San Raffaele Telethon Institute for Gene Therapy
The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1996 as a joint venture between the Fondazione Telethon and Ospedale San Raffaele, with the mission to perform cutting-edge research in gene and cell therapy and to translate its results into therapeutic advances, focusing on genetic diseases.
SR-Tiget represents a multi-disciplinary research environment, which provides a unique blend of scientific expertise in the development of innovative gene and cell therapy strategies, access to relevant preclinical models to evaluate their efficacy and safety, as well as competence in conducting early phase clinical trials. This provides a fertile ground for alliances with industrial partners and launch of start-up companies, which are crucial to secure the skills and resources required to address the regulatory hurdles and manufacturing needs to bring new therapies to registration and make them available to patients.
GOALS & ASSETTS
We employ a basic-to-translational, bench-to-bedside R&D approach encompassing the characterization of biological properties and physiopathological processes, the design and optimization of safety and efficacy of novel gene and cell therapy platforms and their development from preclinical models to first-in-human testing.
|GOAL 1: Advance HSC gene therapy to standard-of-care for rare genetic diseases | Discover more||
GOAL 2: Be at the forefront of gene editing development | Discover more
|GOAL 3: Broaden application of lentiviral vectors to in vivo gene therapy | Discover more||GOAL 4: Devise new cell and gene therapy strategies to modulate immune response | Discover more|
Recognized assets of the Institute include:
- Leadership in lentiviral gene transfer technology
- Pioneering contribution to the genome and epigenome editing field
- State-of-the-art stem cell transplantation center for adult and pediatric patients
- In house facilities and resources supporting translational research and first-in-human phase I/II gene therapy trials
- Strategic alliances with industrial partners and successful launch of startup biotech companies
- Roster of young PIs venturing into relevant new technologies and emerging biological concepts
Epigenetic regulation and targeted genome editing
Angelo Lombardo, Group leader
Gene and neural stem cell therapy for lysosomal storage diseases
Angela Gritti, Group leader
Genomics of the innate immune system
Renato Ostuni, Group leader
Gene transfer into stem cells
Giuliana Ferrari, Group leader
Gene transfer technologies and new gene therapy strategies
Luigi Naldini, Group leader
Human hematopoietic development and disease modeling
Andrea Ditadi, Group leader
Liver gene therapy
Alessio Cantore, Group leader
Mechanisms of inflammation in health and disease
Alessandra Mortellaro, Group leader
Mechanisms of peripheral tolerance
Silvia Gregori, Group leader
Pathogenesis and therapy of primary immunodeficiencies
Alessandro Aiuti, Group leader
Pathogenesis and treatment of immune and bone diseases
Anna Villa, Group leader
Retrovirus-host interactions and innate immunity to gene transfer
Anna Kajaste-Rudnitski, Group leader
Safety of gene therapy and insertional mutagenesis
Eugenio Montini, Group leader
Senescence in stem cell aging, differentiation and cancer
Raffaella Di Micco, Group leader
Translational stem cell and leukemia
Bernhard Gentner, Group leader
Targeted Cancer Gene Therapy
Luigi Naldini, Group leader
Ivan Merelli, Facility manager
Group leader, Pathogenesis and therapy of primary immunodeficiencies Unit
Group leader, Liver gene therapy Unit
Raffaella Di Micco
Group leader, Senescence in stem cell aging, differentiation and cancer Unit
Group leader, Human hematopoietic development and disease modeling Unit
Group leader, Gene transfer into stem cell Unit
Group leader, Translational stem cell and leukemia Unit
Group leader, Mechanisms of peripheral tolerance Unit
Group leader, Gene and neural stem cell therapy for lysosomal storage diseases Unit
Group leader, Retrovirus-host interactions and innate immunity to gene transfer Unit
Group leader, Epigenetic regulation and targeted genome editing Unit
Group leader, Safety of gene therapy and insertional mutagenesis Unit
Group leader, Mechanisms of inflammation in health and disease Unit
Group leader, Gene transfer technologies and new gene therapy strategies Unit
Group leader, Genomics of the innate immune system Unit
Group leader, Pathogenesis and treatment of immune and bone diseases Unit
The SR-Tiget portfolio of gene and cell therapies embraces the full spectrum of drug development. The most advanced steps of the Institute development pipeline are undertaken in the context of strategic alliances with industrial partners or through the spin-off of startup biotechnology companies, which are crucial to secure the resources and the multi‐disciplinary expertise required to attain the ultimate goal of delivering the therapies to patients.
Starting from 2004, the Institute has been involved in a number of strategic alliances set up between OSR, Fondazione Telethon and key industry players in the cell and gene therapy field, including:
- Orchard Therapeutics Limited (2018-ongoing)
- Sanofi (2017-2022)
- Editas Medicine (2016-2018)
- Biogen (2014-2017)
- GlaxoSmithKline (2010-2018)
- Sangamo Therapeutics (2004-2015)
More recently, three startup companies stemmed from SR-Tiget research:
- Genenta Science (launched by OSR), focused on the development of cancer immunotherapy. In 2021, the company was listed on Nasdaq, becoming the first italian biotech on the American stock exchange market.
- Epsilen Bio (launched by OSR and Fondazione Telethon), whose mission is to develop therapies based on the epigenetic silencing platform. In 2021, it has been acquired by Chroma Medicine, giving rise to the largest epigenetic editing company in the world.
- Genespire (launched by OSR and Fondazione Telethon), whose portfolio includes the development of ex vivo gene editing therapies and liver-directed in vivo gene therapies based on advanced lentiviral vectors.
SR-Tiget actively promotes the professional growth of scientists at all stages of their career.
Undergraduate students. SR-Tiget laboratories offer internship opportunities to motivated master students from all universities. Trainees benefit from a dynamic scientific environment and have access to activities organized by the host labs and the Institute (e.g. journal clubs, seminars, retreats, etc.).
PhD students. The Institute hosts PhD students mainly enrolled in the International PhD Course in Molecular Medicine at the Vita-Salute San Raffaele University. In particular a dedicated Gene and Cell Therapy curriculum aims to train young scientists in the study of genetic diseases and in the development of new gene and cell therapy strategies.
Postdoctoral Fellows. SR-Tiget is committed to support postdoctoral researchers in their professional development, providing training aimed at broadening both their research and soft skills and preparing them for the next step of their career.
Discovered a key mechanism in the pancreatic tumor pathogenesis
The study, published on Nature by San Raffaele researchers, identifies a new possible therapeutic target that could slow the process of a pancreatic tumour
In vivo gene therapy equips the immune system against liver metastases
A San Raffaele study demonstrates, in experimental models, the effectiveness of a new gene therapy platform to engineer the immune cells of the liver against liver metastases
Acute B-cell lymphoblastic leukemia: identified a subpopulation of "stem-like" leukemic cells that can influence disease prognosis
Thanks to data obtained with advanced sequencing and transcriptomic technologies, it will be possible to identify early more prognostic criteria and better tailor treatment
The prestigious Consolidator Grant of the European Research Council awarded to Renato Ostuni
Today, the European Research Council (ERC) awarded the Consolidator Grants, Europe's most sought after research funding for scientists at a mid-career stage. Renato Ostuni - Head of the Genomics of the innate immune system Unit at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Associate Professor of Histology at the Vita-Salute San Raffaele University – just won one worth 2 million euros for 5 years.
X-PAND: a new multi-centric project on gene therapy
Ospedale San Raffaele announces the launch of the four-year international, multi-centric project X-PAND funded by the European Union under the Horizon Europe – European Innovation Council program.
The first neutrophil atlas
A research team from SR-Tiget generated for the first time a complete mapping of human neutrophils, cells of fundamental importance for our defense system
Investigational gene therapy shows early potential to treat a serious metabolic disease
18/11/2021 - Strengthened by the long and solid experience on other genetic diseases, the SR-Tiget researchers have potentially corrected the genetic defect responsible for Hurler syndrome
A new strategy to monitor gene therapy long-term safety and efficacy
02/07/2021 - Researchers at San Raffaele developed a new technique, allowing to probe cell free-DNA circulating in the bloodstream to obtain information regarding engineered cells.
Scientists discover a new regulator of antiviral and antitumor immunity
15/06/2021 - Researchers at SR-Tiget identified a novel mechanism responsible for regulating innate immunity, with important implications in oncology, infectious diseases and for optimizing gene therapy protocols.
Gene editing: getting closer to clinical application for a rare genetic disease
22/01/2021 - New study demonstrates that CRIPSR-Cas9 is able to correct the genetic defect underlying hyper IgM syndrome in an animal model of the disease
EC approval for Libmeldy
21/12/2020 - Developed thanks to more than 15 years of research by the San Raffaele Telethon Institute for Gene Therapy in Milan, Libmeldy is the first therapy approved for eligible patients with early-onset MLD
A prestigious ERC grant awarded to Raffaella Di Micco
14/12/2020 - Di Micco’s project will investigate how blood stem cells respond to genetic manipulation, with the goal of making gene therapy safer and more effective
Raffaella Di Micco awarded with the Robertson Stem Cell Prize
27/10/2020 - The prestigious recognition arrives for the first time in Italy and will support Di Micco’s research on blood stem cell therapies for rare genetic diseases
Else Kröner Fresenius Prize for biomedical research to Alessandro Aiuti
06/08/2020 - The Deputy Director of the San Raffaele-Telethon Institute for Gene Therapy in Milan awarded for his contribution to the development of innovative therapies for serious genetic diseases
“Don’t-Eat-Me". Viral vectors resistant to capture by immune cells
24/05/2019 - Researchers obtained gene therapy vectors that escape capture by virus-clearing immune cells in the liver and spleen
Efficacy of gene therapy in treating Wiskott-Aldrich Syndrome
11/04/2019 - The clinical study demonstrates the effectiveness of gene therapy in treating Wiskott-Aldrich Syndrome
The 2019 Louis-Jeantet Prize awarded to Luigi Naldini
22/01/2019 - The director of the San Raffaele Telethon Institute for Gene Therapy wins the prize for his pioneering work on gene therapy
Beta Thalassemia: encouraging evidence for safety and efficacy of gene therapy
18/01/2019 - The study is the first clinical study to suggest that gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia
A new ingredient to potentiate gene therapy
09/11/2018 - A study shows that a naturally occurring compound significantly increases the efficiency of lentiviral vector-mediated gene transfer in blood stem cells
Gene therapy unveils family tree of blood stem cells
02/10/2018 - Study shows the role of different stem cells families both in the earliest and in the steady state phase after BM transplantation
A prestigious ERC grant to improve gene therapy
13/12/2018 - The ERC awarded 291 researchers from 40 different countries: among the winners Anna Kajaste-Rudnitski, group leader at SR-Tiget