San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)

🎤In their own words: why I chose the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) Curious what makes SR-Tiget a place where young scientists thrive and why you should join it too for your #PhD?   👇 Here some of our researchers share their personal reasons for joining the Institute and how the experience is shaping their professional growth. Their voices are a direct invitation to ambitious PhD candidates: join SR-Tiget through #RAREFIND, the #MSCA-COFUND programme shaping the next generation of leaders in #genetherapy and #celltherapy for rare diseases.   🌍 Swipe through their stories and apply here www.rarefind-cofund.eu ⏰ Deadline 31/12/2025

🧬 A novel #genetherapy approach for #GloboidCellLeukodystrophy (GLD) GLD is a devastating neurodegenerative lysosomal storage disorder with limited treatment options, which have little efficacy and carry significant risks. Angela Gritti's team recently developed an innovative gene therapy approach for GLD, engineering a chimeric version of the lacking enzyme, designed for improved secretion and delivery to the brain.   🔬 This strategy led to enhanced enzyme production and secretion, effective cross-correction in patient-derived cells, and sustained enzyme activity in vivo, reaching both the central and peripheral nervous systems.   🧠 These findings - recently published in Molecular Therapy with Federica Cascino and Alessandra Ricca as first authors - demonstrate the feasibility and therapeutic potential of this #genetherapy approach for GLD, potentially overcoming key barriers that have long limited treatment for this and related neurodegenerative disorders.   👉 This work also paves the way for next-generation protein engineering strategies to improve treatment delivery to the brain. Read more here https://lnkd.in/dh_pYNsS

We are thrilled to share that today the European Medicines Agency Committee for Medicinal Products for Human Use announced the positive opinion recommending marketing authorisation for Etuvetidigene autotemcel, an ex vivo hematopoietic stem cell #genetherapy for #WiskottAldrichSyndrome (WAS), a rare and life-threatening immunodeficiency, for which Fondazione Telethon serves as the marketing authorisation holder.   This milestone represents the culmination of a pioneering research, clinical and human journey, powered by the dedication of our scientists, clinicians and support teams. Within SR-Tiget unique ecosystem - one of the few worldwide able to embrace the full journey of #ATMP development - we have   🔍Uncovered the mechanisms underlying the immunological defects of WAS 🧬 Developed a lentiviral vector-based gene therapy approach ✅ Demonstrated preclinical proof of concept for safety and efficacy 👩⚕️ Conducted successful clinical trials 🔬 Studied correction of disease mechanisms and vector safety after gene therapy  🌍 Accompanied 34 patients and their families from 18 countries throughout their treatment journey, whose trust and participation made this achievement possible   Fondazione Telethon, the first non-profit organization to have successfully led the full pathway from laboratory research to regulatory approval - collaborating with industry partners when available - has been instrumental in this success, supporting SR-Tiget’s work from the outset and spearheading this regulatory approval.   The therapy will be made available to patients at Ospedale San Raffaele, a recognised center of excellence in gene therapy for this and other diseases, where the clinical trial phase was conducted.   👉 Read the press release here https://lnkd.in/dYeFfvbw

🧬 Cracking the code of peripheral immune tolerance Following the discovery of regulatory T cells (#Tregs) and the mechanisms of peripheral immune tolerance, SR-Tiget scientists contributed to turn fundamental immunology insights into therapeutic innovation. This second article in our three-part series explores how studies on #IPEX syndrome - a rare, severe autoimmune disease - led to the identification of the #FOXP3 gene, the “master switch” of immune regulation. It was from this insight that SR-Tiget pioneered the development of a gene therapy strategy for this disease using lentiviral vectors, opening the way to correcting immune dysregulation at its genetic root. The article also retraces the discovery of rapamycin's potential to promote Treg expansion ex vivo, an approach that continues to serve as a global benchmark. 🔗 Read the full story https://lnkd.in/dFWG9q2y     Did you miss the first part of the story? Find it here https://lnkd.in/dbVHUmVW

Do you want to be at the forefront of translational research, driving cutting-edge advances in #genetherapy?   A Research Fellow position is immediately available under the supervision of Michela Milani - Project Leader in Alessio Cantore's lab - to work on a novel project developing an innovative in vivo lentiviral vector gene therapy strategy for #hemophiliaA.   👉 More details and how to apply here https://lnkd.in/d-J28d_r

🚀We are proud to announce that the AVANTI project, an initiative by Fondazione Telethon involving its two Institutes San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Telethon Institute of Genetics and Medicine (TIGEM), has been selected as one of the three winning projects of the “Per l’Italia del Futuro” call promoted by Fondazione CDP, non-profit entity by Gruppo Cassa Depositi e Prestiti   In collaboration with Ospedale Pediatrico Bambino Gesù and Ospedale Pediatrico Santobono Pausilipon, AVANTI will   🔎Strengthen the Fondazione Telethon Undiagnosed Diseases Program, integrating genomics, transcriptomics, and epigenomics to accelerate gene and mechanism discovery 🔎Functionally assess patient-specific variants with advanced cellular models and high-throughput assays to enable personalized gene therapies 🔎Build an AI-powered multi-omic platform to monitor safety, efficacy, and individual response to gene therapies—supporting safer, more effective treatments and more timely diagnoses   👉 Read more here https://lnkd.in/dFfGNDsS CDP Cassa Depositi e Prestiti

🔬 From discovery to therapy: the origins of peripheral immune tolerance With the 2025 #Nobel Prize in Physiology or Medicine celebrating the discovery of regulatory T cells (Tregs) and peripheral immune tolerance, we look back at how SR-Tiget has been part of this story since the very beginning.   This article – the first in a three-part series – retraces the pioneering contributions of Maria Grazia Roncarolo and her team Rosa Bacchetta Manuela Battaglia Megan Levings Silvia Gregori, from the identification of #Treg and #Tr1 cells to the discovery of the #FOXP3 gene and its link with #IPEX syndrome, a rare disease that revealed the genetic foundations of immune balance.   Over the next two chapters, the series will explore how these breakthroughs led to ✔️ The first gene therapy approaches for immune dysregulation disorders ✔️ The development of Treg-based and tolerogenic cell therapies   Together, these milestones tell the story of how SR-Tiget has bridged fundamental immunology with clinical translation, paving the way for tomorrow’s precision therapies.   👉 Read the full article here https://lnkd.in/dbVHUmVW

🧠 If you are passionate about stem cell biology and neuroscience, do not miss the chance to work at the intersection of both!   A laboratory technician/research assistant position is immediately available in the lab led by Gabriele Ciceri, 2025 Armenise Harvard Foundation awardee. The successful applicant will contribute to innovative research focused on understanding the timing of neural development in health and disease.   👉 More details and how to apply here https://lnkd.in/gaM6Vw-w

🎓 Looking for a #PhD that nurtures both your scientific #knowledge and your #career?   The #RAREFIND PhD programme offers bespoke, structured training blending cutting-edge research in #genetherapy and #celltherapy at Telethon Institute of Genetics and Medicine (TIGEM) and San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) with a personalised #CareerDevelopment support.   👀Swipe the carousel to get a glimpse on how training, mentoring, and secondments could shape your scientific future as RAREFIND PhD student.   🌍 Learn more and apply: www.rarefind-cofund.eu #MSCA #COFUND