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Italian National Node

Italy is actively involved in EPTRI which sees the participation of 24 Institutions interested in paediatric basic, preclinical and translational research and innovation, among which Universities, Hospitals, and research centers of excellence.

EPTRI-IT is organised as a Joint Research Unit (JRU), coordinated by Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) together with Consiglio Nazionale delle Ricerche (CNR) and the Italian Network for Paediatric Clinical Trials (INCiPiT) which will be respectively in charge of the general management and of chairmanship of the two decisional boards: the General Assembly (GA) and the Scientific Committee (SC).

EPTRI has received the official letters of political and financial support from Ministero dell’Università e della Ricerca on the 13th of February 2020 and from Consiglio Nazionale delle Ricerche on the 28th of January 2020, to support the inclusion of EPTRI in the European Strategic Forum for Research Infrastructures (ESFRI) 2021 Roadmap.

Additionally, all Italian Partners of EPTRI have signed the Memorandum of Understanding which was one of the main documents of the ESFRI application submitted on September 9th, 2020.

Italy has registered a great participation in the EPTRI context analysis surveys developed during the H2020 ID-EPTRI project which shown expertise in the areas of “Paediatric Medicines Discovery” (47%), “Paediatric Biomarkers and Biosamples” (28%), “Developmental Pharmacology” (2,94%), “Paediatric Medicines Formulations” (13,24% ) and “Paediatric Medical Devices” (8,82%).

The main objectives of EPTRI-IT are to reinforce the paediatric research in Italy by promoting and sharing the progress of biological and medical science in paediatrics and to contribute in reducing the serious delays in providing paediatric patients with age-tailored, safe and efficacious advanced medicines and innovative therapies.

Chair of EPTRI-IT
Scientific Committee

Adriana-Ceci

Adriana Ceci
(INCiPiT)
Email: [email protected]

Chair of EPTRI-IT
General Assembly

Sergio-Giannattasio

Sergio Giannatasio
(CNR)
Email: [email protected]

EPTRI-IT Coordinator

Donato-Bonifazi

Donato Bonifazi
(CVBF)
Email: [email protected]

Joint Research Unit Manager

Mariangela-Lupo

Mariangela Lupo
(CVBF)
Email: [email protected]

Liaison Officer

Bonka-Georgieva

Bonka Georgieva
(CVBF)
Email: [email protected]

The EPTRI Italian National Node is composed of the following Institutions:

Consiglio Nazionale delle Ricerche (CNR)

CNR is the largest public research institution in Italy, the only one under the Research Ministry performing multidisciplinary activities. Founded as legal entity on 18 November 1923, CNR’s mission is to perform research in its own Institutes, to promote innovation and competitiveness of the national industrial system, to promote the internationalization of the national research system, to provide technologies and solutions to emerging public and private needs, to advice Government and other public bodies, and to contribute to the qualification of human resources. In the CNR’s research world, the main resource is the available knowledge, which means people, with their skills, commitment and ideas. This capital comprises more than 8.000 employees, of whom more than half are researchers and technologists. Some 4.000 young researchers are engaged in postgraduate studies and research training at CNR within the organization’s top-priority areas of interest. A significant contribution also comes from research associates: researchers, from Universities or private firms, who take part in CNR’s research activities.

CNR participates in EPTRI-IT with 13 Institutes. Read more about them.

Alma Mater Studiorum – Università di Bologna (UNIBO)

Founded in 1088, UNIBO is recognized as the oldest university of the Western world. Nowadays, UNIBO is the second largest university in Italy and one of the most important institutions of higher education across Europe. UNIBO in numbers: 5 operating sites and a permanent headquarter in Buenos Aires, 637 M€ turnover, 32 Departments, 5 Schools, 87.758 students (among which, 6.484 International students), 5.733 permanent staff, 2nd Italian University in QS World University Ranking 2019 for Life Science & Medicine, 30 research outputs per day and 11.000 research outputs per year. Research and Innovation are a priority of UNIBO. Regarding its fundraising ability, UNIBO is very active at both national and the European level in all the research areas. Among its assets, UNIBO encompasses Bologna University Hospital Authority St. Orsola-Malpighi, which is home to the School of Medicine and Surgery. The University Hospital is an internationally acclaimed institution for the study and treatment of diseases, and each year organizes medical conferences and conventions attended by professionals of international fame. In Horizon 2020, UNIBO is involved in 306 funded projects (81 as coordinator) with more than 128 M€ of funding. In the framework of the Societal Challenge 1 – Health, Demographic Change & Wellbeing and the Innovative Medicine Initiative and related funding frameworks, UNIBO is participating in 30 projects, including 5 under IMI-2 JTI and 2 projects selected for funding under the SC1-PHE-CORONAVIRUS-2020-2 call, with a total EU contribution of over 14 M€. Moreover, the University of Bologna has advanced skills in management of large amount of digital data. The interdisciplinary research focuses on healthy ageing, cognitive impairment and musculoskeletal conditions, among others. Research activities spans from the application of virtual/augmented reality (VR/AR) and digital diagnostics and care, to the use of health informatics and mobile technologies.

Health Research

The main research activities developed in UNIBO are referred to twelve main fields:

  1. Ambient Assisted living
  2. Big Data and Omics medicine
  3. Brain Ageing And neurodegeneration
  4. Cancer
  5. Digital Medicine and e-health
  6. Epidemiology, biobanking and Cohorts
  7. Multimorbidity and age-related Diseases
  8. Pharmaceutical biotech
  9. Rare Diseases and Undiagnosed disorder
  10. Regenerative medicine and tissue Engineering
  11. Skeletal Health and Diseases
  12. Systems and Personalised medicine

With four specific paediatrics research domains:

  • Neonatology
  • Emergency pediatrics
  • Pediatric oncology
  • Pediatric neurology
 

Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari (AOPB)

At AOPB, experts, physicians and researchers study especially oncology, endocrinology, hepatology, nutrition, pneumology, respiratory diseases, neurology, rheumatology, cardiology and vascular diseases. Solid organ transplants are actively carried on. AOPB represents a regional reference centre for molecular diagnostics of infectious diseases. At the AOPB Giovanni XXIII Children Hospital, almost every specialty is represented. Patient care and research are specially focused on infectious diseases, nephrology, rheumatology, surgery, cardiology, intensive care, oncology, orthopaedics, hematology, endocrinology, gastroenterology, pulmonology, metabolic disorders and neonatal screening.

Azienda Ospedaliero Universitaria Meyer (AOU Meyer)

Meyer Children’s Hospital is a 250-bed public hospital of Tuscany’s regional health service and is integrated with the University of Florence. The hospital’s healthcare, research and training activities cover all pediatric medical and surgical areas and position it among Italy’s highly specialized hospitals for diagnosis, care and rehabilitation services for children, also as a national reference centre for complex cases, rare diseases, and surgery. Meyer’s researchers leverage state-of-the-art methods and technology to understand, prevent and halt the progression of childhood diseases, develop new and improved diagnostic tools, pave the way for innovative treatments and therapies, carry out high-level clinical studies on medicines and therapies, and evaluate how diseases progress and the impact of treatment. The hospital fosters independent research by guaranteeing services to support the development and execution of research and innovation projects and both non-profit and sponsored clinical trials. Meyer is located in the Florentine foothills in an area characterized by natural beauty; the hospital is currently undergoing a phase of rapid expansion with several construction projects underway that will further strengthen its capacity to provide excellent patient-centered care, undertake innovative research and train the pediatric healthcare workers of tomorrow.

 
 

Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF)

It is a non-profit organisation whose mission is to perform research and education in the pharmaceutical field integrating regulatory, clinical, epidemiological, economical and statistical expertise. It is a consortium between Università degli Studi di Bari ‘Aldo Moro’ (UniBA), Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (FGB), TEDDY – European Network of Excellence for Paediatric Reserach (TEDDY) and Mediterranea – Associazione per lo sviluppo locale (MEDITERRANEA). The main fields of interest are life sciences and biotechnologies, drug development for small populations (paediatric and rare diseases), research management and methodology, monitoring, statistics, regulatory, ethics and pharmacovigilance. CVBF can count on the expertise and collaboration of researchers, scientists, health experts and research centers working together to identify the most appropriate research tools and procedures that reflect the specificity of the paediatric patient population. CVBF has a significant expertise in the project coordination, conducting research activities mainly in the context of national and EU funded projects and being Coordinator of the first paediatric network of excellence under the FP6 (TEDDY), 2 FP7 projects (DEEP, GAPP) and Partner in many projects funded by the European Commission (DG-Research and DG-SANCO).

 
 

Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (FGB)

is a Scientific Research Organisation, registered as a private, not-for-profit Foundation in 2007 to promote research in biological, medical and pharmaceutical areas, with a special attention to populations with specific therapeutic needs, including children and patients affected by rare diseases. During the past 20 years, initially through its founders and then as an organisation, FGB has been an active part of the European common efforts to improve the conditions for the development of innovative and safe medicines in Europe, including the setting up of a large partnership working on novelties and good practices for the European Pharmaceutical System which led to the establishment of the TASK-FORCE IN EUROPE FOR DRUG DEVELOPMENT FOR THE YOUNG – TEDDY, the first European Network of Excellence funded to promote the availability of safe and effective medicines for children in Europe, and its successors GRiP – Global Research in Paediatrics Network of Excellence and c4c – Collaborative Network for European Clinical Trials for Children. Key achievements include the 13 yearly editions of the Foresight Training Course, international courses aimed at training high-specialised professionals in the different fields of regulatory sciences and representing a relevant occasion to disseminate good practices and to promote pharmacological and health science research and innovation among the main stakeholders.

FGB staff members have gained skills and knowledge in the fields of Regulatory Science and Ethics related to paediatrics and rare diseases as well as HTA, they contribute to international research activities by designing Paediatric Investigation Plans (PIPs) and preparing the related dossiers, by preparing guiding documents and templates on paediatric consent and assent forms, by collecting, managing, analysing, sharing, and reusing health data and by producing health technology assessment reports. FGB experts are or have been members of the Paediatric Committee (PDCO) and other scientific committees and working groups at the European Medicines Agency (EMA), the Italian Medicines Agency (AIFA), the Italian National Agency for Regional Health Services (AGENAS) and the Regional Agency for Health and Social Care of Puglia, Italy (AReSS). FGB regularly contributes to public consultations on ethics, paediatrics, rare diseases, clinical trials, preclinical studies, patients’ empowerment in drug development, etc. launched by the EMA and the European Commission.

Within its IT-Lab, researchers and data scientists are engaged in designing, developing and managing platforms, databases and other IT-tools devised to support, manage and monitor research activities (e.g., patients registries, surveys and other data collection tools, e-learning environments, Project Management and Performance Monitoring Systems, etc.).

In the European Paediatric Translational Research Infrastructure – ID-EPTRI project, FGB has been member of the Project Steering Committee and substantially contributed to the Conceptual Design Report (CDR) and the technical design of the future Research Infrastructure as well as in other activities such as the context analysis and the ethical review of the infrastructure.

 
 

Fondazione PENTA Onlus (PENTA)

Penta (Fondazione Penta – for the treatment and cure of HIV (and related diseases) Onlus, www.penta-id.org) was established in 1991 as an independent collaboration among paediatric HIV centres across Europe aiming to address questions about antiretroviral therapy (ART) in HIV infected children. The Penta Foundation was subsequently set up in 2004 as the legal body representing the Penta network. In 2011 Penta evolved to Penta-ID (Infectious Diseases, integrating the expertise acquired in more than 20 years of successful activity in the area of HIV to other paediatric infectious diseases and investigate and implement the best ways to prevent, diagnose and treat diseases in children. To date, Penta sponsored 20 trials in paediatric HIV. The Penta network benefits from international recognition, wide geographical representativeness with over 130 centers in 42 countries, very high-quality research, an outstanding track record and diversified public and private funding. Penta-ID has been recognized as a Level-1 Network for paediatric infectious disease in Europe by EnprEMA. Penta is also member of a WHO Network (Global Accelerator for Paediatric Formulations (GAP-f)) aimed at enhancing the coordination and accelerating the prioritization, investigation, development, introduction, and safe rollout of high-quality medicines in optimal formulations for children in need. Current activities beyond clinical trials and epidemiologic studies, include training/educational programmes, capacity building activities, and development of HIV treatment guidelines.

 
 

Fondazione Telethon (Telethon)

Fondazione Telethon (FT) is one of the major Italian biomedical non-profit organization founded in 1990 at the behest of patient groups to advance biomedical research towards the cure of rare genetic conditions. In its 30 years of activity, FT has invested more than €556 million euro in funding for more than 2,700 research grants and involving over 1,630 scientists for the advancement of knowledge regarding rare genetic diseases and development of treatments. FT is also comprised of two global centres of excellence in the study of rare genetic conditions: the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli (Naples) and the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan.

 
 

IRCCS “Eugenio Medea”, Sezione Scientifica dell’Associazione La Nostra Famiglia (IRCCS E. Medea)

It is dedicated to research, treatment and training in the field of neurological and neuropsychic pathologies. The researchers work on neonatology, neonatal and paediatric intensive care, psychiatry and psychology, otolaryngology, neurology, ophthalmology, musculoskeletal diseases, pneumology and respiratory diseases. The Neurobiology Laboratory studies the paediatric development mechanism in neurology (e.g., movement disorders, infant brain atlas) and in rehabilitation. They also develop new model platforms using fly models. They perform developmental molecular and experimental pharmacology using in-vivo and ex-vivo model to test drugs. Moreover, the researchers use drosophila model to study motor neuron diseases, in particular hereditary spastic paraplegia genes. They use also the imaging technology to study brain and muscle. Furthermore, they work to identify the defect in autophagy process in hereditary spastic paraparesis type 15. At the Bioengineering Laboratory, the researchers study new conventional and small active pharmaceutical ingredients, and develop oral, buccal, sublingual, solid monolithic, solid powder, solid multiparticulates, liquid, and semisolid solutions. They perform also study in medical devices field, prototyping and validating devices and software dedicated to improving quality of life, rehabilitate and promote social inclusion of patients with chronic diseases.

IRCCS Burlo Garofolo (IRCCS B. Garofolo)

The research activity is articulated in five main areas that include chronic diseases with onset in paediatric age, materno-foetal medicine and neonatology, epidemiology, prevention and quality of care, pediatric surgery and rehabilitation, and pediatric neurosciences. IRCCS Burlo Garofolo plays a leading role in the research on molecular bases and identification of diagnostic biomarkers of coeliac disease and primary immunodeficiencies.  Research is also devoted to chronic inflammatory bowel diseases and pediatric rheumatic diseases where it focuses on the identification of novel diagnostic approaches and treatment personalization. IRCCS Burlo Garofolo acts as WHO Collaborating Centre for maternal and child health and is part of the Italian Network for Pediatric Clinical Trials (INCIPIT). The Institute is also a member of the Consortium for Biomedicine of AREA Science Park of Trieste, which gathers major international and national biotechnology research centers and offers sophisticated facilities for Biomedical Research, such as molecular imaging and production of nanovectors.

Istituto Ortopedico Rizzoli (I.O. Rizzoli)

At the Rizzoli, the current research is developed following four main lines of research: (I) oncology in terms of biological characterization of musculoskeletal tumours to identify prognostic factors, studies in pharmacogenomics and preclinical evaluation of new antitumoral drugs, validation of innovative surgical techniques; (II) regenerative and reparative medicine as design and execution of preclinical (in vitro/in vivo) and clinical studies for the treatment of acute, chronic and degenerative diseases of the locomotor system; (III) innovative and prosthetic surgery, design and execution of preclinical (in silico/in vitro/in vivo) and clinical studies on reconstructive surgical techniques; (IV) inflammatory, infectious, degenerative and genetic diseases in terms of defining the ezio-pathogenetic mechanisms of musculoskeletal diseases, identifying the local and systemic modifications caused in the body, and testing innovative therapies.

 
 

Italian Network for Paediatric Clinical Trials (INCiPiT)

It is a no profit consortium composed by the main Italian children’s hospitals, the largest departments of paediatrics as well as national and international paediatric therapeutic networks coordinated by Italian Institutions. It is coordinated by Ospedale Pediatrico Bambino Gesù and counts 23 Partners. Its primary mission is to foster high-quality research on drugs in children, but also improve opportunities for performing paediatric clinical trials, provide training and create competences, harmonize joint national operational models, create guidelines and templates to help serve research, involve children and young people and their families in paediatric research.

 
 

Ospedale Pediatrico Bambino Gesù (OPBG)

It is a scientific hospital which operates in three main sectors: research, assistance and treatment, and training. OPBG covers all the clinical aspects related to the paediatric medicine. Physician and researchers study genetic variation with in-silico and experimental approaches, use microarray to perform expression studies in Shwachman-Diamond syndrome, and discover frameshift mutations that cause congenital diseases. They also study dermatitis, juvenile idiopathic arthritis, cystic fibrosis and paediatric development mechanisms relevant in immunology (e.g., asthma), neurology (e.g., movement disorders, infant brain atlas), oncology (e.g., neuroblastoma, epigenetics, medulloblastoma, lung cancer, and colorectal cancer), infection (e.g., encephalopathy) and hormonal research (e.g., infancy growth, onset of puberty). Moreover, they develop new model platforms using placental and umbilical cord, and 3D organoid cell cultures from paediatric samples. They perform in vivo studies, stem cell biology and regenerative medicine with tissue engineering approaches. Moreover, physician and researchers perform research activities in identification, characterisation and validation of the biomarkers to diagnose, predict and prognostic genetic, immunological, onco-haematological and infectious diseases. Furthermore, they study biomarkers to be used as drug targets in paediatrics, development stage marker and safe marker in response to therapy. OPBG has a biobank with a sector dedicated to paediatric biosamples.

 
 

Ospedale Pediatrico Istituto Giannina Gaslini di Genova (IGG)

The Giannina Gaslini Institute for Scientific Hospitalization and Care (IRCCS) represents an excellence at national and international level for pediatric aspects and more generally for the maternal and child area. The Gaslini Institute pursues purposes of care and assistance to subjects in developmental age, combining it with the research activity of excellence in the biomedical field and with training activity, hosting the structures of the University of Genoa.The institute’s lines of research focus especially on the disciplines of Neuroscience, Rheumatology, Genetics, Nephrology, Onco-hematology, Immunology and Surgical and Rehabilitative Technologies. Network relationships are to be considered fundamental for the prospects of both research and highly specialized clinical activity, which allow the Institute to maintain a strong capacity for attraction of patients from other Italian regions and from numerous European and extra-European countries, as well as determining the conduct of a significant number of clinical trials. The Gaslini Institute is part of numerous national and international networks (e.g., European Reference networks, EATRIS) which group clinical and research centers. In 2019, four new patents were presented by the Institute. In 2019, the agreement between the Giannina Gaslini Institute and the Italian Institute of Technology (IIT) was brought to an operational conclusion, in particular as regards the aspects of the Joint Lab of Genomics, which presents both of high-quality research, and of highly specialized complex diagnostics. As part of this collaboration, the Gaslini Institute proposes itself as an accredited structure responsible for the diagnostic consequences that will derive from the large-scale study and with innovative methodologies of genetic pathologies in developmental age. The increased reliability and productivity of genomic technologies together with the reduction of costs and new knowledge on the genetic basis of human pathologies, suggest a transition of genomics from the research environment to clinical practice in the near future.  The Giannina Gaslini Institute has also a “Pediatric Clinical Trials Service” (acronym SSCP) to support ongoing clinical research activities in the Institute and to implement new projects.

Società Italiana di Farmacologia (SIF)

Founded in 1939, the Italian Society of Pharmacology (SIF) was recognized in 1996 as a non-profit scientific association by the Ministry of University and Scientific and Technological Research. SIF is a member of IUPHAR (International Union of Pharmacology) and EPHAR (Federation of European Pharmacological Societies). The current membership consists of approximately 1,200 Members. Recently within the Society, a Working Group on Pediatric Pharmacology (http://www.sifweb.org/la_societ%C3%A0#Gruppi_di_lavoro) has been established, joined by 44 pharmacologists. To respond to the increasing interest of individual researchers, scientific societies and pharmaceutical companies, the Pediatric Pharmacology Working Group aims to address the many specificities characterizing clinical pediatric research. Among these: (i) the need to develop protocols designed for specific age groups, as protocols used for the adult population may be inadequate for children, as well as those for older children may be inadequate for infants and newborns; (ii) the consolidation of competences for conducting early stage pediatric clinical trials (phase I / II, pharmacokinetic / pharmacodynamic studies) and the development of pharmacokinetic models suitable for children; (iii) the identification of biomarkers and new non-invasive parameters for drug efficacy assessment; (iv) the development of formulations suitable for children of various ages; (v) the application of innovative biostatistics methodologies; (vi) new preclinical protocols for the development of pediatric medicines and ATMP and (vii) the design of studies to evaluate long-term efficacy and safety. These activities are also implemented within SIF via the involvement of other existing SIF working groups, such as that SIF4RARE (Rare disease and Orphan Drugs) and Neuropsycopharmacology for specific projects on pediatric diseases.

 
 

TEDDY European Network of Excellence for Paediatric Research (TEDDY)

TEDDY European Network of Excellence for Paediatric Research is a scientific organization arisen from the Network of Excellence named “Task-force in Europe for Drug Development for the Young”, created in 2005 within the TEDDY project (LSHB-CT-2005-005216) funded under the Sixth Framework Programme of the European Commission with the aim to favour the integration of the paediatric research activities, a social awareness on the importance of the paediatric medicines across and the improvement of the relevant ethical, legal and regulatory frameworks. The main objective of the TEDDY Network is to promote children tailored medicine, guarantying children rights and well-being. It addresses methodological, ethical, legal and social issues of research (from the preclinical phase to clinical evaluations and medicines utilization, access policies and education) and implements good practices and tools to plan, design and perform clinical studies at national and international level. TEDDY is a category 1 network member of Enpr-EMA, the European Network of Paediatric Research at the European Medicines Agency and registered at ENCePP (the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance). TEDDY can count on the multidisciplinary expertise and collaboration of researchers, scientists, health experts and paediatric clinical research centres working together to identify the most appropriate research tools and procedures that reflect the specificity of the paediatric patient population in compliance with children rights. It collaborates with existing paediatric networks, and research organizations with the goal to promote and foster scientific and technological excellence in an ethically sound paediatric research in Europe. Moreover, TEDDY has developed a European Paediatric Medicines Database (EPMD) including information on medicines approved for children by EMA (indication, dosages, by age populations, studies and trials). TEDDY has also developed a long-standing experience and expertise focused on innovative methodologies and ethical, legal, and societal issues relevant for paediatric research also developed within EU funded projects and in collaboration with European institutions such as the European Medicine Agency, the European Commission, and the Council of Europe.  TEDDY is particularly engaged in increasing awareness on the topics of paediatric research and encouraging the participation of children in research. TEDDY provides support, education and information to patients and families regarding their participation in clinical trials. To this aim, different tools and educational resources with different format (e.g., serious games, comics, videos on the development of clinical trials) have been developed. Regarding the patients’ empowerment and the involvement in the paediatric clinical research, TEDDY has promoted the creation of several Young Persons Advisory Group (YPAG) in Italy, Albania, Greece, Romania and Czech Republic. TEDDY is also strongly involved in the initiative of the Council of Europe on strengthening children’s participation in the decision-making process on matters regarding their health.Thanks to the international recognition and the wide geographical representativeness, TEDDY is able to provide the paediatric community with: advice on Paediatric Medicine Development Issues; advice on relevant Ethical, Legal and Societal Issues (ELSI) in a children/fundamental rights perspective (e.g. data protection, right to health protection…); advice on children empowerment and engagement in paediatric studies; easy dissemination of relevant information; documents and recommendations in the sector of paediatric study methodology and ELSI issues.

 
 

Università degli Studi di Cagliari (UniCa)

UniCa has a broad range of expertise and research, which extends from basic and applied research to patient-centered clinical research. Among the activities of interest for EPTRI, researchers at the Department of Biomedical Sciences, and at his Section of Neuroscience and Clinical Pharmacology (SNCP) in particular, are involved in basic and clinical research in neuroscience and in neuropsychopharmacology. They use in vivo and in vitro animal models and neurophysiological, neurochemical and behavioural techniques to study the neurobiology of brain systems involved in neurodevelopmental disorders, such as autism and schizophrenia, the effect of drugs of abuse and other environmental insults in vulnerable periods of development (prenatal life and adolescence). Researchers are actively involved in drug discovery in pharmacology by utilizing state-of-the-art equipment located in laboratories and in the Core Research Facility (CeSAR) at UniCa. Furthermore, within the SNCP, the Unit of Child and Adolescent Neuropsychiatry, with both in-patient and out-patient facilities, is actively involved in industry-sponsored and investigator-initiated clinical research in a wide range of developmental neuropsychiatric disorders, also supported by specific EU Health Grants (PERS, STOP, ADDUCE, MATRICS, Conect4Children). In collaboration with the Institute for Genetic and Biomedical Research (CNR-IRGB) and the Institute of Neuroscience (IN-CNR), researchers pursue several research lines focused on the genetics of Sardinian population, and on neurobiology of neuropsychiatric disorders. Additionally, other research activities within UniCa span from neuroscience, neurology, cardiology, vascular diseases, nutrition, infection diseases, oncology, immunology, genetics, psychology and psychiatry.

 
 

Università degli Studi di Milano (UniMi)

The Pharmaceutical Technology and Regulatory Affairs Unit and the Laboratory of Cellular and Molecular Biology Applied to Neurodevelopmental Diseases are active parts at the University of Milan that has a long-standing expertise in neurology, immunology, oncology, nutrition, cardiology, vascular diseases and infectious diseases. The Laboratory of Cellular and Molecular Biology Applied to Neurodevelopmental Diseases focuses its research activities on mechanisms leading to neurodevelopmental disorders and autism spectrum disorders. Basic, translational and pre-clinical studies related to Rett syndrome and CDKL5 deficiency disorders are currently active. Along the year, the Laboratory has developed several cellular (primary neurons and astrocytes, neurons derived from neuroprecursor stem cells) and animal models for the study of Rett syndrome and the CDKL5 deficiency disorder. Studies on stem cell biology including neural progenitor cell transplantation considered as a promising therapeutic strategy for the treatment of a long list of neurodegenerative disorders such as multiple sclerosis, ischemic stroke, spinal cord injury, Huntington’s and Parkinson diseases. Using animal models and animal embryonic stem cells, the Laboratory also performs studies aimed at identifying and testing novel drug targets for neonatal populations. Further, in order to detect novel therapeutic targets for pediatric neurological disorders, they are developing high throughput cell-based platforms for drug-screening based on transcriptional profiles; efficacy of selected candidates will be then tested in vivo at different developmental ages including perinatal animals. At the Pharmaceutical Technology and Regulatory Affairs Unit, the research concerns the design of topically applied dosage forms, such as semisolids and (trans) dermal patches. The researchers perform the evaluation of skin penetration and permeation of active ingredients by means of ex vivo methodologies in order to study passive strategies, such as chemical enhancers and supersaturation, for enhancing absorption. They are also focused on optimizing test procedures for the quality control of these formulations. The researchers also perform formulation study and development of microparticulate systems or orodispersible film, intended to be used for personalized medicines, and mucoadhesive dosage forms for the treatment of buccal pathologies. The ex vivo transmucosal permeation and the effects of the active ingredient on in vitro bioadhesive properties are also studied. Finally, the research also involves the pharmaceutical legislation and regulatory affairs connected to the production, distribution, and dispensation of medicinal products and other healthcare products.

 
 

Università degli Studi di Padova (UNIPD)

At UNIPD, several departments perform research on the human health to identify the mechanisms of diseases and medicines adapted to cure them. The research is mainly focused on the study of the mitochondrial dynamics, intracellular ion channels, circadian rhythms, sexual signals and the interaction between the body and the environment thanks to the collaboration of experts from different fields like genomics, bioinformatics, etc. The study of the signals is considered crucial to understand the healthy and non-healthy functioning of the living organisms, populations and communities. Moreover, some of the research lines are on neurofibromatosis for the identification and molecular characterisation of new mutations; cytogenetics to characterise the chromosomal rearrangements; molecular biology which validate splicing mutations using hybrid mini genes, characterise genetic anomalies, and study foetal DNA from maternal plasma for the diagnosis of genetic diseases; mitochondrial physiopathology which study the molecular basis and the therapies for the defects in the mitochondrial respiratory chain; the development of animal models like S. cerevisiae, C. elegans and zebrafish; and the clinical teratology regarding woman that entered in contact with teratogenic substances. Other interesting fields applied to paediatric research are epidemiology, clinical research, innovative therapies, applied biology, applied neuroinformatics and strategic cardiovascular engineer. At UNIPD are also available competences in pathogenesis of infective diseases, infections and immunity, cancer biology and the application of advanced therapies on human tumors, stem cell research, and regenerative medicine. There is also the identification of new molecular targets for tailored therapies aimed at identifying new drug/small molecule innovative therapies validated in vitro and in vivo.

 
 

Università degli Studi di Parma (UniPr)

The researchers work in the fields of cardiology and vascular diseases, endocrinology, hepatology, immunology, oncology, haematology, neurology, rheumatology, genetics, pneumology, respiratory diseases, infectious diseases and musculoskeletal disorders. At the Department of Biomedical-Biotechnology and Translational Science, the researchers are engaged on several projects, they study the effects of tetracalcium phosphate and biphasic tetracalcium phosphate powders on human osteoblasts, printed 3D bone in surgery for planning and training, Crohn diseases and its resistance effects on novel drugs, and diabetes mellitus. Moreover, they study the mutation of NOTCH1 gene in the T-cell of lymphoblastic leukaemia discovering its therapeutic effect. They successfully performed the treatment of granular cell tumour on the tongue with diode laser. At the Department of Clinical and Experimental Medicine, researchers study novel approaches to improve the killing of myeloma cell by monoclonal antibodies, perform animal studies on juvenile animal studies (e.g., rat) and in vitro studies with the aims to understand how aptamers recognize fibronectin and confer improved bioactivity to biomaterials promoting new bones formation. They are active in genetic field with several projects that concern genotyping, phenotyping, genetic variations, genetic mutations, and gene polymorphism. At the Department of Life Science, researchers perform identification and experimental validation of novel drugs using zebrafish model and yeast model, and study nanostructure for the detection of bacteria, the application of genetic studies to therapy for hereditary optic neuropathies, and spike protein of SARS-CoV-2 and its interaction on human cells.

Università di Bari Aldo Moro (UniBa)

It is a multidisciplinary university, which offers a wide range of academic programs in different disciplinary fields. At the Department of Pharmacy and Pharmaceutical Sciences, the researchers perform educational and research activities on drug design, parallel synthesis of focused molecular libraries, in vitro target validation, in silico predictive toxicology, pre-formulation and formulation studies in paediatric, and prospective repurposing development process. They develop palatable oral liquid formulations of insoluble drugs using complexing excipients such as cyclodextrins, which improve both aqueous solubility and stability of interacting drugs. The unit has all the facilities to produce microparticulate-based formulations by prilling and spry-drying technologies, useful to mask the unfavourable taste of active pharmaceutical principles, increasing the acceptability of the paediatric patients. Innovative tailored formulation based on nanotechnology and 3D printing are also explored in order to generate patient centric dosage forms. Moreover, this department offers a wide expertise and technical equipments for the production and optimisation of different pharmaceutical formulations, and a computational laboratory for molecular modelling of drug and excipients.

Università di Milano Bicocca (Bicocca)

It is a multidisciplinary university, which offers a wide range of academic programs in different disciplinary fields. The research areas include life science, physical sciences and engineering, social sciences and humanities. The researchers work in cardiology and vascular diseases, neurology, hepatology, haematology, pneumology, respiratory diseases, oncology, rheumatology, infectious diseases, immunology and genetics. The researchers study paediatric development mechanisms in oncology (e.g., neuroblastoma, epigenetics, glioma), but also new treatment based on immunotherapy (e.g., natural kill cell mediated) and chemotherapy. They perform in vitro and in vivo studies using animal models (e.g., rat), proteomics to study TRIP8b protein in brain. Moreover, they use genetics approaches to identify shared polygenic risks and causal associations in amyotrophic lateral sclerosis. They also investigate glioma microenvironment using molecular and imaging biomarkers to overcome treatment resistance. They study the effects of different type of molecules (e.g., crizotinib) in patients with anaplastic lymphoma kinase positive refractory to chemotherapy. They also perform stem cell and regenerative medicine with tissue engineering approaches. Recently, they study the infective mechanism, the effects in children, adult and older of COVID-19, and compare and analyse data using genetics approaches.

Università di Pavia (UniPavia)

The Pediatric Unit is an active part of the Department of Clinical, Surgical, Diagnostic and Pediatric Sciences of the University of Pavia. The research activities of the Pediatric Unit cover most of the areas of pediatric translational science. The research unit takes part in several projects of the “current research” programs of Fondazione IRCCS Policlinico San Matteo, some of which aimed to investigate new approaches for diagnosing pediatric food allergies and the immune system of the upper airways in children. Also, pediatric researchers are currently working on basic, translational, and clinical studies related to the pathophysiology and management of pediatric allergies (asthma, atopic dermatitis, IgE-mediated food allergy, allergic rhinitis). Notably, an active field of research is based on identifying clinical phenotypes and potential endotypes of severe asthma to develop an approach aimed at personalized medicine and biological therapies in pediatrics. The researchers are also working on the clinical and immunological assessment of emerging allergic disorders, such as eosinophilic gastrointestinal diseases, identifying novel molecular targets and potential non-invasive diagnostic tools. Recently, in collaboration with the National Institute of Health (NIH), pediatric researchers of the University of Pavia are performing studies to discover possible immunological mechanisms underlying the different clinical expression of COVID-19 diseases in children. Researchers: Prof. Gian Luigi Marseglia, e-mail: [email protected]; Prof. Amelia Licari: amelia.lica[email protected].

 
 

At TorVergata University, the research is mainly oriented on cutting-edge themes in the study of life like molecular mechanisms of diseases like neurodegenerative diseases and regulation of processes of cancerogenesis. Specifically, a huge interest is dedicated to the study of planned cell death, autophagy and implication of mitochondria, and development and physiopathology of nervous system. The laboratories apply tecniques like bioinformatics from protein and DNA 3D structure prediction to shotgun metagenomics. The researchers also study animal models for infectious diseases and experimental oncology, development of vaccines and development of new materials to be used in prosthesis, interaction host-pathogen in tuberculosis, HIV, HBV and HCV, and identification of immunotherapeutic strategies for infections. Other lines of research are related to biochemistry (with study on antioxidant enzymes, redox signals, and dysregulation of adipose tissues), genetics (on regulation of mRNA coding for ribosomes), virology (new strategy for the control of viral replication, stress answers in the viral infection and cancer, cellular signalling in infections) and microbiology (which studies environmental contamination of antibiotics).