Project description DEENESFRITPL Advanced gene-editing tools for safer gene therapy Gene therapy offers a promising approach to treat incurable diseases by silencing, activating or recoding specific genes. In addition to conventional gene transfer strategies, newly emerged gene-editing technologies provide the potential for precise manipulation of the genome. The EU-funded UPGRADE project aims to address certain gene therapy limitations associated with insertional mutagenesis, off-target effects, or limited efficiency. Researchers will develop disruptive new technologies for precision gene and epigenome editing, as well as for gene insertion. Following stringent validation, the project will lead to advanced medicinal products and versatile tools that could be used for a number of diseases. Show the project objective Hide the project objective Objective Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications. Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles, including: risk of insertional mutagenesis by gene transfer vectors, limited efficiency and durability of some gene correction strategies, off target effects of editing tools, poor tissue targeting and immune response to editing components and delivery vehicles. UPGRADE will offer radical new solutions to overcome these hurdles. We will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion, and stringently characterize their specificity and cellular responses. We will combine these improved technologies with advanced viral and non-viral vectors enabling cell/tissue targeting and immune evasion, to generate prototypes of advanced medicinal products (AMP). The safety and efficacy profile of each AMP will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia) paradigmatic for unmet medical need and potential long-term cure, if the limitations of current gene therapy strategies are overcome. These AMPs represent versatile products portable to the treatment of several other diseases because of related pathogenesis or correction strategies, thus providing the basis for tackling diseases affecting large patient groups. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesbasic medicineimmunologymedical and health sciencesclinical medicineophthalmologymedical and health sciencesclinical medicinehematology Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-BHC-09-2018 - Innovation platforms for advanced therapies of the future Call for proposal H2020-SC1-BHC-2018-2020 See other projects for this call Sub call H2020-SC1-2018-Single-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator FONDAZIONE TELETHON ETS Net EU contribution € 3 412 968,75 Address Via varese 16/b 00185 Roma Italy See on map Region Centro (IT) Lazio Roma Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Participants (10) Sort alphabetically Sort by Net EU contribution Expand all Collapse all Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. INNOVAVECTOR SRL Italy Net EU contribution € 110 000,00 Address Via campi flegrei 34 80078 Pozzuoli See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Sud Campania Napoli Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. OSPEDALE SAN RAFFAELE SRL Italy Net EU contribution € 1 365 625,00 Address Via olgettina 60 20132 Milano See on map Region Nord-Ovest Lombardia Milano Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. NEXT GENERATION DIAGNOSTIC SRL Italy Net EU contribution € 64 375,00 Address Via campi flegrei 34 80078 Pozzuoli See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Sud Campania Napoli Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSIDAD POMPEU FABRA Spain Net EU contribution € 2 767 377,50 Address Placa de la merce, 10-12 08002 Barcelona See on map Region Este Cataluña Barcelona Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITA DEGLI STUDI DI TRENTO Italy Net EU contribution € 1 333 798,75 Address Via calepina 14 38122 Trento See on map Region Nord-Est Provincia Autonoma di Trento Trento Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 TECHNISCHE UNIVERSITAET DRESDEN Germany Net EU contribution € 1 107 000,00 Address Helmholtzstrasse 10 01069 Dresden See on map Region Sachsen Dresden Dresden, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 VRIJE UNIVERSITEIT BRUSSEL Belgium Net EU contribution € 1 328 412,50 Address Pleinlaan 2 1050 Bruxelles / brussel See on map Region Région de Bruxelles-Capitale/Brussels Hoofdstedelijk Gewest Région de Bruxelles-Capitale/ Brussels Hoofdstedelijk Gewest Arr. de Bruxelles-Capitale/Arr. Brussel-Hoofdstad Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 NANTES UNIVERSITE France Net EU contribution € 1 182 500,00 Address 1 quai de tourville 44000 Nantes See on map Region Pays de la Loire Pays de la Loire Loire-Atlantique Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Germany Net EU contribution € 1 106 062,50 Address Geschwister scholl platz 1 80539 Muenchen See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 1 125,00 THE GENERAL HOSPITAL CORPORATION United States Net EU contribution € 1 217 710,00 Address Fruit street 55 02114 Boston ma See on map Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
