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

🌟 Join the SR-Tiget community through our brand-new Newsletter 🌟 The #genetherapy and #celltherapy field is advancing at an unprecedented speed, rapidly becoming a new pillar of modern precision medicine. As this global community grows, SR-Tiget remains committed to fostering connections through various initiatives, the latest of which is our newly launched Newsletter. Why subscribe? 🌐 𝗖𝗼𝗻𝗻𝗲𝗰𝘁 𝘄𝗶𝘁𝗵 𝗮 𝗴𝗹𝗼𝗯𝗮𝗹 𝗰𝗼𝗺𝗺𝘂𝗻𝗶𝘁𝘆: engage with a diverse network of scientists and clinicians, who are passionate about innovating personalized medicine  🔬𝗖𝘂𝘁𝘁𝗶𝗻𝗴-𝗲𝗱𝗴𝗲 𝘂𝗽𝗱𝗮𝘁𝗲𝘀: keep up with the latest SR-Tiget breakthroughs  📅 𝗕𝗲 𝘁𝗵𝗲 𝗳𝗶𝗿𝘀𝘁 𝘁𝗼 𝗸𝗻𝗼𝘄: do not miss out on upcoming events, initiatives, and unique opportunities to engage with us 🔍𝗕𝗲𝗵𝗶𝗻𝗱 𝘁𝗵𝗲 𝘀𝗰𝗲𝗻𝗲𝘀 𝗶𝗻𝘀𝗶𝗴𝗵𝘁𝘀: get a glimpse into the vibrant life of our community 💡 𝗕𝗲 𝗶𝗻𝘀𝗽𝗶𝗿𝗲𝗱: discover the stories of SR-Tiget #Alumni, showcasing leadership across diverse fields and learn about far-reaching influence of our work 🚀Kick-off: January 2025 👉 Become part of the SR-Tiget community and subscribe now by filling this form https://lnkd.in/dpCwBXdG

🚀 From goals to achievements: 2024 SR-Tiget year-in-review   As 2024 is soon coming to an end, we take a moment to reflect on a year filled with groundbreaking research, inspiring community achievements and significant advancements in #genetherapy. Here are some of the highlights that made 2024 remarkable   🚩 5 prestigious grants from European Research Council (ERC) to junior and senior scientists, among many other funding awards 🚩 Research breakthroughs, such as the first demonstration of durable and efficient epigenetic silencing in vivo https://lnkd.in/dVEVYMat and a landmark study confirming the long-term #safety of lentiviral hematopoietic stem cell gene therapy https://lnkd.in/dj9unuw2 🚩 Important steps forward in the #ATMPs space, including the FDA approval of Lenmeldy - originally developed by SR-Tiget research - representing a milestone for MLD patients and their families 🚩 Initiatives focused on expanding the SR-Tiget #network, such as active partecipation to multiple European consortia, strong contribution to the launch of the Italian Society of Gene and Cell Therapy #SITGEC and co-organisation of the successfull #ESGCT24 Meeting 🚩 An excellent SR-Tiget Retreat, fostering science and a great sense of community 🚩A €46.6 million Series B financing to Genespire - startup co-funded by SR-Tiget scientists - to advance its first paediatric in-vivo gene therapy into the clinic 🚩Last but not least, the treatment of 8 more patients affected by 3 distinct rare genetic diseases, coming from 4 different countries   A heartfelt thank you to all SR-Tiget teams, collaborators and supporters. Together, we will continue to transform science and lives through innovative gene therapies. Let’s make 2025 another impactful year!   Fondazione Telethon Ospedale San Raffaele Libera Università Vita-Salute San Raffaele

📺 Curious to see SR-Tiget scientists on air? Like every year, many researchers of our community will speak about #genetherapy and #rarediseases during the Fondazione Telethon fundraising TV marathon starting tomorrow. Communicating science to the general public is an essential part of the role of scientists and a skill of pivotal importance that is often not nurtured enough. If you as researcher have the chance to be involved in #publicengagement activities, do not miss the opportunity to increase public understanding of science and #inspire future generations of scientists!   👇 Find below the schedule of events with the contribution of SR-Tiget scientists

🚀 Exploring Careers Beyond SR-Tiget SR-Tiget is a multifaceted #R&D hub where breakthrough scientific discoveries go hand in hand with the development of cutting edge technologies, enabling key contributions to the field of #genetherapy and beyond.   This week, we are turning the spotlight on Angelo Amabile, who did a PhD and a Postdoctoral experience under the mentorship of #AngeloLombardo and #LuigiNaldini at SR-Tiget, where he developed a pioneering epigenome editing platform. Afterwards, he decided to pursue his career in the field of advanced therapeutics and now holds the roles of Assistant Professor and Scientific Director of the RNA and Nanoparticle Therapeutics Core at the Icahn School of Medicine at Mount Sinai.   👇To learn more about Angelo's story, the lessons learned along the way and the advice he offers to the young scientists, check out his interview

🧬 Are you interested in taking part in the optimisation, scale-up and preclinical validation of protocols for the development of innovative #genetherapy and #celltherapy products?   🧪 Do you have a proven experience in chromatography techniques?   👉 A Laboratory Technician/Research Assistant position is immediately available in the Vector Core area of the SR-Tiget Process Development Lab led by Marina Radrizzani More details and how to apply here https://lnkd.in/dFJvRrr9

🥇 Congratulations Pamela Quaranta – Postdoctoral Fellow in the lab of Alessandro Aiuti – for being awarded the #prize for the best Basic Research work by the Società Italiana di Ematologia within the XIII edition of the Under 40 in Hematology, an initiative dedicated to promoting, enhancing and rewarding the scientific work of the best under 40 Italian researchers working in the field of #hematology!   👉 SR-Tiget is proud that in the last 6 years, 6 scientists of its community were awarded this prize.

🎓 A warm welcome to the brilliant students who just enrolled in their #PhD course at Libera Università Vita-Salute San Raffaele and at Università degli Studi di Milano-Bicocca and joined SR-Tiget labs!   As you embark on this exciting journey, we encourage you to make the most out of this time 👉 embracing the challenges and opportunities that lie ahead 👉 collaborating with your peers, also beyond the walls of the Institute 👉 never hesitating to seek guidance from your Mentors 👉 having fun doing science   We wish you a great start!   Selene Ceriotti Francesca Marabotti Laura Alessandrini Alessia Zangari Claudia Castiglioni Valentina Consoli Luca Frosio Eleonora Carito Andrea Carpinteiro Cabra Francesca Vincenti

🚀 We are thrilled to announce that two SR-Tiget researchers, Daniela Cesana and Alessio Cantore, have been awarded European Research Council (ERC) Consolidator Grants! These are 2 of the 6 #ERCCoG assigned to SR-Tiget Researchers in the last 6 years.   ➡️ Daniela's project focuses on dissecting the impact, safety and effectiveness of #genetherapy at the whole organismal level by cell-free DNA signatures. Together with her team, she will investigate the role of cell-free #DNA as reliable #biomarker to assess the efficacy of gene therapy, ultimately guiding more personalized therapeutic interventions.   ➡️ Alessio is going to investigate hepatocyte heterogeneity during #liver growth and unravel its implications for #invivo gene engineering, to ultimately develop safe, effective, and durable gene therapies to treat diseases of hepatic metabolism in pediatric patients. This project will pave the way to advanced therapeutic options for currently incurable #metabolicdiseases.   👉 Find out more here https://lnkd.in/dEH3qPpC   Ospedale San Raffaele Libera Università Vita-Salute San Raffaele Fondazione Telethon   #ATMP

🚀#Synergy is the key enabling factor for progress: this is one of the key messages that we take home after the three intense days of the 2024 SR-Tiget Scientific Retreat!   The event has been a great occasion to reunite the whole SR-Tiget community in the beautiful location of Lazise and to share: 🔬 The latest exciting scientific advancements and strategic perspectives of the Institute from basic to clinical research, highlighting the strong #translational mission that makes SR-Tiget a unique R&D hub 🧬 Roundtable discussions on pressing topics, such as #ATMP development and challenges, facilitating exchanges at all levels 🤝🏻 Informal social interactions among all members of the #community, integrating new entries (99 since the last Retreat!) and fostering a culture of mutual sharing and trust   Last but not least, we had the privilege to admire and vote for the extraordinary pictures submitted by members of our community to the SR-Tiget "Science & Art Photography Contest: Capturing Discovery, Images of Our Everyday Life". This has been an enriching opportunity to remind us that everyone's vision and experience of science is different and that integrating diverse #perspectives is what makes science thrive and bloom.   Thanks to everyone that made this Retreat possible and participated with such commitment and enthusiasm, ultimately making it a success!

SR-Tiget is actively working to advance the #ClinicalDevelopment of a promising #genetherapy approach for Autosomal Recessive #Osteopetrosis (ARO), a rare and fatal genetic disorder leading to severe bone abnormalities due to defective osteoclasts. Current treatment options are limited to hematopoietic stem cell transplantation (HSCT), which carries significant risks.   🔍 In a recent study published in Frontiers in Endocrinology, Anna Villa, Valentina Capo, and team with Sara Penna as first author demonstrated the potential of gene therapy using a murine model of ARO. ✔️ Lentiviral gene therapy effectively reversed bone defects and achieved survival rates similar to HSCT ✔️ Hematopoietic stem cell mobilization improved cell collection for therapy ✔️ Non-genotoxic conditioning offered a viable alternative to traditional, more toxic conditioning regimens while allowing survival and bone correction. 💡 These findings pave the way for implementing an effective gene therapy protocol, which could drastically reduce the complications associated with transplantation in young, severely affected osteopetrotic patients, offering a more timely and safer treatment option. Read the full study for more insights 👉https://lnkd.in/dshzXmje