Stefano Ferrari, PhD
Head of R&D, Fondazione Banca degli Occhi del Veneto, Venice, ITALY
Education & work experience:
Stefano Ferrari was born in Verona (Italy) on 9/6/1970. In 1995, he took his degree in Biological Science at the University of Padova, Italy. In 1996, he was awarded a 3-month short-term fellowship from the “Association Francaise pour la Lutte contre la Mucoviscidose (AFLM)” and he moved to the “genetic chemistry” laboratory directed by Prof JP Behr at the Faculty of Pharmacy in Strasbourg (France). In November 1996 he began his 3-year PhD-studentship on a project focused on gene therapy approaches for cystic fibrosis at the Dept. of Paediatrics/CRIBI Biotechnology Centre of the University of Padova. In 1998, after being awarded a fellowship from the UK Cystic Fibrosis Trust, he moved to the Department of Gene Therapy of the Imperial College School of Medicine (London, UK) directed by Prof. EWFW Alton. He remained at the Imperial College as Research Associate until 2003 and worked on projects assessing the efficiency of viral/non-viral vectors for the correction of the genetic defects in cell/animal models of Cystic Fibrosis. In March 2003, he was offered a full-time position as research scientist at the Veneto Eye Bank Foundation (Venice, Italy) and has since worked on cell/gene therapy projects for the correction of disorders affecting the skin (e.g., epidermolysis bullosa) and the ocular surface (e.g., limbal stem cell deficiency). In 2006 he actively took part to the first gene therapy clinical trial on a patient affected by Junctional Epidermolysis Bullosa through transplantation of genetically corrected epidermal stem cells (Mavilio F, Nature Medicine 2006; 12: 1397-1402). He has been one of the Editors of the “Progress & Prospects” review series on Gene Therapy (2002-2011) and co-author of 2 patent applications describing the use of paramyxoviruses for gene therapy of cardiovascular disorders and cystic fibrosis. He is author of 6 book chapters and more than 50 peer-reviewed papers in international journals.
- Cell and gene therapy approaches for disorders of the ocular surface
- Rare genetic disorders of the eye (cornea and retina)
- Advanced therapy medicinal products and GMP regulations
- Eye banking and biobanking
- Corneal regeneration by means of cell-, gene- and drug-based therapies
- Molecular and genetic bases of Age Related Macular Degeneration
The Veneto Eye Bank Foundation (FBOV; http://research.fbov.org) is a non-profit organization and one of the leading eye banks in Europe (more than 4000 ocular tissues collected in 2013). A Research Centre and a Cell Factory for manipulation of corneal epithelial stem cells for clinical applications were established in 2002. From 2003 to 2008, 166 patients affected by Limbal Stem Cell Deficiency (LSCD) and unresponsive to previous medical and surgical treatments (such as corneal transplantation) received autologous cultured limbal stem cells, isolated and expanded in vitro from 0,5-1mm3 biopsies obtained from the healthy fellow eye. Rates of success ranged between 60% to 80%, when partially successful cases were also considered (Di Iorio et al., The Ocular Surface 2010; 8: 146-153) and clinical results reported by Colabelli Gisoldi et al. (Cornea 2010; 29: 715-722), Rama et al. (N. Engl. J. Med. 2010; 363: 147-155) and Marchini et al. (Clinical and Experimental Ophthalmology 2012; 4: 255-267). In 2006, FBOV and colleagues at the University of Modena reported the first-ever successful gene therapy trial for a rare genetic disorder of the skin by transplanting skin grafts made of genetically corrected autologous cultured epithelial stem cells onto a young patient with Junctional Epidermolysis Bullosa (Mavilio et al., Nature Medicine 2006; 12: 1397-1402). Following the European guidelines that have classified stem cell-based therapies as “medicinal products”, GMP standards were implemented and further 26 patients transplanted in 2012 and 2013. Research activities carried out in our Research Centre aim at developing new therapeutic strategies and protocols that would implement the pipeline of medicinal products to be used in clinical trials. Current research projects are aimed at:
- Replacing the scaffolds currently used for limbal stem cell transplantation (fibrin glue and amniotic membrane) with corneal lenticules for anterior lamellar keratoplasty. This would allow to replace both the stroma and the epithelium at once, with just one single surgery.
- Evaluating whether oral mucosa and conjunctiva stem cells can differentiate into corneal epithelial cells, thus allowing patients with bilateral LSCD to be transplanted with autologous cells and avoid the need for immunosuppressive therapy and the risks associated with allograft immunologic rejection.
- Evaluating the capacity of Bone Marrow Mesenchymal Stem Cells to differentiate into corneal epithelial cells and generate a corneal epithelium in vitro, as a potential treatment for patients with bilateral LSCD.
- Assessing whether p63 gene silencing approaches could lead to the correction of the genetic defect in corneal stem cells and path the way for the therapeutic transplantation of genetically corrected corneal stem cells for the treatment of LSCD in patients EEC syndrome.
- Employing gene therapy approaches to treat corneal ulcers and accelerate the wound healing after transplantation of corneas, in order to improve the allograft survival rates.
- Validating a human cornea perfusion apparatus that mimic the natural eye environment and allow to test the effects of drugs, eye drops, etc. on the corneal surface and the underlying stroma.
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Fondazione Banca degli Occhi del Veneto ONLUS
Via Paccagnella 11
c/o Padiglione G Rama
Fondazione Banca degli Occhi del Veneto ONLUS
30174 Zelarino (Venezia) Italy