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Unlocking Precision Gene Therapy

Total Cost €


EC-Contrib. €






 UPGRADE 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.

upgrade    efficiency    limitations    cure    mutagenesis    therapy    solutions    editing    risk    realize    precise    storage    hematopoiesis    conventional    immune    retina    heart    complementing    insertional    seven    profile    paradigmatic    hypercholesterolemia    treatment    hurdles    models    limited    portable    clinical    medicinal    blood    combine    amps    correction    silence    wasting    full    off    viral    precision    amp    remarkable    basis    reached    activate    skeletal    diseases    liver    site    tissue    safety    strategies    stringently    medical    indications    disease    vehicles    disorders    vectors    efficacy    durability    overcome    muscle    unmet    genome    radical    cell    market    evasion    entering    tissues    definitive    specificity    generate    cellular    sequence    groups    opening    insertion    incurable    treatments    transfer    technologies    poor    gene    pathogenesis    otherwise    shown    tools    transgene    patient    components    epigenome    tackling    disruptive    scope    recode    broaden    versatile    validated    prototypes   

Project "UPGRADE" data sheet

The following table provides information about the project.


Organization address
address: VIA VARESE 16/B
city: ROMA
postcode: 185

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 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


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
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
6    UNIVERSITE DE NANTES FR (NANTES CEDEX 1) participant 1˙107˙500.00


 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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The information about "UPGRADE" are provided by the European Opendata Portal: CORDIS opendata.

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