As a leader in the academic development and early clinical testing of advanced gene therapy products, Spoke 10 is managed by IRCCS Bambino Gesù Children’s Hospital in Rome in collaboration with other Institutions whose activities have dramatically contributed to the advancement in this in Italy. Spoke 10 affiliates include San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Tettamanti Foundation, National Research Council of Italy (CNR), University of Campania (UNICAMPANIA), University of Padua (UNIPD), and Gabriele d’Annunzio University of Chieti and Pescara (UNICH), togheter with industrial partners Takis Biotech, IRBM Research Group, InnovaVector, Performing Beyond Limits (PBL), Chiesi Group, Stevanato Group and Eurofins Biolab.
A Scientific Institute for Research, Hospitalization and Health Care and an Academic Medical Center. OPBG is Italy’s main pediatric hospital and acts as a referral center at national and international level. The hospital stands out for its pioneering and innovative role in advanced cell and gene therapies. Indeed, OPBG scientists have a relevant record of accomplishment in research and clinical experience within the field of Gene and Cell Therapy (GCT). The academic OPBG GMP Facility, approved by the national competent authority (AIFA) for GCT, has relevant experience in the transduction of somatic human cells with viral vectors (retro and lentiviruses). In the contest of the National Center, OPBG will be the coordinator of Spoke 10.
Settore studi clinici oncoematologici e terapie cellulari
Unità terapia genica dei tumori
Settore studi clinici oncoematologici e terapie cellulari
Officina Farmaceutica
Settore produzione Officina Farmaceutica
Settore studi clinici oncoematologici e terapie cellulari
Settore studi clinici oncoematologici e terapie cellulari
Settore studi clinici oncoematologici e terapie cellulari
Unità di terapia genica dei tumori
Unità di genetica ed epigenetica dei tumori pediatrici
Settore assicurazione qualità Officina Farmaceutica
Settore assicurazione qualità Officina Farmaceutica
Unità di terapia genica dei tumori
Unità di terapia genica dei tumori
Settore produzione Officina Farmaceutica
Unità di terapia genica dei tumori
Unità di terapia genica dei tumori
Unità di terapia genica dei tumori
Settore controllo qualità Officina Farmaceutica
Settore assicurazione qualità Officina Farmaceutica
Settore produzione Officina Farmaceutica
Settore produzione Officina Farmaceutica
Unità di terapia genica dei tumori
Tettamanti Foundation has consolidated expertise in the preclinical characterization of CAR-T cells against ALL and AML developing in recent years a non-viral gene transfer approach that has been patented (EP3215605: Improved method for the generation of genetically modified cells) for the production of CAR-CIK cells.
San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) aims to advance biomedical research toward the diagnosis, cure, and prevention of human rare genetic diseases. FT counts two intramural research Institutes: the Telethon Institute for Genetics and Medicine (TIGEM) located in Pozzuoli and the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) located in Milan. SR-TIGET has provided seminal contributions to the gene and cell therapy field with relevant discoveries in vector design, gene transfer, and gene editing strategies, which have led to several successful trials of novel ATMPs with >140 patients treated and 2 products already approved on the EU market. In addition, SR-TIGET comprises expertise and facilities for ATMP process development and scale-up as well as a GLP Assays Center certified by ISS for method validation and in vivo biodistribution/toxicology studies of ATMP.
National Research Council of Italy (CNR) scientists have provided significant contributions to several research activities with high scientific relevance with an understanding of the impact of RNA metabolism on human health. CNR explores novel delivery methods for therapeutic molecules, including the development of new AAV vectors for the therapy of neurological diseases.
University of Campania (UNICAMPANIA) participants have consolidated expertise in defining gene therapy for inherited retinal dystrophies (IRDs) confirmed by the participation to the first ocular gene therapy clinical trial worldwide for the treatment of an IRD (i.e. Leber Congenital Amaurosis caused by mutations in the RPE65 gene).
Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche
Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche
Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche
Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche
Dipartimento di Scienze e Tecnologie Ambientali Biologiche e Farmaceutiche (DISTABiF)
Dipartimento di Medicina di Precisione
Dipartimento di Salute Mentale e Fisica e Medicina Preventiva
Dipartimento di Economia
Dipartimento di Medicina Sperimentale
University of Padua (UNIPD) has large experience with GMP grade production of vectors for gene therapy, testified by successful clinical trials and by their past direction of the Gene Therapy Program, Boston Children’s Hospital, and their launch of dedicated startups like Altheia Science.
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Medicina
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Dipartimento di Salute della Donna e del Bambino
Gabriele d’Annunzio University of Chieti and Pescara (UNICH) scientists have strong scientific experience on several relevant scientific topics likewise leukemia, as well as on the study of T cells and extracellular vesicles. The scientist team operates at the Center for Advanced Studies and Technology (CAST), where they conduct synergistic activities cooperating with the core facilities (genomic, proteomic, microscopy, flow cytometry) to develop and translate several cell therapies to clinical practice.
Dipartimento di Farmacia
Dipartimento di Medicina e Scienze dell’Invecchiamento
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Dipartimento di Neuroscienze, Imaging e Scienze Cliniche
Dipartimento di Medicina e Scienze dell’Invecchiamento
Dipartimento di Farmacia
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Dipartimento di Farmacia
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Dipartimento di Farmacia
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Dipartimento di Medicina e Scienze dell’Invecchiamento
Dipartimento di Scienze Mediche, Orali e Biotecnologiche
Takis Biotech provides significant contributions to the discovery and development of innovative Vaccines based on viral vectors and DNA/mRNA platform technology, leveraging on its strong record of accomplishment in drug discovery. As one of its main assets, in vivo electro-gene-transfer (EGT) can be used for a variety of clinically useful applications, from vaccine development to somatic gene therapy.
IRBM Research Group is an organization with world-class expertise and capabilities in the discovery and development of peptide, small molecule, and antibody-based therapeutics and a strong track record of developing drugs that have entered the different phases of clinical development and in some cases have been approved for marketing. In collaboration with other members of the spoke, IRBM will use its proprietary high-complexity monoclonal antibodies phage library to screen biospecimens from patients with hematologic malignancies to identify novel targets and novel antibodies, which could be used for CAR-T or NK-CAR cell therapy.
InnovaVector is an innovative start-up born as NewCo of the Telethon Foundation, absorbing the skills of the Adeno-Associated Viral (AAV) Vector Core group of the Telethon Institute of Genetics and Medicine (TIGEM). InnovaVector’s team boasts consolidated experience in the production of AAV vectors from R&D to GMP grade, to support the pre-clinical and clinical development of AAV-based products.
Performing Beyond Limits (PBL Srl) is an innovative SME whose core business is the design and realization of customized automated industrial lines for biomedical and pharmaceutical sectors. A part from high expertise in the development of inspection and formulation industrial systems, PBL has gained a well-proven experience and know-how in the development of innovative platforms for ATMPs (e.g.. cell and gene therapy, tissue engineering, RNA-based vaccines…). PBL represents a key partner for creation of customized and flexible automated machines for the manufacturing of RNA&Gene therapies, making them more affordable, scaled-up and standardized.
Eurofins Biolab is a global leader in testing and research services by supporting research through optimizing current analytical procedures and exploring innovative methodologies.
Stevanato Group is a global leader in providing advanced solutions for drug containment, drug delivery systems, and pharmaceutical services. With a specialization in glass primary packaging for pharmaceuticals and diagnostics, Stevanato excels in producing high-quality innovative solutions like glass containers, syringes, and cartridges for injectable drugs. The company’s expertise spans engineering, manufacturing, and delivering state-of-the-art solutions in drug packaging, drug delivery devices, and analytical services, which cater to the needs of pharmaceutical companies worldwide. Stevanato is renowned for its focus on innovation, sustainability, and commitment to advancing technologies in pharmaceutical packaging and drug delivery systems.
Chiesi Group is an international research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. With its headquarters in Parma (Italy) and with over 85 years of experience, Chiesi operates within 31 countries and has more than 6,500 employees. Its research and development center in Parma works alongside six other important R&D hubs in France, the USA, Canada, China, the UK, and Sweden.