UPGRADE SIGNED
Unlocking Precision Gene Therapy
Total Cost €
0EC-Contrib. €
0Partnership
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0UPGRADE project word cloud
Explore the words cloud of the UPGRADE project. It provides you a very rough idea of what is the project "UPGRADE" about.
Project "UPGRADE" data sheet
The following table provides information about the project.
| Coordinator |
FONDAZIONE TELETHON
Organization address contact info |
| Coordinator Country | Italy [IT] |
| Total cost | 14˙996˙955 € |
| EC max contribution | 14˙995˙830 € (100%) |
| Programme |
1. H2020-EU.3.1.3. (Treating and managing disease) |
| Code Call | H2020-SC1-2018-Single-Stage-RTD |
| Funding Scheme | RIA |
| Starting year | 2019 |
| Duration (year-month-day) | from 2019-01-01 to 2023-12-31 |
Partnership
Take a look of project's partnership.
| # | ||||
|---|---|---|---|---|
| 1 | FONDAZIONE TELETHON | IT (ROMA) | coordinator | 5˙027˙968.00 |
| 2 | UNIVERSIDAD POMPEU FABRA | ES (BARCELONA) | participant | 2˙767˙377.00 |
| 3 | UNIVERSITA DEGLI STUDI DI TRENTO | IT (TRENTO) | participant | 1˙333˙798.00 |
| 4 | VRIJE UNIVERSITEIT BRUSSEL | BE (BRUSSEL) | participant | 1˙328˙412.00 |
| 5 | THE GENERAL HOSPITAL CORPORATION | US (BOSTON MA) | participant | 1˙217˙710.00 |
| 6 | UNIVERSITE DE NANTES | FR (NANTES CEDEX 1) | participant | 1˙107˙500.00 |
| 7 | TECHNISCHE UNIVERSITAET DRESDEN | DE (DRESDEN) | participant | 1˙107˙000.00 |
| 8 | LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN | DE (MUENCHEN) | participant | 1˙106˙062.00 |
Map
Project 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.
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