator at TIGEM, and John F. Crowley, then CEO of Amicus Therapeutics and father of two children with Pompe disease. That moment of empathy and action sparked a journey that continues today — one built on collaboration, care, and the belief that research can change lives. 📖 Read the full story here: 👉https://lnkd.in/dfyGA3DE Alberto Auricchio Diego Luis Medina carmine Settembre Chiara Di Malta Roman Polishchuk #AndreaBallabio Marco Sardiello Nunzia Pastore Antonietta Tarallo Nicolina Cristina Sorrentino Maria Cretella #NicolaBrunettiPierri National Center for Gene Therapy and Drugs based on RNA Technology #PompeDisease #RareDisease #GeneTherapy #ERT #Biomarkers #LysosomalStorageDisorders #TFEB #TranslationalResearch #OMICS #TIGEM #FondazioneTelethon #InternationalPompeDay #HealthcareInnovation #ScienceWithHeart #PNRR

The ESGCT Spring School was held this week, marking a pivotal step in preparing the next generation of leaders in gene and cell therapy. Spearheaded by Alberto Auricchio—President of the European Society of Gene and Cell Therapy (ESGCT) and Director of TIGEM (Telethon Institute of Genetics and Medicine)—this initiative highlights the crucial role of education in driving progress in one of the most dynamic fields in medicine. As more gene therapies reach the market, offering life-changing options for patients with previously untreatable conditions, the need for highly skilled scientists is greater than ever. “Our goal isn’t just to teach,” Auricchio emphasized in his opening remarks. “We want to inspire a new generation of researchers ready to meet the complex challenges of genetic diseases and push the boundaries of what’s possible.” At its core, ESGCT’s mission is about more than just knowledge transfer—it’s about building community. Through immersive training and strategic support, the ESGCT Spring School provides young scientists not only with technical skills but also with invaluable access to a global network of experts across academia, industry, and regulatory sectors. “Our strength lies in connecting emerging talent with the broader research community,” said Auricchio. “It’s in these connections that real innovation happens.” 🌍💡 Maria Cretella Marija Tomovic Mariarosaria Cancelliere Alessandro De Falco Paola Arena Teresa Imperatore Anna Valanzano #GeneTherapy #ESGCT #RareDiseases #NextGenScience #BiotechChallenges #CellTherapy #Innovation #Mentorship #TIGEM #TranslationalResearch #FondazioneTelethon

New findings from Pastore's group, published in Cell Death & Differentiation, reveal an unexpected role for the tumor suppressor gene Folliculin (FLCN) in liver cancer. The study demonstrates that liver-specific deletion of Flcn in mice leads to progressive liver damage and spontaneous development of tumors with features of cholangiocarcinoma, particularly in males. Mechanistically, the work identifies TFE3 as the principal driver of carcinogenesis and mTORC1 hyperactivation in the absence of FLCN, highlighting a non-canonical regulatory pathway of liver tumor development. These results provide new insights into how dysregulation of MiT/TFE transcription factors contributes to hepatic tumorigenesis and identify TFE3 as a potential therapeutic target. 👉 Read the Full article here: https://lnkd.in/dvr-45iC #LiverCancer #mTOR #TFE3 #FLCN #CancerBiology

🌍 World Health Day 2025 | Health for All — Also for Those with Rare Diseases Today, on World Health Day, we are reminded that health is a universal right. But for the over 300 million people worldwide living with a rare disease, that right is often harder to access, and even harder to maintain. At TIGEM, we study rare genetic diseases not only to better understand their mechanisms — but to help build a future in which those affected can live longer, better, and healthier lives. Because health belongs to everyone. Even — and especially — to those facing the daily challenges of conditions that are often invisible, misunderstood, or neglected. 🔬 80% of rare diseases are of genetic origin. 👶70% of rare diseases start in childhood. 🧬 Yet only 5% of rare diseases have an approved treatment to date. This is why our research matters. This is why inclusion matters. This is why, on World Health Day, we stand alongside patients, families, clinicians, and scientists to say: There is no "health for all" without health for people living with rare diseases. #WorldHealthDay2025 #HealthForAll #RareDisease #GeneticResearch #Inclusion #ScienceForHope

🚀 Exciting News! 🚀 We are proud to announce that Andrea Ballabio is part of the prestigious MENTOR Program EU, a cutting-edge doctoral training network dedicated to studying the mTOR signaling pathway—a key regulator of cell growth and metabolism. As a Principal Investigator, Professor Ballabio will contribute his expertise in genetics and lysosomal biology, reinforcing MENTOR’s mission to advance research on rare diseases, cancer, and metabolic syndromes. We look forward to the breakthroughs this initiative will bring! Read an insight here: https://lnkd.in/d_7VtgN4 #mTOR #Genetics #MedicalResearch #MentorProgram #MSCA #Innovation

#TIGEMnewcomer 🔬 From Sweden to Italy: Andrea Pasquadibisceglie’s journey in Computational Structural Biology After gaining international experience at Kungliga Tekniska högskolan (KTH) in Stockholm, Andrea is now back in Italy as a Staff Scientist at TIGEM. His work focuses on designing hyperactive enzymes to enhance gene therapy for rare diseases, using AI and physics-based methods. 💡 "TIGEM gave me the opportunity to return to Italy and make a difference. I want to push the boundaries of research on rare diseases." Exciting times ahead for Andrea and his mission to advance molecular therapies! Read the full interview here: https://lnkd.in/dyrSNKPv #ComputationalBiology #StructuralBiology #TIGEM #Innovation

🎉 We are thrilled to share the success of the latest #HydeParkCoroglio event, featuring our esteemed Prof. Alberto Auricchio, Scientific Director at Tigem in Pozzuoli. Prof. Auricchio delivered an insightful talk, offering an in-depth perspective on #research and #biotechnology, highlighting the immense potential of the field and the challenges we navigate daily. The discussion also delved into key financial strategies related to our research center, showcasing how #technologytransfer can turn groundbreaking scientific discoveries into tangible solutions with a meaningful impact on society. We are proud to have had our Scientific Director as a speaker at this remarkable event and extend our gratitude to everyone who participated, making this gathering a moment of growth and inspiration! #HydeParkCoroglio #Innovation #ScienceAndFinance #Collaboration #Future

🚀 Gene Therapy Between Academic Research and Biotech: A Talk by Alberto Auricchio We are excited to announce an upcoming talk by Alberto Auricchio, a leading expert in the field of academic research and biotechnology innovation. In this session, he will explore a virtuous model of academic research successfully translating into innovative therapeutic solutions. From identifying molecular anomalies to developing breakthrough treatments, his insights will showcase how cutting-edge research is reshaping the landscape of rare genetic disease therapies. Join us to dive into the future of biotech-driven innovation and the power of scientific discovery in improving lives. #Innovation #Biotechnology #AcademicResearch #RareDiseases #Therapeutics #ScienceToSolutions

🚀 From Psoriasis to Lowe Syndrome: A Big Step Forward for Rare Disease Therapeutics Thrilled to share that a Phase II clinical trial for Lowe syndrome—a rare, life-limiting genetic disorder—will soon begin at Ospedale Pediatrico Bambino Gesù, thanks to the new established collaboration between Fondazione Telethon and CanFite BioPharma. 🔍 The molecule at the center of this trial, Piclidenoson, originally developed by Can-Fite BioPharma for psoriasis, was identified as a promising candidate for Lowe syndrome through high-throughput screening and mechanistic studies led by #AntonellaDeMatteis, and Leopoldo Staiano. 🧪 The screening campaign was made possible thanks to High Content Screening Facility at TIGEM, manged by Diego Luis Medina, a core platform enabling automated testing of thousands of compounds in disease-relevant models. 💡 Repurposing existing drugs is a game-changer in rare disease research: it allows us to reduce development time, costs, and risk—critical factors when patient populations are small and options are few. In this case, a drug already in late-stage trials for a common condition is being redirected to meet a profound unmet need. This is a shining example of how basic science, strategic vision, and operational excellence can come together to bring real hope to rare disease patients and their families. 🤝 A special thanks to Annamaria Merico from Fondazione Telethon’s Business Development Office, who played a pivotal role in establishing the agreement with Can-Fite and making this trial possible. Read an insight here: https://lnkd.in/dJnq6FzF   #RareDiseases #DrugRepurposing #TIGEM #FondazioneTelethon #LoweSyndrome #TranslationalResearch #InnovationInBiotech #HighContentScreening #CanFite #ClinicalTrials #LifeSciences