Hologene 7 as a model of development of an advanced therapy based on genetically corrected stem cells - Project POR-FESR 2014-2020
The project involves the excellences in the field of regenerative medicine and genomics for the gene therapy of Epidermolysis Bullosa (EB), a severe genetic disease, better known as Butterfly Children Disease, that makes their skins and mucosa extremely weak and exposed to serious blisters and lesions. The project exploits an interdisciplinary approach that encompasses all the phases of patient care, from diagnosis to treatment, making therefore Modena an international pivotal center for EB.
The strategic goal of the project is to strengthen the competitive supply of ER regenerative medicine through the enhancement of the industrial research of the High Tech Network laboratories and the standardization of the development and application model to make it exportable to other genetic pathologies of coating epitels, In collaboration with local businesses. In this respect, the specific objectives of the project are: - the development of a product and a clinical protocol of exogenous gene therapy by genetically modified epidermal stem cells for the treatment of the Ebdectomy form; - Optimization of the massively parallel sequencing platform for EB's molecular diagnosis and the launch of the authorization routine for routine diagnosis and for the provision of a genetic counseling service for families with EB at POLICLINICO; - the transfer of clinical know-how to the medical staff of the POLYLINIC Dermatology Department, in collaboration with the EB-Haus Austria in Salzburg for the specific management of the patient EB; - enhancing project outputs and the participatory approach adopted as a model for the treatment of other rare diseases and as a pivot for the creation of a network of Hub & Spoke centers that, with the implementation of an EB-Hub in Modena.
The project is coordinated by CIDSTEM UNIMORE. The pre-clinical industrial research part is conducted by CIDSTEM at the Regenerative Medicine Center "Stefano Ferrari" of the University of Modena and Reggio Emilia. The diagnostic part is handled by CGR UNIMORE, which will develop an early diagnosis service. The clinical part is cared for by the complex structure of Dermatology of the POLICLINICO of Modena in collaboration with JSB SOLUTIONS. While the production of transgenic epidermis will be performed at the CIDSTEM GMP workshop in conjunction with the university spin-off HOLOSTEM ADVANCED THERAPIES and PHARMACEUTICAL CHURCHES. The DEMOCENTER Foundation of Modena, which deals with the dissemination and dissemination of results, completes the partnership.
The Interdepartmental Centre for Stem Cells and Regenerative Medicine (CIDSTEM) is located into the Centre for Regenerative Medicine “Stefano Ferrari” of the University of Modena e Reggio Emilia. The centre has a pivotal role in epithelial stem cell-based advanced therapies and has a 2000 sq meters GMP facility (AIFA Authorization n. aM - 169/2014). Reference person: Stefania Bettinelli firstname.lastname@example.org
The Center for Genomic Research (CGR) was created to coordinate and promote the very high individual skills of the genomics research groups of the University of Modena. The Center provides services in the fields: medical, biotech, pharmaceutical and food industry. It offers industrial research, clinical and technological research to local health departments, universities, research institutions and entrepreneurship, particularly with regard to small and medium-sized enterprises (S.M.E.). Persona di riferimento: Enrico Tagliafico email@example.com
The Hospital-University Polyclinic of Modena is a teaching hospital of the Faculty of Medicine of the University of Modena and Reggio Emilia, with the aim of efficiently producing and delivering health care services, from basic to those of high complexity, scientifically proven effective and appropriate, paying attention to the psychological and social needs of the assisted persons. The Polyclinic ensures adequate welfare support and active participation in training activities of the Faculty of Medicine and promotes research activities, with particular reference to actions with significant and positive impact on the activity of care and training. Reference person: Filippo Martani firstname.lastname@example.org
Holostem Terapie Avanzate is the first Italian biotechnological company devoted to the development, manufacture, registration and distribution of Advanced Therapy products based on cultures of epithelial stem cells both for cell therapy and gene therapy. The main aim of Holostem Terapie Avanzate is to promote epithelial stem cell-based Regenerative Medicine for patients with no alternative therapeutic solutions. Persona di riferimento: Stefania Bettinelli email@example.com
Chiesi Farmaceutici is an international group oriented to research, with over 80 years of experience and headquartered in Parma. Chiesi researches, develops and markets innovative drugs in respiratory therapeutic areas, neonatology, rare diseases and of specialized medicine in which is in progress the development of advanced products in the field of proteomics, gene and stem cells therapy. In 2015 the company has developed a turnover of 1.46 billion Euros, an increase of 9.4% compared to 2014. Reference person: Roberta Buti firstname.lastname@example.org
JSB Solutions is a consulting company specialized in compliance, process optimization and development of IT solutions in the pharmaceutical and industrial sectors. Accredited in 2016 as a CRO, offers today a range of services in the following areas: Clinical Studies, GMP and GCP Quality Assurance support, Regulatory Affairs, Computer System Validation, Equipment & Utilities Validation, Manteinance Management, Training, QA & QC support and Software Development. Reference person: Valentina Grazzini email@example.com
Democenter-Sipe Foundation is an innovation center of the High Technology Network of Emilia-Romagna Region. Democenter-Sipe supports innovation acting as a facilitator and interface between research centers, public bodies and companies, dealing with the dissemination and transfer of the research activities and results. Reference person: Laura Aldrovandi firstname.lastname@example.org